Nine accepted abstracts demonstrate broad potential of the companys HSC gene therapy approach to treat severe neurodegenerative diseases and immunological disorders

BOSTON and LONDON, Oct. 13, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the acceptance of nine abstracts at the upcoming European Society of Gene & Cell Therapy Congress (ESGCT) taking place virtually from October 19-22.

Clinical and pre-clinical data from across the companys hematopoietic stem cell (HSC) gene therapy portfolio will be featured in two oral and seven poster presentations, including an update on the ongoing proof-of-concept study of OTL-201 for the treatment of Mucopolysaccharidosis type IIIA (MPS-IIIA, also known as Sanfilippo syndrome type A), pre-clinical data from OTL-204 in frontotemporal dementia (FTD), as well as proof-of-principle for longitudinal monitoring of vector integration sites using Liquid Biopsy Integration Site sequencing (LiBIS-seq).

Additionally, Orchards scientific advisory board member and clinical collaborator Alessandra Biffi, M.D., professor of pediatrics, University of Padua and chief of the Pediatric Onco-hematology Unit of Padua Hospital, will be giving an invited presentation on the HSC gene therapy landscape for the treatment of neurodegenerative disorders, which will include an overview of several of the companys investigational programs.

The presentations are listed below, and the full program is available online on the ESGCT website. All times are Central European Summer Time (CEST).

Oral Presentation Details:

Haematopoietic reconstitution dynamics of mobilized peripheral blood- and bone marrow-derived haematopoietic stem/progenitor cells after gene therapyPresenting Author: Andrea Calabria, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: OR049Date/Time: Friday, October 22, 2021 at 10:01 CEST

Longitudinal monitoring of vector integration sites in in vivo GT approaches by Liquid-Biopsy-Integration-Site-SequencingPresenting Author: Daniela Cesana, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: OR058Date/Time: Friday, October 22, 2021 at 12:46 CEST

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Poster Presentation Details:

All posters will be available on demand starting October 19, 2021 on the ESGCT website.

Development of an ex vivo Gene Therapy for Frontotemporal Dementia (FTD)Presenting Author: Yuri Ciervo, Ph.D., division of pediatric Hematology,Oncology and Stem Cell Transplantation, Womans and Child Health Department, University of Padova, Padova, ItalyAbstract Number: P077

Optimized Lentiviral Transduction Process for ex vivo CD34+ Hematopoietic Stem Cell Gene Therapy Drug Product ManufacturePresenting Author: Saranya Elavazhagan, Orchard TherapeuticsAbstract Number: P271

Clinical Trial Update: Ex-vivo autologous stem cell gene therapy in MPSIIIAPresenting Author: Brian Bigger, Ph.D., University of ManchesterAbstract Number: P361

Dissecting bone remodelling mechanisms and hematopoietic stem cell gene therapy impact in Mucopolysaccharidosis type I Hurler bone defectsPresenting Author: Ludovica Santi, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: P157

Hematopoietic reconstitution and lineage commitment in HSC GT patients are influenced by the disease backgroundPresenting Author: Andrea Calabria, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: P181

Kinetics and composition of haematopoietic stem/progenitors mobilized cells upon G-CSF and Plerixafor administration in transplant donor or patients undergoing autologous gene therapyPresenting Author: Luca Basso-Ricci, San Raffaele Telethon Institute for Gene TherapyAbstract Number: P174

Role of peripheral blood circulating haematopoietic stem/progenitor cells during physiological haematopoietic maturation and after gene therapyPresenting Author: Pamela Quaranta, San Raffaele Telethon Institute for Gene TherapyAbstract Number: P186

About Orchard TherapeuticsAt Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patients own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard has a deep pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About OrchardInvestors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaBenjamin NavonDirector, Corporate Communications+1 857-248-9454Benjamin.Navon@orchard-tx.com

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Orchard Therapeutics Outlines Comprehensive Presence at the European Society of Gene & Cell Therapy Congress - Yahoo Finance

Bone Marrow Stem Cells Comments Off on Orchard Therapeutics Outlines Comprehensive Presence at the European Society of Gene & Cell Therapy Congress – Yahoo Finance | October 16th, 2021

TEL AVIV, Israel, Oct. 13, 2021 /PRNewswire/ --BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a late clinical-stage biopharmaceutical company focused on oncology, today announced positive results from a pharmacoeconomic study evaluating the cost-effectiveness of using investigational drug Motixafortide as a primary stem cell mobilization (SCM) agent on top of granulocyte colony stimulating factor (G-CSF), versus G-CSF alone, in multiple myeloma patients undergoing autologous stem cell transplantation (ASCT). The study was performed by the Global Health Economics and Outcomes Research (HEOR) team of IQVIA, and was a pre-planned study conducted in parallel with the GENESIS Phase 3 trial. These results, together with the highly significant and clinically meaningful data from the GENESIS trial, strongly support the potential use of Motixafortide, on top of G-CSF, as the standard of care in SCM for ASCT.

The study concluded that the addition of Motixafortide to G-CSF (the current standard of care) is associated with a statistically significant decrease in health resource utilization (HRU) during the ASCT process, compared to G-CSF alone. Based on the significantly higher number of mobilized cells and the lower number of apheresis sessions, lifetime estimates show quality-adjusted-life-year (QALY) benefits and net cost savings of ~$17,000 (not including the cost of Motixafortide), versus G-CSF alone. The study findings, combined with model estimates, suggest that the use of Motixafortide, on top of G-CSF, as the standard of care in mobilization for ASCT, could be a cost-effective option in the US, based on accepted willingness-to-pay (WTP) values for healthcare payers.

"The compelling cost savings identified by this rigorously designed study strongly support the Company's view that Motixafortide, in combination with G-CSF, can become the new standard of care as an upfront, or primary, therapy for all multiple myeloma patients undergoing autologous stem cell transplantation," stated Philip Serlin, Chief Executive Officer of BioLineRx. "Based on data from the GENESIS trial showing that nearly 90% of patients collected an optimal number of cells for transplantation following a single administration of Motixafortide and in only one apheresis session, versus less than 10% for G-CSF alone, the pharmacoeconomic study demonstrates that use of Motixafortide on top of G-CSF can save $17,000 per patient, not including the cost of Motixafortide. These cost savings should leave substantial room in the future to optimize our pricing strategy for Motixafortide at product launch and thereafter, if approved.

"It is also important to note that fewer administrations and apheresis sessions confer meaningful safety and time benefits to patients. In addition, the significantly higher median number of cells collected in one apheresis session ~11 million using Motixafortide on top of G-CSF versus ~2 million for G-CSF alone not only enables transplantation of an optimal number of cells, with the potential to significantly save on time to engraftment, it also permits the retention of enough cells for cryopreservation in the event that an additional transplantation is required in the future. Lastly, higher levels of certainty regarding the number of apheresis sessions required for mobilization could enable more efficient utilization of apheresis units at transplantation institutions, where there is often a shortage of available machines.

"We believe the data from the GENESIS study, together with the results from this pharmacoeconomic study, set Motixafortide apart from all other mobilization agents either currently available or in development. If approved, Motixafortide represents a significant advancement in SCM to the benefit of patients and payers alike, and, to that end, we remain on track to submit a New Drug Application (NDA) to the FDA in the first half of next year," Mr. Serlin concluded.

About the Pharmacoeconomic Study

The pharmacoeconomic study analyzed healthcare resource utilization (HRU) observed during the Phase 3 GENESIS trial, which randomized 122 patients into two arms: Motixafortide plus G-CSF (n=80) or placebo plus G-CSF (n=42). HRU data points collected include: (1) the number of Motixafortide and G-CSF doses, as well as the number of apheresis sessions performed, in primary mobilization; (2) the percentage of patients needing rescue mobilization due to poor primary mobilization, including the number of apheresis sessions needed and the number of G-CSF and plerixafor doses required; and (3) hospitalization costs related to conditioning and transplantation, including length of stay. Quality-adjusted life years gained (QALY) from published literature were also incorporated into the model. Motixafortide plus G-CSF was associated with a statistically significant HRU decrease during the autologous stem cell transplantation process compared to standard-of-care G-CSF alone. Given the higher number of mobilized cells and lower number of apheresis sessions, lifetime estimates show quality-adjusted-life-year (QALY) benefits and net cost savings of ~$17,000 (not including the cost of Motixafortide), versus the current standard of care.

About the GENESIS Phase 3 Trial

The GENESIS Phase 3 trial (NCT03246529) was initiated in December 2017. GENESIS was a randomized, placebo-controlled, multicenter study, evaluating the safety, tolerability and efficacy of Motixafortide and G-CSF, compared to placebo and G-CSF, for the mobilization of hematopoietic stem cells for autologous transplantation in multiple myeloma patients. The primary objective of the study was to demonstrate that only one dose of Motixafortide on top of G-CSF is superior to G-CSF alone in the ability to mobilize 6 million CD34+ cells in up to two apheresis sessions. Additional objectives included time to engraftment of neutrophils and platelets and durability of engraftment, as well as other efficacy and safety parameters. The study successfully met all primary and secondary endpoints with an exceptionally high level of statistical significance (p<0.0001), including approximately 90% of patients who mobilized the target number of cells for transplantation with only one administration of Motixafortide and in only one apheresis session.

About Stem Cell Mobilization for Autologous Stem Cell Transplantation

Autologous stem cell transplantation (ASCT) is part of the standard treatment paradigm for a number of blood cancers, including multiple myeloma, non-Hodgkin's lymphoma and other lymphomas. In eligible patients, ASCT is performed after initial (induction) therapy, and, in most cases, requires consecutive-day clinic visits for the mobilization and apheresis (harvesting) phases, and full hospitalization for the conditioning chemotherapy and transplantation phases until engraftment. The associated burden is therefore significant patients experience clinically relevant deteriorations in their quality of life during ASCT, and healthcare resource use throughout the ASCT phases is particularly intense. Therefore, new interventions impacting the ASCT process have the potential for relieving some of the clinical burden for transplanted patients, the logistical burden for the apheresis units, and the financial burden for healthcare providers and payers.

Described simply, ASCT consists of: (1) mobilizing the patient's own stem cells from his/ her bone marrow to the peripheral blood for removing (harvesting) via an apheresis procedure; (2) freezing and storing the harvested cells until they are needed for transplantation; (3) providing a conditioning treatment, such as high-dose chemotherapy or radiation, to kill the remaining cancer cells the day before transplant; and (4) infusing the stored stem cells back to the patient intravenously via a catheter.

To mobilize the patient's stem cells from the bone marrow to the peripheral blood for harvesting, the current standard of care includes the administration of 5-8 daily doses of granulocyte colony stimulating factor (G-CSF), and the performance of 1-4 apheresis sessions. For patients unable to mobilize sufficient numbers of cells for harvesting during this primary mobilization phase, rescue therapy is carried out, consisting of 1-4 doses of plerixafor on top of G-CSF, and the performance of an additional number of apheresis sessions as necessary. In light of this, an agent with superior mobilization activity may significantly reduce the mobilization and harvesting burden and associated risks of the ASCT process and lead to significant clinical and resource benefits.

About BioLineRx

BioLineRx Ltd. (NASDAQ/TASE: BLRX) is a late clinical-stage biopharmaceutical company focused on oncology. The Company's business model is to in-license novel compounds, develop them through clinical stages, and then partner with pharmaceutical companies for further clinical development and/or commercialization.

The Company's lead program, Motixafortide (BL-8040), is a cancer therapy platform that was successfully evaluated in a Phase 3 study in stem cell mobilization for autologous bone-marrow transplantation, as well as reporting positive results from a pre-planned pharmacoeconomic study, and is currently in preparations for an NDA submission. Motixafortide was also successfully evaluated in a Phase 2a study for the treatment of pancreatic cancer in combination with KEYTRUDA and chemotherapy under a clinical trial collaboration agreement with MSD (BioLineRx owns all rights to Motixafortide), and is currently being studied in combination with LIBTAYO and chemotherapy as a first-line PDAC therapy.

BioLineRx is also developing a second oncology program, AGI-134, an immunotherapy treatment for multiple solid tumors that is currently being investigated in a Phase 1/2a study.

For additional information on BioLineRx, please visit the Company's website at http://www.biolinerx.com, where you can review the Company's SEC filings, press releases, announcements and events.

Various statements in this release concerning BioLineRx's future expectations constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include words such as "may," "expects," "anticipates," "believes," and "intends," and describe opinions about future events. These forward-looking statements involve known and unknown risks and uncertainties that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Factors that could cause BioLineRx's actual results to differ materially from those expressed or implied in such forward-looking statements include, but are not limited to: the initiation, timing, progress and results of BioLineRx's preclinical studies, clinical trials and other therapeutic candidate development efforts; BioLineRx's ability to advance its therapeutic candidates into clinical trials or to successfully complete its preclinical studies or clinical trials; BioLineRx's receipt of regulatory approvals for its therapeutic candidates, and the timing of other regulatory filings and approvals; the clinical development, commercialization and market acceptance of BioLineRx's therapeutic candidates; BioLineRx's ability to establish and maintain corporate collaborations; BioLineRx's ability to integrate new therapeutic candidates and new personnel; the interpretation of the properties and characteristics of BioLineRx's therapeutic candidates and of the results obtained with its therapeutic candidates in preclinical studies or clinical trials; the implementation of BioLineRx's business model and strategic plans for its business and therapeutic candidates; the scope of protection BioLineRx is able to establish and maintain for intellectual property rights covering its therapeutic candidates and its ability to operate its business without infringing the intellectual property rights of others; estimates of BioLineRx's expenses, future revenues, capital requirements and its needs for additional financing; risks related to changes in healthcare laws, rules and regulations in the United States or elsewhere; competitive companies, technologies and BioLineRx's industry; risks related to the COVID-19 pandemic; and statements as to the impact of the political and security situation in Israel on BioLineRx's business. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on February 23, 2021. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law.

Contact:Tim McCarthyLifeSci Advisors, LLC+1-212-915-2564[emailprotected]

or

Moran MeirLifeSci Advisors, LLC+972-54-476-4945[emailprotected]

SOURCE BioLineRx Ltd.

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BioLineRx Announces Positive Results from Pharmacoeconomic Study Positioning Motixafortide as Potential Standard of Care in Stem Cell Mobilization -...

Bone Marrow Stem Cells Comments Off on BioLineRx Announces Positive Results from Pharmacoeconomic Study Positioning Motixafortide as Potential Standard of Care in Stem Cell Mobilization -… | October 16th, 2021

NEW YORK, Oct. 14, 2021 /PRNewswire/ --BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, will present findings from a multicenter, open label clinical trial of NurOwn in progressive multiple sclerosis. The study, "Phase 2 Safety and Efficacy Study of Intrathecal MSC-NTF cells in Progressive Multiple Sclerosis," will be delivered in an oral presentation today at the fully digital37thCongress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS).

The Phase 2 clinical trial was designed to evaluate intrathecal administration of NurOwn (autologous MSC-NTF cells) in participants with progressive MS. The study achieved the primary endpoint of safety and tolerability. It demonstrated a reduction of neuroinflammatory biomarkers and an increase in neuroprotective biomarkers in the cerebrospinal fluid (CSF) and consistent improvement across MS functional outcome measures, including measures of walking, upper extremity function, vision and cognition.

"We were pleased that this study demonstrated safety, preliminary evidence of efficacy and relevant biomarker outcomes in patients with progressive multiple sclerosis, in an area of high unmet need," said Jeffrey Cohen, M.D., Director of Experimental Therapeutics at the Cleveland Clinic Mellen Center for MS and principal investigator for the trial. "These results should be confirmed in a randomized placebo-controlled trial."

The study was sponsored by Brainstorm Cell Therapeutics with additional financial support for biomarker analyses from the National Multiple Sclerosis Society Fast-Forward Program. It was conducted at four U.S. MS centers of excellence:

"We very much appreciate the tremendous collaboration among many premier organizations, for their generous sharing of expertise, support and data, which enabled the important balance between scientific rigor and ethical treatment of progressive MS participants in the trial," said Ralph Kern, M.D., MHSc., President and Chief Medical Officer, Brainstorm Cell Therapeutics. "We are holding discussions with key MS experts, and seeking guidance from the FDA to determine next steps for the development of NurOwn in progressive MS."

"The National MS Society is pleased to support the biomarker portion of this study through our commercial funding program Fast Forward," said Mark Allegretta, Ph.D., Vice President, Research. "We're encouraged to see evidence that the biomarker analysis showed proof of concept for detecting neuroprotection and reduced inflammation."

About the trial

The Phase 2 open-label studyevaluated the safety and efficacy of intrathecal administration of autologous MSC-NTF cells in patients with primary or secondary progressive MS. The primary study endpoint was safety and tolerability. Secondary efficacy endpoints included: timed 25-foot walk (T25FW); 9-Hole Peg Test (9-HPT); Low Contrast Letter Acuity (LCLA); Symbol Digit Modalities Test (SDMT); 12 item MS Walking Scale (MSWS-12); as well as cerebrospinal fluid (CSF) and blood biomarkers. Clinical efficacy outcomes were compared with matched (n=48) participants in the Comprehensive Longitudinal Investigation of Multiple Sclerosis (CLIMB) registry, Tanuja Chitnis, MD Brigham and Women's Hospital and the Ann Romney Center for Neurologic Diseases, and 255 patient randomized double blind placebo controlled NN-102 SPRINT-MS Study, courtesy NIH/NINDS, PI: Robert J. Fox, MD, MS, FAAN, Cleveland Clinic, CTR: NCT01982942. Baseline characteristics from these two cohorts were similar allowing for comparison of efficacy results, comparisons with SPRINT-MS were with the placebo arm of this study.

Mean age of participants was 47 years, 56% were female, and mean baseline EDSS score was 5.4. 18 participants were treated, 16 (80%) received all 3 treatments and completed the entire study; 2 study discontinuations were due to procedure-related adverse events. No deaths or treatment-related adverse events due to worsening of MS were observed.

In responder analyses, 14% and 13% of MSC-NTF treated participants showed at least a 25% improvement in T25FW and 9-HPT (combined hands) respectively, compared to 5% and 0% in matched CLIMB patients and 9% and 3% in SPRINT. Twenty-seven percent (27%) showed at least an 8-letter improvement in LCLA (binocular, 2.5% threshold) and 67% showed at least a 3-point improvement in SDMT, compared to 6% and 18% in CLIMB and 13% and 35% in SPRINT, respectively.

Mean improvements of +0.10 ft/sec in T25FW and -0.23 sec in 9-HPT (combined hands), were observed in MSC-NTF treated participants, compared to a mean worsening of -0.07 ft/sec and +0.49 sec in CLIMB and -0.06 ft/sec and +0.28 sec in SPRINT, respectively. MSC-NTF treated participants showed a mean improvement of +3.3 letters in LCLA (binocular, 2.5% threshold) and 3.8 points in SDMT, compared to a mean worsening of -1.07 letters in LCLA (binocular, 2.5% threshold) and mean improvement of +0.10 in SDMT, in CLIMB and -0.6 and -0.1 in SPRINT. In addition the MSFC-4 Composite Z-score of T25W, 9-HPT, SDMT and LCLA showed a 0.18 point improvement in MSC-NTF treated participants, while CLIMB and SPRINT showed decreases of -0.02 and -0.05.

Furthermore, 38% of treated patients showed at least a 10-point improvement in the MSWS-12 a patient reported outcome that evaluates the impact of MS on walking function, whereas this outcome was not evaluated in CLIMB or SPRINT.

CSF biomarkers obtained at 3 consecutive time points, showed increases in neuroprotective molecules (VEGF, HGF, NCAM-1,Follistatin, Fetuin-A) and decreases in neuroinflammatory biomarkers (MCP-1, SDF-1, sCD27 and Osteopontin).

About NurOwn

The NurOwntechnology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells are designed to effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwntechnology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug designation status from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has completed a Phase 3 pivotal trial in ALS (NCT03280056); this trial investigated the safety and efficacy of repeat-administration of autologous MSC-NTF cells and was supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). BrainStorm completed under an investigational new drug application a Phase 2 open-label multicenter trial (NCT03799718) of autologous MSC-NTF cells in progressive multiple sclerosis (MS) and was supported by a grant from the National MS Society (NMSS).

For more information, visit the company's website atwww.brainstorm-cell.com.

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future NurOwnmanufacturing and clinical development plans, constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may," "should," "would," "could," "will," "expect,""likely," "believe," "plan," "estimate," "predict," "potential," and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, the prospects for regulatory approval of BrainStorm's NurOwntreatment candidate, the initiation, completion, and success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm's NurOwntreatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture, or to use third parties to manufacture, and commercialize the NurOwntreatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

Contacts:

Investor Relations:Eric GoldsteinLifeSci Advisors, LLCPhone: +1 (646) 791-9729egoldstein@lifesciadvisors.com

Media:Mariesa Kemble kemblem@mac.com

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Phase 2 Clinical Trial Data of NurOwn in Progressive MS Will Be Presented at the 37th Congress of the European Committee for Treatment and Research in...

Bone Marrow Stem Cells Comments Off on Phase 2 Clinical Trial Data of NurOwn in Progressive MS Will Be Presented at the 37th Congress of the European Committee for Treatment and Research in… | October 16th, 2021

GRAND RAPIDS, Mich. As day turned to darkness, a crowd gathered at Monroe North on Saturday in Grand Rapids for the annual Light the Night celebration.

Its an inspirational community event, said Anne Bradley, Light the Night campaign development manager.

People eventually made their way to the street and showed support in each stride for blood cancer patients, their families and the countless others impacted by the disease.

Among the participants was Colette Smiley. Her white light gave a hint as to why she showed up.

I am a leukemia survivor, said Smiley. It completely alters your life.

In 2014, doctors diagnosed Smiley with Acute Myeloid Leukemia, or AML. Its an often fatal cancer that forms in a persons blood and bone marrow.

Smiley credits her treatment plan and a strangers stem cells for helping her enter remission within weeks of her diagnosis.

I come here to celebrate, to celebrate each re-birthday that I have, but to also thank LLS for [what] they do each and every day and each and every year for blood cancer patients, said Smiley.

According to the Leukemia and Lymphoma Society, blood cancers impact an estimated 1.5 million people in the United States, with a person diagnosed once every three minutes.

Leukemia is the most common cancer diagnosed in children and adolescents younger than 20 years old. It accounts for 25.1 percent of all cancer cases in the age group.

Symptoms of blood cancers often only appear in advanced stages, but can include ones similar to a severe cold or flu. Survival rates at one point hovered around 3 percent, but now, its up to 95 percent.

Its our goal and our mission to end blood cancer and to improve the quality of life for patients and their families, said Bradley.

All proceeds from Light the Night benefit LLS, which says 80 cents of every dollars goes directly to their mission.

Attendees, though, found a deeper value in the night.

We all live different stories, said Smiley. We all work in different places and our families are different and our backgrounds are different, but something brought us together that we certainly didnt wish for, blood cancer, but its brought us together and we can support each other now.

Light the Night hopes to collect $1 million in Michigan.

To donate, click here.

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'Light the Night' returns to Grand Rapids, raising awareness, donations for blood cancers - Fox17

Bone Marrow Stem Cells Comments Off on ‘Light the Night’ returns to Grand Rapids, raising awareness, donations for blood cancers – Fox17 | October 16th, 2021

A genetic syndrome that affects bones

Osteogenesis Imperfecta (OI) is a hereditary disorder occurring in 1:10,000 births and characterised by osteopenia (bone loss) and skeletal fragility (fractures). Secondary features include short stature, skeletal deformities, blue sclera and dentinogenesis imperfect. (1) There is a large clinical variability in OI, and severity ranges from mild to lethal, based on radiological characteristics. Genetically, OI is a collagen-related syndrome. Type I collagen is a heterotrimeric helical structure synthesized by bone-forming cells (osteoblasts), and it constitutes the most abundant protein of the skeletal organic matrix. (2) Synthesis of type I collagen is a complex process. (3) Collagen molecules are cross-linked into fibrils (which confer tensile strength to the bones). Those are then mineralised by hydroxy-apatites (which provides compressive strength) and assembled into fibres.

Dominant mutations in either the COL1A1 or the COLA1A2 genes are responsible for up to 90% of all OI cases. These mutations (more than 1,000 of which have been identified) lead to impairment of collagen structure and production, which in either quantitative or qualitative bone extracellular matrix (ECM) defects. Mutations affecting ECM structure have serious health consequences because the skeleton protects visceral organs and the central nervous system and provides structural support. Bones also store fat in the yellow bone marrow found within the medullary cavity, whilst the red marrow located at the end of long bones is the site of haematopoiesis. In addition, the ECM constitutes a reservoir of phosphate, calcium, and growth factors, and is involved in trapping dangerous molecules.

Stem cell therapy for OI aims to improve bone quality by harnessing the ability of mesenchymal stem cells (MSC) to differentiate into osteoblasts, with the rationale that donor cells would engraft into bones, produce normal collagen and function as a cell replacement. Stem cells have, therefore, been proposed for the treatment of OI (4) and, in particular, prenatal foetal stem cell therapy (foetal stem cells injected into a foetus, i.e. foetal-to-foetal) approach, which offers a promising route to effective treatment. (5) Human foetal stem cells are more primitive than stem cells isolated from adult tissues and present advantageous characteristics compared to their adult counterparts, i.e. they possess a higher level of plasticity, differentiate more readily into specific lineages, grow faster, senesce later, express higher levels of adhesion molecules, and are smaller in size. (6,7) Prenatal cell therapy capitalises on the small size of the foetus and its immunological naivete. In addition, stem cells delivered in utero benefit from the expansion of endogenous stem cells and may prevent organ injury before irreversible damage. (8)

However, human foetal stem cells used are isolated from either foetal blood drawn by cardiac puncture, either during termination of pregnancy or during ongoing pregnancy, albeit using an invasive procedure associated with a high risk of morbidity and mortality for both the foetus and the mother (9). Foetal cells can also be isolated from the first-trimester liver (following termination of pregnancy) and such cells are currently used in The Boost Brittle Bones Before Birth (BOOSTB4) clinical trial, which aims to investigate the safety and efficacy of transplanting foetal derived MSCs prenatally and/or in early postnatal life to treat severe Osteogenesis Imperfecta (OI) (10). Alternatively, foetal stem cells can be isolated during ongoing pregnancy from the amniotic fluid, either during mid-trimester amniocentesis or at birth (11,12) or from the chorionic villi of the placenta during first-trimester chorionic villi sampling (13).

We have demonstrated that human fetal stem cells isolated from first trimester blood possess superior osteogenic differentiation potential compared to adult stem cells isolated from bone marrow and to fetal stem cells isolated from first trimester liver. We showed that in utero transplantation of these cells in an experimental model of severe OI resulted in a drastic 75% decrease in fracture rate incidence and skeletal brittleness, and improvement of bone strength and quality.(14) A similar outcome was obtained using placenta-derived foetal stem cells (15) and amniotic fluid stem cells following perinatal transplantation into experimental models. (16,17)

Understanding the mechanisms of action of donor cells will enable the engineering of donor cells with superior efficacy to stimulate bone formation and strengthen the skeleton. Despite their potential to differentiate down the osteogenic lineage, there is little evidence that donor cells contribute to regenerating bones through direct differentiation, due to the very low level of donor cell engraftment reported in all our studies. When placed in an osteogenic microenvironment in vitro, foetal stem cells readily differentiate into osteoblasts and produce wild type collagen molecules. However, there are insufficient proofs that collagen molecules of donor cell origin contribute to the formation of the host bone ECM to confer superior resistance to fracture.

It is now well accepted that stem cells can influence the behaviour of target cells through the release of paracrine factors and, therefore, contribute to tissue regeneration indirectly. We have indeed recently shown that donor stem cells stimulate the differentiation of resident osteoblasts, which were unable to fully mature in the absence of stem cell treatment. (16,17) We are now focusing our efforts on understanding the precise molecular mechanisms by which donor cells improve skeletal health to counteract bone fragility caused by various OI-causative mutations.

References

Please note: This is a commercial profile

2019. This work is licensed under aCC BY 4.0 license.

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Stem cell & gene therapy to treat osteogenesis imperfecta: hype or hope - Open Access Government

Bone Marrow Stem Cells Comments Off on Stem cell & gene therapy to treat osteogenesis imperfecta: hype or hope – Open Access Government | October 5th, 2021

SYDNEY, Oct. 5, 2021 /PRNewswire/ -- Clinical stage drug development company Pharmaxis Ltd (ASX: PXS) today announced further positive results of data analysis from a phase 1c clinical trial (MF-101) studying its drug PXS-5505 in patients with the bone marrow cancer myelofibrosis for 28 days at three dosage levels.

Assessment with Pharmaxis' proprietary assays of the highest dose has shown inhibition of the target enzymes, LOX and LOXL2, at greater than 90% over a 24-hour period at day 7 and day 28. The trial safety committee has reviewed the results and having identified no safety signals, has cleared the study to progress to the phase 2 dose expansion phase where 24 patients will be treated at the highest dose twice a day for 6 months.

Pharmaxis CEO Gary Phillips said, "We are very pleased to have completed the dose escalation phase of this study with such clear and positive findings.We will now immediately progress to the phase 2 dose expansion study where we aim to show PXS-5505 is safe to be taken longer term with the disease modifying effects that we have seen in the pre-clinical models. The trial infrastructure and funding is in place and we are on track to complete the study by the end of 2022."

Independent, peer-reviewed research has demonstrated the upregulation of several lysyl oxidase family members in myelofibrosis.The level of inhibition of LOX achieved in the current study at all three doses significantly exceeds levels that caused disease modifying effects with PXS-5505 in pre-clinical models of myelofibrosis with improvements in blood cell count, diminished spleen size and reduced bone marrow fibrosis. LOXL2 was inhibited to a similar degree and based on pre-clinical work such high inhibition is likely replicated for other LOX family members (LOXL1, 3 and 4).[1] Study data can be viewed in the full announcement.

Commenting on the results of the trial, Dr Gabriela Hobbs, Assistant Professor, Medicine, Harvard Medical School & Clinical Director, Leukaemia, Massachusetts General Hospital said, "Despite improvements in the treatment of myelofibrosis, the only curative therapy remains an allogeneic stem cell transplantation, a therapy that many patients are not eligible for due to its morbidity and mortality. None of the drugs approved to date consistently or meaningfully alter the fibrosis that defines this disease. PXS-5505 has a novel mechanism of action by fully inhibiting all LOX enzymes. An attractive aspect of this drug is that so far in healthy controls and in this phase 1c study in myelofibrosis patients, the drug appears to be very well tolerated. This is meaningful as approved drugs and those that are undergoing study, are associated with abnormal low blood cell counts. Preliminary data thus far, demonstrate that PXS-5505 leads to a dramatic, >90% inhibition of LOX and LOXL2 at one week and 28 days. This confirms what's been shown in healthy controls as well as mouse models, that this drug can inhibit the LOX enzymes in patients. Inhibiting these enzymes is a novel approach to the treatment of myelofibrosis by preventing the deposition of fibrosis and ultimately reversing the fibrosis that characterizes this disease."

The phase 1c/2a trial MF-101 cleared by the FDA under the Investigational New Drug (IND) scheme aims to demonstrate that PXS-5505, the lead asset in Pharmaxis' drug discovery pipeline, is safe and effective as a monotherapy in myelofibrosis patients who are intolerant, unresponsive or ineligible for treatment with approved JAK inhibitor drugs. Trial sites will now open to recruit myelofibrosis patients into the 6-month phase 2 study in Australia, South Korea, Taiwan and the USA.

An effective pan-LOX inhibitor for myelofibrosis would open a market that is conservatively estimated at US$1 billion per annum.

While Pharmaxis' primary focus is the development of PXS-5505 for myelofibrosis, the drug also has potential in several other cancers including liver and pancreatic cancer where it aims to breakdown the fibrotic tissue in the tumour and enhance the effect of chemotherapy treatment.

Trial Design

Name of trial

PXS5505-MF-101: A phase 1/2a study to evaluate safety, pharmacokinetic and pharmacodynamic dose escalation and expansion study of PXS-5505 in patients with primary, post-polycythaemia vera or post-essential thrombocythemia myelofibrosis

Trial number

NCT04676529

Primary endpoint

To determine the safety of PXS-5505 in patients with myelofibrosis

Secondary endpoints

Blinding status

Open label

Placebo controlled

No

Trial design

Randomised, multicentre, 4 week duration phase 1 (dose escalation) followed by 6 month phase 2 (dose expansion)

Treatment route

Oral

Treatment frequency

Twice daily

Dose level

Dose escalation: three escalating doses

Dose expansion: one dose

Number of subjects

Dose escalation: minimum of three patients to maximum of 18 patients

Dose expansion: 24 patients

Subject selection criteria

Patients with primary or secondary myelofibrosis who are intolerant, unresponsive or ineligible for treatment with approved JAK inhibitor drugs

Trial locations

Dose escalation: Australia (2 sites) and South Korea (4 sites)

Dose expansion: Australia, Korea, Taiwan, USA

Commercial partners involved

No commercial partner

Reference: (1) doi.org/10.1002/ajh.23409

AUTHORISED FOR RELEASE TO ASX BY:

Pharmaxis Ltd Disclosure Committee. Contact: David McGarvey, Chief Financial Officer and Company Secretary: T +61 2 9454 7203, E [emailprotected]

Join the Pharmaxis mailing listhere

Follow us on LinkedInand Twitter

About Pharmaxis

Pharmaxis Ltd is an Australian clinical stage drug development company developing drugs for inflammatory and fibrotic diseases, with a focus on myelofibrosis. The company has a highly productive drug discovery engine built on its expertise in the chemistry of amine oxidase inhibitors, with drug candidates in clinical trials. Pharmaxis has also developed two respiratory products which are approved and supplied in global markets, generating ongoing revenue.

Pharmaxis is developing its drug PXS-5505 for the bone marrow cancer myelofibrosis which causes a build up of scar tissue that leads to loss of production of red and white blood cells and platelets. The US Food and Drug Administration has granted Orphan Drug Designation to PXS-5055 for the treatment of myelofibrosis and permission under an Investigational Drug Application (IND) to progress a phase 1c/2 clinical trial that began recruitment in Q1 2021. PXS5505 is also being investigated as a potential treatment for other cancers such as liver and pancreatic cancer.

Other drug candidates being developed from Pharmaxis' amine oxidase chemistry platform are targeting fibrotic diseases such as kidney fibrosis, NASH, pulmonary fibrosis and cardiac fibrosis; fibrotic scarring from burns and other trauma; and inflammatory diseases such as Duchenne Muscular Dystrophy.

Pharmaxis has developed two products from its proprietary spray drying technology that are manufactured and exported from its Sydney facility; Bronchitol for cystic fibrosis, which is approved and marketed in the United States, Europe, Russia and Australia; and Aridol for the assessment of asthma, which is approved and marketed in the United States, Europe, Australia and Asia.

Pharmaxis is listed on the Australian Securities Exchange (PXS). Its head office, manufacturing and research facilities are in Sydney, Australia. http://www.pharmaxis.com.au

About PXS-5505

PXS-5505 is an orally taken drug that inhibits the lysyl oxidase family of enzymes, two members LOX and LOXL2 are strongly upregulated in human myelofibrosis. In pre-clinical models of myelofibrosis PXS-5505 reversed the bone marrow fibrosis that drives morbidity and mortality in myelofibrosis and reduced many of the abnormalities associated with this disease. It has already received IND approval and Orphan Drug Designation from the FDA.

About Myelofibrosis

Myelofibrosis is a disorder in which normal bone marrow tissue is gradually replaced with a fibrous scar-like material. Over time, this leads to progressive bone marrow failure. Under normal conditions, the bone marrow provides a fine network of fibres on which the stem cells can divide and grow. Specialised cells in the bone marrow known as fibroblasts make these fibres.

In myelofibrosis, chemicals released by high numbers of platelets and abnormal megakaryocytes (platelet forming cells) over-stimulate the fibroblasts. This results in the overgrowth of thick coarse fibres in the bone marrow, which gradually replace normal bone marrow tissue. Over time this destroys the normal bone marrow environment, preventing the production of adequate numbers of red cells, white cells and platelets. This results in anaemia, low platelet counts and the production of blood cells in areas outside the bone marrow for example in the spleen and liver, which become enlarged as a result.

Myelofibrosis can occur at any age but is usually diagnosed later in life, between the ages of 60 and 70 years. The cause of myelofibrosis remains largely unknown. It can be classified as either JAK2 mutation positive (having the JAK2 mutation) or negative (not having the JAK2 mutation).

Source: Australian Leukemia Foundation: https://www.leukaemia.org.au/disease-information/myeloproliferative-disorders/types-of-mpn/primary-myelofibrosis/

Forward-looking statements

Forwardlooking statements in this media release include statements regarding our expectations, beliefs, hopes, goals, intentions, initiatives or strategies, including statements regarding the potential of products and drug candidates. All forward-looking statements included in this media release are based upon information available to us as of the date hereof. Actual results, performance or achievements could be significantly different from those expressed in, or implied by, these forward-looking statements. These forward-looking statements are not guarantees or predictions of future results, levels of performance, and involve known and unknown risks, uncertainties and other factors, many of which are beyond our control, and which may cause actual results to differ materially from those expressed in the statements contained in this document. For example, despite our efforts there is no certainty that we will be successful in developing or partnering any of the products in our pipeline on commercially acceptable terms, in a timely fashion or at all. Except as required by law we undertake no obligation to update these forward-looking statements as a result of new information, future events or otherwise.

CONTACT:

Media: Felicity Moffatt: T +61 418 677 701, E [emailprotected]

Investor relations:Rudi Michelson (Monsoon Communications) T +61 411 402 737, E [emailprotected]

SOURCE Pharmaxis Limited

http://www.pharmaxis.com.au

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Pharmaxis Cleared To Progress To Phase 2 Bone Marrow Cancer Trial - PRNewswire

Bone Marrow Stem Cells Comments Off on Pharmaxis Cleared To Progress To Phase 2 Bone Marrow Cancer Trial – PRNewswire | October 5th, 2021

Myles Glass has spent the past several years living life on the sidelines in a wheelchair, wishing for a better day. That day came in November 2020.

INDIANAPOLIS A 13-year-old boy living with sickle cell disease has been given a second chance at life, thanks to his mother.

Myles Glass has been through more in his young life than most adults. For the past few years, Glass has spent his days in and out of Riley Hospital for Children.

"[I] kind of have to look on the bright side of things. Being in the hospital, I meet new nurses and kids who go through what I go through. It's kind of hard to go through that at my age," Glass said.

He was diagnosed with sickle cell disease as a newborn. According to the Centers for Disease Control and Prevention, African Americans make up the largest number of people with the disease in the U.S.

Sickle cell disease is an inherited condition that impacts red blood cells and causes pain, infections and extreme fatigue. These symptoms keep Glass from doing things he loves.

"For him, it's kind of like we have to have him in a bubble," said his mother, Melissa Sanders.

Glass has spent the past several years living life on the sidelines in a wheelchair, wishing for a better day.

"[I would] hope that one day, I can do what kids do, like playing football and basketball," Glass said.

That day came in November 2020 when his mother donated bone marrow for a stem cell transplant, curing him of sickle cell disease.

"I was able to give him a second life with being a donor so that he can somewhat be a normal kid," Sanders said.

Riley Hospital for Children Dr. Seethal Jacob, who has been working with Glass and his family, said one baby every two minutes is born with sickle cell disease. She also said studies show there is a clear disparity for funding for this disease.

"There's been a lot of neglect when it comes to the disease itself. I think it's important to pay attention to the population it affects. I think that likely tells the story why sickle cell disease has been a neglected disease for so long," Jacob said.

Despite his challenges, Glass is staying positive and making strides in his physical therapy at Riley Hospital for Children.

"He's already been through harder things than most people will ever go through. I think anything else in life is going to be a piece of cake," said his physical therapist, Sarah Johnson.

"This gives me a glimpse of hope that even though you may have been diagnosed with this disease, it's not the end of the world," Sanders said.

For Glass, this is just the beginning. He hopes his story encourages other people living with sickle cell disease to keep moving forward.

"I know it's hard now, but you'll get through it. You'll be able to do what kids do your own age," Glass said.

Click here for more information on sickle cell disease and treatment options.

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Indianapolis mother gives 13-year-old son with sickle cell disease a 2nd chance at life - WTHR

Bone Marrow Stem Cells Comments Off on Indianapolis mother gives 13-year-old son with sickle cell disease a 2nd chance at life – WTHR | October 5th, 2021

TUCSON, Ariz., Oct. 5, 2021 /PRNewswire/ --On November 15th, 2021, healthcare professionals and the general public are invited to participate in World Cord Blood Day 2021 (www.WorldCordBloodDay.org) via a free online conference and live educational events being held around the globe. Registration is now open (free, public welcome).

Cord blood is the blood left in the umbilical cord and placenta following the birth of a child. It is rich in life-saving stem cells. While cord blood has been used for over 30 years, Covid-19 has renewed interest in this medical resource given its unique regenerative qualities and the fact that most cord blood currently stored was collected prior to the pandemic. These units are naturally Covid-free, an advantage over many other stem cell sources. Yet, cord blood is still thrown away as medical waste in the majority of births worldwide. Education is key to changing this practice and World Cord Blood Day 2021 will provide the perfect opportunity for OBGYNs, midwives, transplant doctors, nurses, parents and students to learn about this vital medical resource.

During World Cord Blood Day 2021, participants will learn how cord blood is used to treat over 80 life-threatening diseases such as leukemia and lymphoma, bone marrow failure, immune deficiency diseases and inherited blood disorders such as thalassemia and sickle cell disease. Leading transplant doctors and researchers will also highlight cord blood's role in the emerging fields of gene therapy and regenerative medicine to potentially treat cerebral palsy, autism, stroke and more.

Organized by Save the Cord Foundation, a 501c3 non-profit, World Cord Blood Day 2021 is officially sponsored by QuickSTAT Global Life Science Logistics, recognized leader in medical shipping and healthcare logistics. Inspiring Partners include Be the Match (NMDP), World Marrow Donor Association (WMDA-Netcord), AABB Center for Cellular Therapies, Cord Blood Association, and the Foundation for the Accreditation of Cellular Therapy (FACT).

"QuickSTAT, part of Kuehne+Nagel, is proud to sponsor the 5th annual World Cord Blood Day to help support and educate the healthcare community and expectant parents about the life-saving value of cord blood stem cells. We're excited to play a role in the research and development of cord blood derivative therapies by providing logistics supply chain solutions to cord blood, biotech and pharmaceutical companies worldwide," said Monroe Burgess, VP Life Science Commercial Marketing, QuickSTAT.

Visit http://www.WorldCordBloodDay.org to learn how you can participate. Show your support on social media: @CordBloodDay, #WorldCordBloodDay, #WCBD21

About Save the Cord FoundationSave the Cord Foundation (a 501c3 non-profit) was established to advance cord blood education providing non-commercial information to health professionals and the public regarding methods for saving cord blood, as well as current applications and the latest research. http://www.SaveTheCordFoundation.org.

About QuickSTAT Global Life Science LogisticsEvery day, QuickSTAT, a part of Kuehne+Nagel, safely and reliably moves thousands of critical shipments around the world. For over forty years, QuickSTAT has been entrusted with transporting human organs and tissue for transplant or research, blood, blood products, cord blood, bone marrow, medical devices, and personalized medicine, 24/7/365. QuickSTAT's specially trained experts work with hospitals, laboratories, blood banks and medical processing centers, and utilize the safest routes to ensure integrity, temperature control and chain of custody throughout the transportation process. Learn more at http://www.quickstat.aero.

Contact:Charis Ober(520) 419-0269[emailprotected]

SOURCE Save the Cord Foundation

http://www.SaveTheCordFoundation.org

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Ready to Treat Over 80 Life-Threatening Diseases, Discover the Potential of Cord Blood during World Cord Blood Day 2021 - PRNewswire

Bone Marrow Stem Cells Comments Off on Ready to Treat Over 80 Life-Threatening Diseases, Discover the Potential of Cord Blood during World Cord Blood Day 2021 – PRNewswire | October 5th, 2021

A student has completed the London Marathon alongside the stem cell donor who saved her life.

icky Lawrence, 21, from Moseley, Birmingham, was diagnosed with severe aplastic anaemia in 2008, when she was eight years old, a condition in which the bone marrow does not produce an adequate number of new blood cells.

Thanks to Elliott Brock, a physiotherapist from Mersea Island, Essex, Ms Lawrence received a transplant that same year.

Ms Lawrence sent Mr Brock a letter in 2015 and the pair met for the first time.

Fast forward to 2021 and they have just completed the London Marathon in support of Anthony Nolan.

Ms Lawrence, who is in her fourth year of a medical degree at Newcastle University, told the PA news agency that completing the marathon was absolutely amazing.

She said: Crossing the finish line was so emotional, not just because wed run 26 miles, but running 26 miles alongside the man who saved your life is a pretty big feat.

Ms Lawrence added: A big slogan of Anthony Nolan is without your support, there is no cure.

Without Elliott donating his stem cells to a stranger, I would not be here. I wouldnt have made it to Christmas. I would never have had the opportunities Ive had to go to university, to study abroad, to play hockey.

Him donating his stem cells gave me a second life and there are still so many people that need a transplant that are not finding the matches they need, especially among the ethnic minority community.

Unfortunately if you are of ethnic minority background, you only have a 37% chance of finding a match.

Mr Brock, 42, who wore a mask and cape during the race, said: That was a tongue of check nod [to the fact that the] easiest way to be called a hero is to donate your bone marrow.

I cannot emphasise to people enough that it is pain-free.

He added: It was just a day of celebration for London to celebrate having their marathon back.

The crowds were amazing and obviously to be side-by-side with the girl whose life, through the amazing work of Anthony Nolan, I managed to save sort of 13 years ago was just surreal really.

Its a lovely story of how my simple act made such a massive difference and we are able to celebrate it so many years after.

Anthony Nolan chief executive Henny Braund said: We are so grateful to Vicky and Elliott for running to raise funds and awareness of Anthony Nolan and the lifesaving work that we do.

Every day five Vickys, patients with blood cancer or a blood disorder, start their search for an Elliott.

If youre aged 16-30 and in good health, please consider joining the Anthony Nolan stem cell register. You could potentially save a life.

More information on how to join the stem cell register can be found at: http://www.anthonynolan.org/help-save-a-life/join-stem-cell-register

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Student completes London Marathon with the man who saved her life - Independent.ie

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Published: Oct. 4, 2021 at 6:00 AM EDT

NEW YORK, Oct. 4, 2021 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, announced today that Stacy Lindborg, Ph.D., Executive Vice President and Head of Global Clinical Research, will deliver a presentation at the2021 Cell & Gene Meeting on the Mesa, being held as a hybrid conferenceOctober 12-14, and October 19-20, 2021.

Dr. Lindborg's presentation highlights the expansion of Brainstorm's technology portfolio to include autologous and allogeneic product candidates, covering multiple neurological diseases. The most progressed clinical development program, which includes a completed phase 3 trial of NurOwn in ALS patients, remains the highest priority for Brainstorm. Brainstorm is committed to pursuing the best and most expeditious path forward to enable patients to access NurOwn.

Dr. Lindborg's presentation will be in the form of an on-demand webinar that will be available beginning October 12. Those who wish to listen to the presentation are required to registerhere. At the conclusion of the 2021 Cell & Gene Meeting on the Mesa, a copy of the presentation will also be available in the "Investors and Media" section of the BrainStorm website underEvents and Presentations.

About the 2021 Cell & Gene Meeting on the Mesa

The meeting will feature sessions and workshops covering a mix of commercialization topics related to the cell and gene therapy sector including the latest updates on market access and reimbursement schemes, international regulation harmonization, manufacturing and CMC challenges, investment opportunities for the sector, among others. There will be over 135 presentations by leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering and broader regenerative medicine technologies.

The conference will be delivered in a hybrid format to allow for an in-person experience as well as a virtual participation option. The in-person conference will take place October 12-14 in Carlsbad, CA. Virtual registrants will have access to all content via livestream during program dates. Additionally, all content will be available on-demand within 24 hours of the live program time. Virtual partnering meetings will take place October 19-20 via Zoom.

About NurOwn

The NurOwntechnology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells are designed to effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwntechnology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug designation status from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has completed a Phase 3 pivotal trial in ALS (NCT03280056); this trial investigated the safety and efficacy of repeat-administration of autologous MSC-NTF cells and was supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). BrainStorm completed under an investigational new drug application a Phase 2 open-label multicenter trial (NCT03799718) of autologous MSC-NTF cells in progressive multiple sclerosis (MS) and was supported by a grant from the National MS Society (NMSS).

For more information, visit the company's website atwww.brainstorm-cell.com.

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future NurOwnmanufacturing and clinical development plans, constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may," "should," "would," "could," "will," "expect,""likely," "believe," "plan," "estimate," "predict," "potential," and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, the prospects for regulatory approval of BrainStorm's NurOwntreatment candidate, the initiation, completion, and success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm's NurOwntreatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture, or to use third parties to manufacture, and commercialize the NurOwntreatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

ContactsInvestor Relations:Eric GoldsteinLifeSci Advisors, LLCPhone: +1 646.791.9729egoldstein@lifesciadvisors.com

Media:Paul TyahlaSmithSolvePhone: + 1.973.713.3768Paul.tyahla@smithsolve.com

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The above press release was provided courtesy of PRNewswire. The views, opinions and statements in the press release are not endorsed by Gray Media Group nor do they necessarily state or reflect those of Gray Media Group, Inc.

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BrainStorm to Present at the 2021 Cell & Gene Meeting on the Mesa - WWNY

Bone Marrow Stem Cells Comments Off on BrainStorm to Present at the 2021 Cell & Gene Meeting on the Mesa – WWNY | October 5th, 2021

StemExpress to use utilize the Thermo Fisher Accula rapid PCR testing system to provide event attendees with accurate results in 30 minutes.

Published: Oct. 5, 2021 at 2:33 PM CDT|Updated: 4 hours ago

SACRAMENTO, Calif., Oct. 5, 2021 /PRNewswire/ --StemExpress is proud to announce that they will be the official COVID-19 testing provider for 2021's Meeting on the Mesa, a hybrid event bringing together great minds in the cell and gene biotech sphere. It has partnered with Alliance for Regenerative Medicine to comply with the newly implemented California state COVID-19 vaccination and testing policy regarding gatherings with 1,000 or more attendees. This partnership will allow the vital in-person networking aspect of the event to commence while protecting the health and safety of participants and attendees.

In-person networking commences at the 2021 Cell and Gene Meeting on the Mesa with COVID-19 testing options provided by StemExpress.

As a leading global provider of human biospecimen products, StemExpress understands the incredible impact that Meeting on the Mesa has on the industry and has been a proud participant for many years. For over a decade, StemExpress has provided the cell and gene industry with vital research products and holds valued partnerships with many of this year's participants. As such, it understands the immense value that in-person networking provides and is excited to help bring this element back to the meeting safely and responsibly.

StemExpress has been a trusted provider of widescale COVID-19 testing solutions since early 2020 - providing testing for government agencies, public health departments, private sector organizations, and the public nationwide. For Meeting on the Mesa, StemExpress is offering convenient testing options for unvaccinated attendees and those traveling from outside of the country. Options will include take-home RT-PCR COVID Self-Testing Kits and on-site, rapid PCR testing for the duration of the event. The self-testing kit option allows attendees to test for COVID in the days leading up to the event for a seamless admission and the days following the event to confirm they haven't been exposed. The on-site rapid testing option utilizes the new Thermo Fisher Accula, offering in-person testing at the event with results in around 30 minutes. StemExpress is excited to bring these state-of-the-art COVID testing solutions to the frontlines of the Cell & Gene industry to allow for safe in-person connections.

The StemExpress partnership with Alliance for Regenerative Medicine seeks to empower the entire cell and gene industry with a long-awaited opportunity to return to traditional networking practices. It is well known that innovation doesn't exist in a vacuum - allowing great minds to come together is a sure way to spur scientific growth and advance cutting-edge research, giving hope for future cures.

Cell and Gene Meeting on the Mesa will take place October 12th, 2021, through October 14th, 2021, at Park Hyatt Aviara,7100 Aviara Resort Drive Carlsbad, CA 92011. To learn more about the event, please visit MeetingOnTheMesa.com.

For more information about COVID testing solutions for businesses and events, visit https://www.stemexpress.com/covid-19-testing/.

About StemExpress:

Founded in 2010 and headquartered in Sacramento, California, StemExpress is a leading global biospecimen provider of human primary cells, stem cells, bone marrow, cord blood, peripheral blood, and disease-state products. Its products are used for research and development, clinical trials, and commercial production of cell and gene therapies by academic, biotech, diagnostic, pharmaceutical, and contract research organizations (CRO's).

StemExpress has over a dozen global distribution partners and seven (7) brick-and-mortar cellular clinics in the United States, outfitted with GMP certified laboratories. StemExpress runs its own non-profit supporting STEM initiatives, college and high school internships, and women-led organizations. It is registered with the U.S. Food and Drug Administration (FDA) and is continuously expanding its network of healthcare partnerships, which currently includes over 50 hospitals in Europe and 3 US healthcare systems - encompassing 31 hospitals, 35 outpatient facilities, and over 200 individual practices and clinics.

StemExpress has been ranked by Inc. 500 as one of the fastest-growing companies in the U.S.

About the Alliance for Regenerative Medicine:

The Alliance for Regenerative Medicine (ARM) is the leading international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies. ARM promotes legislative, regulatory, reimbursement and manufacturing initiatives to advance this innovative and transformative sector, which includes cell therapies, gene therapies and tissue-based therapies. Early products to market have demonstrated profound, durable and potentially curative benefits that are already helping thousands of patients worldwide, many of whom have no other viable treatment options. Hundreds of additional product candidates contribute to a robust pipeline of potentially life-changing regenerative medicines and advanced therapies. In its 12-year history, ARM has become the voice of the sector, representing the interests of 400+ members worldwide, including small and large companies, academic research institutions, major medical centers and patient groups. To learn more about ARM or to become a member, visit http://www.alliancerm.org.

Media Contact: Anthony Tucker, atucker@stemexpress.com

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The above press release was provided courtesy of PRNewswire. The views, opinions and statements in the press release are not endorsed by Gray Media Group nor do they necessarily state or reflect those of Gray Media Group, Inc.

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StemExpress Partners with the Alliance for Regenerative Medicine to Provide COVID-19 Testing for the Cell and Gene Meeting on the Mesa - WSAW

Bone Marrow Stem Cells Comments Off on StemExpress Partners with the Alliance for Regenerative Medicine to Provide COVID-19 Testing for the Cell and Gene Meeting on the Mesa – WSAW | October 5th, 2021

Bone marrow transplantation, also referred to as hematopoieticular somatic cell transplantation, may be a sort of major surgery . It involves the transplantation of multidimensional, immature, and constantly dividing stem-cells from bone marrow, duct , or other sources. It are often autologous, polyglobulogenic or maybe syngenetic. This treatment are often wont to treat a good range of great diseases, like MS , red blood cell disease, paralysis agitans , disease , bone marrow cancer, leukemia, bone infection, myeloma , age related degeneration and more.

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Market Dynamics

High prevalence of cancer is predicted to propel growth of the worldwide bone marrow transplant market. as an example , consistent with Leukemia and Lymphoma Society, 176,200 people within the US are expected to be diagnosed with leukemia, lymphoma or myeloma in 2019. Moreover, increasing adoption of bone marrow transplant is additionally expected to assist in growth of the market. as an example , in August 2020, CytoDyn Inc., a late-stage biotechnology company, announced its efforts to duplicate Berlin and London patients HIV cure by using leronlimab during bone marrow transplant for five HIV patients who even have cancer.

Availability of effective therapies for the treatment of acute graft versus host disease is predicted to supply lucrative growth opportunities for players within the global bone marrow transplant market. as an example , in September 2020, Avalon GloboCare Corp., a clinical-stage developer of cell-based technologies and therapeutics, launched its new allogeneic mesenchymal stromal cell therapeutic platform a possible therapy for COVID-19 and for bone marrow transplant related complications of acute graft versus host disease.

However, bone marrow transplant may cause various complications like acute graft versus host disease, which is predicted to hinder growth of the worldwide bone marrow transplant market.

Among regions, the center East is predicted to witness significant growth within the global bone marrow transplant market, due to increasing adoption of bone marrow transplant within the region. as an example , in July 2020, Abu Dhabi Stem Cells Centre (ADSCC) and Sheikh Khalifa Medical City announced the primary ever successful bone marrow transplant administered within the UAE.

Competitive Analysis

Major players operating within the global bone marrow transplant market include, Lonza Group Ltd., Merck Millipore Corporation, Sanofi-Aventis LLC, AllCells LLC, STEMCELL Technologies, and American Type Culture Collection (ATCC) Inc.

Key players within the global bone marrow transplant market are focused on adopting collaboration and partnership strategies to expand their product portfolio. as an example , in November 2019, Lonza Group Ltd. partnered with Cryoport, Inc. within the cell and gene therapy field and across Lonzas vein-to-vein delivery network.

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Bone Marrow Transplant Market: North America to Dominate Market through 2027; Europe to Witness Steady Growth Over 2021-2027 UNLV The Rebel Yell -...

Bone Marrow Stem Cells Comments Off on Bone Marrow Transplant Market: North America to Dominate Market through 2027; Europe to Witness Steady Growth Over 2021-2027 UNLV The Rebel Yell -… | September 1st, 2021

DBMR has added a new report titled Bone Marrow-Derived Stem Cells (BMSCS) Market with analysis provides the insights which bring marketplace clearly into the focus and thus help organizations make better decisions. With a devotion and commitment of supreme level of resilience and integrated approaches, Bone Marrow-Derived Stem Cells (BMSCS) Market research report has been structured. The report also puts a light on growth opportunity assessment (GOA), customer insights (CI), competitive business intelligence (CBI), and distribution channel assessment (DCA). This world class market report analyses and evaluates the important industry trends, market size, market share estimates, and sales volume with which industry can speculate the strategies to increase return on investment (ROI). The statistics have been represented in the graphical format for an unambiguous understanding of facts and figures.

An influential Bone Marrow-Derived Stem Cells (BMSCS) Market report brings into focus plentiful of factors such as the general market conditions, trends, inclinations, key players, opportunities, and geographical analysis which all aids to take business towards the growth and success. This report provides the broader perspective of the market place with its comprehensive market insights and analysis which eases surviving and succeeding in the market. Moreover, this market report explains better market perspective in terms of product trends, marketing strategy, future products, new geographical markets, future events, sales strategies, customer actions or behaviors. Hence, the credible report brings into the focus, the more important aspects of the market or industry.

Bone marrow-derivedstem cells(BMSCS) market is expected to gain market growth in the forecast period of 2020 to 2027. Data Bridge Market Research analyses the market to growing at a CAGR of 10.4% in the above-mentioned forecast period. Increasing awareness regarding the benefits associates with the preservation of bone marrow derived stem cells will boost the growth of the market.

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The major players covered in the bone marrow-derived stem cells (BMSCS) market report are CBR Systems, Inc, Cordlife Sciences India Pvt. Ltd., Cryo-Cell International, Inc.ESPERITE N.V., LifeCell International Pvt. Ltd., StemCyte India Therapeutics Pvt. Ltd, PerkinElmer Inc, Global Cord Blood Corporation., Smart Cells International Ltd., Vita 34 among other domestic and global players. Market share data is available for Global, North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and South America separately. DBMR analysts understand competitive strengths and provide competitive analysis for each competitor separately.

Some of the factors such as introduction of novel technologies for the preservation of stem cells and their storage, surging investment that will help in research activities leading to stem cells benefits, adoption of hemotopoietic stem cell transplantation system will accelerate the growth of the bone marrow-derived stem cells (BMSCS) market in the forecast period of 2020-2027. Various factors that will create opportunities in the bone marrow-derived stem cells (BMSCS) market are increasing occurrences of various diseases along with rising applications in emerging economies.

Large cost of operation and strict regulatory framework will restrict the growth of bone marrow-derived stem cells (BMSCS) market in the above mentioned forecast period. Ethical concern leading to stem cells will become the biggest challenge in the market growth.

Global Bone Marrow-Derived Stem Cells (BMSCS) Market By Service Type (Sample Preservation and Storage, Sample Analysis, Sample Processing, Sample Collection and Transportation), Application (Personalized Banking Applications, Research Applications, Clinical Applications), Country (U.S., Canada, Mexico, Germany, Italy, U.K., France, Spain, Netherland, Belgium, Switzerland, Turkey, Russia, Rest of Europe, Japan, China, India, South Korea, Australia, Singapore, Malaysia, Thailand, Indonesia, Philippines, Rest of Asia- Pacific, Brazil, Argentina, Rest of South America, South Africa, Saudi Arabia, UAE, Egypt, Israel, Rest of Middle East & Africa), Market Trends and Forecast to 2027

Global Bone Marrow-Derived Stem Cells (BMSCS) Market Scope and Market Size

Bone marrow-derivedstem cells(BMSCS) market is segmented on the basis of service type and application. The growth amongst these segments will help you analyse meagre growth segments in the industries, and provide the users with valuable market overview and market insights to help them in making strategic decisions for identification of core market applications.

Thisbonemarrow-derived stem cells (BMSCS) market report provides details of new recent developments, trade regulations, import export analysis, production analysis, value chain optimization, market share, impact of domestic and localised market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, strategic market growth analysis, market size, category market growths, application niches and dominance, product approvals, product launches, geographic expansions, technological innovations in the market. To gain more info on bone marrow-derived stem cells (BMSCS) market contactData Bridge Market Researchfor anAnalyst Brief, our team will help you take an informed market decision to achieve market growth.

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Healthcare Infrastructure Growth Installed Base and New Technology Penetration

Bone marrow-derived stem cells (BMSCS) market also provides you with detailed market analysis for every country growth in healthcare expenditure for capital equipments, installed base of different kind of products for bone marrow-derived stem cells (BMSCS) market, impact of technology using life line curves and changes in healthcare regulatory scenarios and their impact on the bone marrow-derived stem cells (BMSCS) market. The data is available for historic period 2010 to 2018.

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Competitive Landscape and Bone Marrow-Derived Stem Cells (BMSCS) Market Share Analysis

Bone marrow-derived stem cells (BMSCS) market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, production capacities, company strengths and weaknesses, product launch, product width and breadth, application dominance. The above data points provided are only related to the companies focus related to bone marrow-derived stem cells (BMSCS) market.

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Bone Marrow-Derived Stem Cells (BMSCS) Market : Size & Trends Shows a Rapid Growth by 2028 UNLV The Rebel Yell - UNLV The Rebel Yell

Bone Marrow Stem Cells Comments Off on Bone Marrow-Derived Stem Cells (BMSCS) Market : Size & Trends Shows a Rapid Growth by 2028 UNLV The Rebel Yell – UNLV The Rebel Yell | September 1st, 2021

Medical science is starting to license and use drugs and procedures that change the genetic code inside the bodys cells, and to correct the bad code that can give rise to conditions such as cancer and the auto-immune diseases. Since HIV is a disease that results from a virus inserting such a piece of bad code into our genes, such therapies could be used to snip out that code and effect a cure.

This was what attendees at last months International AIDS Society Conference on HIV Science (IAS 2021) heard at the workshop on curing HIV. The workshop opened with two introductory talks by Professor Hans-Peter Kiem, the chair of gene therapy at the Fred Hutchinson Cancer Research Center in Seattle in the US (the Fred Hutch) and, in a joint presentation, by the Fred Hutchs Dr Jennifer Adair and Dr Cissy Kityo of the Joint Clinical Research Centre (JCRC) in Kampala, Uganda.

The latter talk was a sign of acknowledgement that, while the prospects for genetic medicine are brighter than ever before, their expense and sophistication do not fit well with the global epidemiology of HIV, which mainly affects the worlds poorest and most disadvantaged communities. Despite this, Fred Hutch and JCRC have embarked upon a joint research programme to develop within the next few years a genetic therapy treatment for HIV that could be realistically scaled up for use in lower-income settings.

A unit of heredity, that determines a specific feature of the shape of a living organism. This genetic element is a sequence of DNA (or RNA, for viruses), located in a very specific place (locus) of a chromosome.

A type of experimental treatment in which foreign genetic material (DNA or RNA) is inserted into a person's cells to prevent or fight disease.

To eliminate a disease or a condition in an individual, or to fully restore health. A cure for HIV infection is one of the ultimate long-term goals of research today. It refers to a strategy or strategies that would eliminate HIV from a persons body, or permanently control the virus and render it unable to cause disease. A sterilising cure would completely eliminate the virus. A functional cure would suppress HIV viral load, keeping it below the level of detection without the use of ART. The virus would not be eliminated from the body but would be effectively controlled and prevented from causing any illness.

The body's mechanisms for fighting infections and eradicating dysfunctional cells.

In cell biology, a structure on the surface of a cell (or inside a cell) that selectively receives and binds to a specific substance. There are many receptors. CD4 T cells are called that way because they have a protein called CD4 on their surface. Before entering (infecting) a CD4 T cell (that will become a host cell), HIV binds to the CD4 receptor and its coreceptor.

HIV cure research pioneer Dr Paula Cannon of the University of Southern California, chairing the session, said: After several decades of effort and false starts, gene therapies now hold out promise for diseases that were previously untreatable.

Hans-Peter Kiem acknowledged the pivotal role of community advocacy in supporting cure research, noting that his project, defeatHIV, was one of the first beneficiaries of a grant from the Martin Delaney Collaboratories, named after the celebrated US treatment activist who died in 2009.

The other factor that gave impetus to HIV cure research was, of course, the announcement that someone had been cured: Timothy Ray Brown, whose HIV elimination was first announced in 2008 and who came forward publicly in 2010. He died in 2019 from the leukaemia whose treatment led to his HIV cure but by then had had 13 years of post-HIV life. He had survived long enough to talk with Adam Castillejo, the second person cured of HIV, and encourage him to come forward too.

Timothy and Adams stories showed that HIV could be cured, and with a crude form of gene therapy too: cancer patients, they were both given bone marrow transplants from donors whose T-cells lacked the gene for the CCR5 receptor, which is necessary for nearly all HIV infection.

But there have only been two cures for two reasons: firstly, bone marrow transplant is itself a very risky procedure involving deleting and replacing the entire immune system of already sick patients. In 2014 Browns doctor, Gero Hutter, reported that Timothy Ray Brown was only one of out of eight patients on whom the procedure had been tried, but that all the others had died.

Secondly, compatible bone marrow donors are hard to come by as it is, and restricting them to the 1% or so of people who lack the CCR5 receptor, all of them of northern European ancestry, means very few people could benefit from this approach. Attempting transplant with T-cells that do not lack CCR5, in the hope that replacing the immune system with cells from a person without cancer will also get rid of their HIV anyway, has produced temporary periods of undetectable HIV off therapy, but the virus has always come back.

(People like Brown and Castillejo, whose HIV infection was cured by medical intervention, need to be distinguished from people who seem to have spontaneously cured themselves, such as Loreen Willenberg: such people are of course of great interest to cure researchers, but the trick is to make it happen consistently in other people.)

Brown and Castillejos cures, as transplants, were so-called allogenic, meaning that the HIV-resistant cells came from another person. Better would be autogenic transplants, in which immune system cells are taken from a person with HIV, genetically altered in the lab dish to make them resistant to HIV, and then re-introduced. This type of procedure written about for aidsmap as long ago as 2011 by treatment advocate Matt Sharp, who underwent one.

The repertoire of gene therapies is not restricted to CCR5 deletion. Gene therapy is immensely versatile, and could be used in a number of ways.

Instead of using gene therapy to make cells resistant to HIV, it could directly repair defective genes in cells by means of cut-and-paste technology such as CRISPR/Cas9. This is already being used in trials for some genetic conditions such as cystic fibrosis and sickle-cell anaemia. Given that HIV-infected cells are also defective in the sense that they contain lengths of foreign DNA that shouldnt be there, they are amenable to the same molecular editing. Early trials have produced promising results but the challenge, as it has been in a lot of gene therapy, is to ensure that the cells containing DNA are almost entirely eliminated.

One way of doing this is not to delete the HIV DNA from infected cells but to preferentially kill off the cells themselves by creating so-called chimeric antigen receptor (CAR) T-cells. These are T-lymphocytes whose genes have been modified so that their usual receptors such as CD4 or CD8 have been replaced with receptors attuned very specifically to antigens (foreign or unusual proteins) displayed by infected cells and cancer cells. A couple of CAR cell therapies are already licensed for cancers; the problem with HIV is that the reservoir cells do not display immune-stimulating antigens on their surfaces. This means that CAR T-cells would have to be used alongside drugs such as PD-1 inhibitors that stop the cells retreating into their quiescent reservoir phase, an approach demonstrated at IAS 2021.

A couple of other approaches could be used to produce either vaccines or cures. One is to engineer B-cells so they produce broadly neutralising antibodies. A way of tweaking them to do this, called germline targeting, is covered was also discussed at IAS 2021, but if we manage to generate B-cells that can do this, we could then in theory directly edit their genes to make them do the same thing.

"Timothy Ray Brown and Adam Castillejo were both given bone marrow transplants from donors whose T-cells lacked the gene for the CCR5 receptor."

The other way is to induce cells to make viral antigens or virus-like particles that the immune system then reacts to. Scientists have been working on this technique for 20 years and it triumphed last year when the Pfizer and Moderna vaccines against the SARS-CoV-2 virus had over 90% success in suppressing symptomatic COVID-19. These vaccines are not genetic engineering in the sense of altering the genome of cells; rather, they introduce a product of the genetic activation in cells, the messenger RNA that is produced when genes are read and which is sent out into the rest of the cell to tell it to make proteins.

However because HIV is more variable and less immunogenic than SARS-CoV-2, the vaccine induced by the RNA would have to be something that looked much more like a whole virus than just the bare spike protein induced by the Pfizer and Moderna vaccines. If there was such a vaccine could be used both therapeutically as well as in prevention, by stimulating an immune reaction to activated HIV-infected cells. Moderna have announced they will now resume the HIV vaccine research they were working on when COVID-19 hit.

The problem with all these more gentle procedures is that it has proved difficult to replace all the HIV-susceptible cells with the HIV-resistant or HIV-sensitised ones: although engraftment takes place, meaning that the autologous cells are not rejected by the body and are able to establish a population for some time (in some animal experiments, replacing as much as 90% of the native immune cells), eventually the unaltered immune cells tend to win out because the introduced cells lack the deep reservoir of replenishing cells.

Kiem said that the way scientists have been trying to get round this is to only select and alter so-called haematopoeic stem cells (HSCs). These rare and long-lived cells, found in the bone marrow, are the replenishing reservoir of the immune system. They differentiate when they reproduce and give rise to all the immune cells that do different things: CD4 and CD8 T-lymphocytes, B-cells that make antibodies, macrophages that engulf pathogens, dendritic cells, monocytes, natural killer cells, and others.

Altering HSCs genetically so that they are able to fight HIV in one way or another could in theory give rise to a persistent, HIV-resistant immune system. They could in theory lie in wait and be ready to produce effector cells of various types. They would be ready when a new HIV infection comes along (if used as a vaccine) or when HIV viral rebound happens and there is detectable virus in the body (if used as part of a cure). If a person with CAR-engineered stem cells could have repeated cycles of treatment interruption, their HIV reservoir could in theory slowly be deleted.

"Gene therapies are astonishingly expensive."

As mentioned above, although genetic medicine shows enormous promise, the complexity and expense of its techniques means that at present it is unlikely to benefit most people who really need it.

Hans-Peter Kiem said that currently about 60 million people have conditions that could benefit from gene therapy. The vast majority of these either have HIV (37 million) or haemoglobinopathies blood-malformation diseases such as sickle-cell anaemia and thalassaemia that are also concentrated in the lower-income world (20 million).

Dr Jennifer Adair, one of the first researchers to have proposed collaboration on gene therapies for HIV with African institutes, said that gene therapies have already been licensed for conditions such as thalassaemia, spinal muscular atrophy, T-cell lymphoma and a form of early-onset blindness.

But they are astonishingly expensive. The worlds most expensive drug tag goes, depending on which source you read, either to Zynteglo, a genetic medicine correcting malformed beta-haemoglobin and licensed in the US for thalassaemia, or Zolgensma, a drug licensed in Europe and given to children to correct the defective gene that results in spinal muscular atrophy.

Both cost about 1.8 million for a single dose. The price is not just due to the cost of the complex engineering used to make them, but because they are used to treat rare diseases and so have a small market.

At present the technology need to engineer autogenic genetically engineered cells is, if anything, even more expensive and complex than that needed to introduce allogenic cells. It can involve in the region of ten staff and a workspace of 50 square metres per patient. Recently a so-called gene therapy in a box has been made available that can reduce the area needed to produce autogenic genetically-engineered cells from 50 to less than one square metre, and the staff need to one or two, But what is really needed is genetic engineering in a shot; a therapy similar to a vector or RNA vaccine that can be introduced as an injection and produces the genetic changes needed within the body.

Undaunted by the challenges, the US National Institutes of Health are collaborating with the Bill and Melinda Gates foundation to work on a combined programme of HIV and sickle-cell-anaemia genetic therapy (given that something that works for one could be adapted to work with the other).

And the Fred Hutchinson Center has teamed up with the Joint Clinical Research Centre in Uganda with the very ambitious goal of making a genetic therapy that would be at least ready for human testing within two years in an African setting, and that could be scaled up to be economical for Africa if successful.

Dr Cissy Kityo of JCRC in Uganda told the conference that as of 2020, there were 373 trials of gene therapy products registered, of which 35 were in phase III efficacy trials. The global budget for regenerative medicine, which includes genetic therapy and related techniques, was $19.9 billion, having jumped by 30% since the previous year. The US Food and Drug Administration projects that based on the current rate of progress and the development pipeline, they may be licensing around 100 gene-therapy products a year by 2025.

This branch of medicine is no longer exotic, she said. Now steps have to be taken to trial gene therapies in the people who needed them most, and to turn the exotic into the affordable, she added.

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Could gene therapies be used to cure more people with HIV? - aidsmap

Bone Marrow Stem Cells Comments Off on Could gene therapies be used to cure more people with HIV? – aidsmap | September 1st, 2021

DetailsCategory: DNA RNA and CellsPublished on Tuesday, 31 August 2021 18:23Hits: 284

Encouraging data confirming activity in a solid tumor indication presented on first nine patients at low dose cohorts in ongoing autologous CAR-T trial in metastatic castrate-resistant prostate cancer

Three patients showed a greater than 50% decline in prostate-specific antigen (PSA) and concordant PSMA-PET imaging results, including one patient at lowest dose with evidence of complete tumor elimination

Favorable safety profile with modest overall rates of CRS and no neurotoxicity observed

SAN DIEGO, CA, USA I August 31, 2021 I Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary genetic engineering platform technologies to create cell and gene therapeutics with the capacity to cure, today announced preliminary results from its Phase 1 clinical trial of P-PSMA-101, the Company's solid tumor autologous CAR-T product candidate to treat patients with metastatic castrate-resistant prostate cancer (mCRPC). These data will be presented at the 6th Annual CAR-TCR Summit virtual meeting at 10:00am ET today in a presentation entitled, "P-PSMA-101 is a High-Tscm Autologous CAR-T Targeting PSMA Producing Exceptionally Deep and Durable Responses in Castration-Resistant Metastatic Prostate Cancer."

"We are excited about the preliminary data from our Phase 1 trial of P-PSMA-101, which provides further evidence of the effectiveness of our CAR-T platform for solid tumor cancers," said Eric Ostertag, M.D., Ph.D., Chief Executive Officer of Poseida, who will present at the CAR-TCR Summit. "To date, other CAR-T therapeutics have not had much success outside of hematologic malignancies. The deep and durable responses in our trial demonstrate that CAR-T products have the potential to work well against solid tumors, even at low doses, when using the appropriate technology platform."

Efficacy:

As of the cutoff date, the study had enrolled a total of nine patients with mCRPC: five patients at Dose A who each received a single treatment of 0.25X10E6 cells/kg (an average of about 20M cells), and four patients at Dose B, who each received a single treatment of 0.75X10E6 cells/kg (an average of about 60M cells). All patients received a lymphodepletion regimen consisting of 30 mg/m2 fludarabine + 300 mg/m2 cyclophosphamide. Patients were heavily pre-treated, having received an average of six prior lines of therapy with a median time since diagnosis of 6.4 years.

Key findings included:

-Five patients dosed showed measurable declines in PSA levels-Three patients treated showed a greater than 50% decline in PSA levels and had concordant improvements in PSMA-PET imaging-One patient demonstrated evidence of complete tumor elimination and remains in a durable response of greater than five months at the time of this presentation

"This innovative Poseida PSMA-directed CAR T cell platform has demonstrated a robust anti-tumor response in patients with metastatic castration resistant prostate cancer," commented Susan F. Slovin, M.D., Ph.D., Associate Vice Chair of Academic Administration at Memorial Sloan Kettering Cancer Center and investigator on the trial. "This is the first time that I have seen such impressive responses with an immunotherapy product. The responses of my patients in the trial are far beyond my expectations."

Safety and Tolerability:

P-PSMA-101 demonstrated a favorable safety and tolerability profile. After a previously reported case of Macrophage Activation Syndrome (MAS) exacerbated by patient non-compliance, only three cases of possible Cytokine Release Syndrome (CRS) were observed, which were all low grade (1/2) and were managed well with early treatment. No cases of neurotoxicity (CRES/ICANS) were observed as of the cutoff date.

The Phase 1 trial is an open label, multi-center, 3+3 dose-escalating study designed to assess the safety of P-PSMA-101 in up to 40 adult subjects with mCRPC. The primary objectives of this study are to determine the safety, efficacy, and maximum tolerated dose of P-PSMA-101. Additional information about the study is available at http://www.clinicaltrials.gov using identifier: NCT04249947.

"We believe the key to success in solid tumors is a product with a high percentage of desirable stem cell memory T cells (Tscm)," said Matthew Spear, M.D., Chief Medical Officer of Poseida. "In this study, we have demonstrated that a high-percentage Tscm CAR-T product can home to the bone marrow and, in at least one case, completely eliminate tumor. This bone marrow homing property may be particularly important for bone avid diseases such as prostate adenocarcinoma. Importantly, the favorable tolerability associated with our Tscm CAR-T products has carried over to prostate cancer where we have so far seen manageable cytokine release syndrome and no neurotoxicity."

Company-Hosted Conference Call and Webcast Information

Poseida's management team will host a conference call and webcast today, August 31, 2021 at 11:00am ET. The dial-in conference call numbers for domestic and international callers are (866) 939-3921 and (678) 302-3550, respectively. The conference ID number for the call is 50220147. Participants may access the live webcast and the accompanying presentation materials on Poseida's website at http://www.poseida.com in the Investors section under Events and Presentations. An archived replay of the webcast will be available for 30 days following the event.

Additional CAR-TCR Summit Highlights

Presentation: "Developing CAR-T Cells for Multiple Myeloma: From Autologous to Allogeneic"Session Date/Time: Wednesday, September 1, 2021, 4:00pm ETPresenter: Matthew Spear, M.D., CMO, Poseida Therapeutics

This presentation will outline Phase 1 and 2 development of the Company's lead autologous P-BCMA-101 CAR-T therapy and insights that were used to develop a fully allogeneic version, P-BCMA-ALLO1 that is expected to enter the clinic soon. The presentation will be part of the afternoon session on the Clinical Management Track.

Presentation: "Advancing Nonviral Manufacturing for Multi-Product Allogeneic T-Cell Therapies"Session Date/Time: Wednesday, September 1, 2021, 4:30pm ETPresenter: Devon Shedlock, Ph.D., SVP Research & Development, Poseida Therapeutics

This presentation will discuss how Poseida's piggyBac DNA Delivery System, Cas-CLOVER Site-specific Gene Editing System and Booster Molecule are used to manufacture multi-product, fully allogeneic T-cell therapies. The Company will also discuss how efficient multiplexed Cas-CLOVER gene editing exhibits low to no off-target editing or translocations as determined by next-generation sequencing, and how the Company's Booster Molecule helps to protect against the "allo tax," maintaining a favorable high-stem cell memory T cell (Tscm) product and enabling up to hundreds of doses in a single manufacturing run. This presentation will be part of the afternoon session on the Manufacturing Track.

Presentation: "Developing 'Off-the-Shelf' CAR-T Cells for Bone Marrow Transplant Conditioning"Session Date/Time: Thursday, September 2, 2021, 9:00am ETPresenter: Nina Timberlake, Ph.D., Associate Director, Research (Gene Therapy), Poseida Therapeutics

This presentation will discuss leveraging the piggyBac DNA Delivery System and Cas-CLOVER Site-specific Gene Editing System to generate off-the-shelf fully allogeneic CAR-T cells to specifically target hematopoietic cells in the bone marrow. This potential therapeutic could be used as a non-myeloablative conditioning regimen for hematopoietic stem cell transplant or as a therapeutic for the treatment of acute myeloid leukemia (AML). The presentation will occur as part of the conference's Focus Day, "CAR-TCR Beyond Oncology: Fundamental Biology & Mechanisms of Action Beyond Oncology."

The full presentations at the CAR-TCR Summit will be made available on Poseida's website at their respective session times.

About Poseida Therapeutics, Inc.

Poseida Therapeutics is a clinical-stage biopharmaceutical company dedicated to utilizing our proprietary genetic engineering platform technologies to create next generation cell and gene therapeutics with the capacity to cure. We have discovered and are developing a broad portfolio of product candidates in a variety of indications based on our core proprietary platforms, including our non-viral piggyBac DNA Delivery System, Cas-CLOVER Site-specific Gene Editing System and nanoparticle- and AAV-based gene delivery technologies. Our core platform technologies have utility, either alone or in combination, across many cell and gene therapeutic modalities and enable us to engineer our wholly-owned portfolio of product candidates that are designed to overcome the primary limitations of current generation cell and gene therapeutics. To learn more, visit http://www.poseida.com to connect with us on Twitter and LinkedIn.

SOURCE: Poseida Therapeutics

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Poseida Therapeutics Presents Preliminary Results from Phase 1 Trial of P-PSMA-101 at the 6th Annual CAR-TCR Summit | DNA RNA and Cells | News...

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REGULATED INFORMATION

No statistically significant difference in knee pain reduction between JTA-004, placebo and active comparator, 3months after treatment; favorable JTA-004 safety profile similar to placebo and comparator

Prime focus on the continued development and expansion of its mesenchymal stromal cell based allogeneic cell and gene therapy platform

Management to host conference call today at 4pm CEST / 10am EST - details provided below

Gosselies, Belgium, 30August 2021, 7am CEST BONE THERAPEUTICS (Euronext Brussels and Paris: BOTHE), the cell therapy company addressing unmet medical needs in orthopedics and other diseases, today announces that the Phase III knee osteoarthritis study with its enhanced viscosupplement JTA-004 did not meet the primary and consequently the key secondary endpoints.

The primary objective of the JTA-004 Phase III study was to demonstrate the efficacy of JTA-004 in reducing osteoarthritic knee pain compared to placebo as measured by the WOMAC pain subscale three months after treatment. A key secondary objective was the comparison between JTA-004 and comparator Hylan G-F 20 in knee pain relief at month 3. Despite JTA-004s favorable safety profile, the study did not achieve its main objectives as no statistically significant difference in pain reduction could be observed between any of the treatment, placebo and comparator groups, with all treatment arms showing similar efficacy.

A statistically significant difference in favor of JTA-004 and the active comparator versus placebo was seen in a post-hoc analysis in a subset of patients with higher pain scores at entry.

The Company, in collaboration with existing and potential partners, will consider the options for the future of JTA-004 development.

The execution of the study was flawless and a good safety profile was observed in line with previous results. These JTA-004 efficacy results are disappointing. Knee osteoarthritis studies are recognized across the industry to be challenging to evaluate. They are also frequently complicated by a high placebo effect. We will continue to analyze the data and will consider potential next steps, said Miguel Forte, Chief Executive Officer of Bone Therapeutics. We are now fully committed to the clinical development of our advanced MSC allogeneic cell and gene therapy platform. Bone Therapeutics is concentrating on the development of this platform for the large market of orthopedic indications, with ALLOB. The progress with this platform has enabled us to expand it to other indications, including immunomodulation.

Bone Therapeutics is focused on the development of its core assets, the allogeneic cell therapy platform, including ALLOB. ALLOB is currently being evaluated in a randomized, double-blind, placebo-controlled Phase IIb study in 178patients with fresh tibial fractures at risk of delayed or non-union. 5% to 10% of complicated long bone fractures evolve to delayed union and non-union. This study will assess the potential for a single percutaneous injection of ALLOB to accelerate fracture healing and prevent late-stage complications in these patients. Recruitment is expected to be completed in the first half of 2022 and topline results by the end of 2022. Should the pandemic continue, Bone Therapeutics may have to re-evaluate these timelines and, in that eventuality, will communicate again to the market.

Bone Therapeutics is intensifying its efforts to expand its preclinical and clinical pipeline with additional indications by enhancing and professionalizing the therapeutic capacity of its cell and gene therapy platform. This includes the development of a next generation of genetically engineered mesenchymal stromal cells (MSC) and the use of highly scalable and versatile cell sources such as induced pluripotent stem cells (iPSC).

Conference call

The management of Bone Therapeutics will host a conference call today at 4:00 pm CEST / 10:00 am EST. To participate in the conference call, please select your dial-in number from the list below quoting the conference ID 825 1002 3115#:

Belgium: +32 2 290 9360France: +33 1 7095 0103United Kingdom: +44 208 080 6592United States: +1 646 876 9923

About JTA-004 and Phase III knee osteoarthritis study

JTA-004 is Bone Therapeutics next generation of intra-articular injectable for the treatment of osteoarthritic pain in the knee. It consists of a unique mix of hyaluronic acid - a natural component of knee synovial fluid, plasma proteins, and a fast-acting analgesic. JTA-004 intends to provide added lubrication and protection to the cartilage of the arthritic joint and to alleviate osteoarthritic pain.

The JTA-004 Phase III study is a controlled, randomized, double-blind trial. It evaluates the potential of a single, intra-articular injection of JTA-004 to reduce osteoarthritic pain in the knee, compared to placebo or Hylan G-F 20, the leading osteoarthritis treatment on the market. The study is being conducted in 22 centers across six European countries as well as Hong Kong. More than 700 patients with mild to moderate symptomatic knee osteoarthritis were treated in this study.

About Knee Osteoarthritis

Osteoarthritis (OA), also known as degenerative joint disease, is the most common chronic joint condition in which the protective cartilage in the joints progressively break down resulting in joint pain, swelling, stiffness and limited range of motion. The knee is one of the joints that are mostly affected by osteoarthritis, with an estimated 250 million cases worldwide.

The prevalence of knee osteoarthritis (KOA) is expected to increase in the coming years due to increasingly aging and obese population. Currently, there is no cure for KOA and treatments focus on relieving and controlling pain and symptoms, preventing disease progression, minimizing disability, and improving quality of life. Most drugs prescribed to KOA patients are topical or oral analgesics and anti-inflammatory drugs. Ultimately, severe KOA leads to highly invasive surgical interventions such as total knee replacement.

About Bone Therapeutics

Bone Therapeutics is a leading biotech company focused on the development of innovative products to address high unmet needs in orthopedics and other diseases. The Company has a diversified portfolio of cell therapies at different stages ranging from pre-clinical programs in immunomodulation to mid stage clinical development for orthopedic conditions, targeting markets with large unmet medical needs and limited innovation.

Bone Therapeutics core technology is based on its cutting-edge allogeneic cell and gene therapy platform with differentiated bone marrow sourced Mesenchymal Stromal Cells (MSCs) which can be stored at the point of use in the hospital. Currently in pre-clinical development, BT-20, the most recent product candidate from this technology, targets inflammatory conditions, while the leading investigational medicinal product, ALLOB, represents a unique, proprietary approach to bone regeneration, which turns undifferentiated stromal cells from healthy donors into bone-forming cells. These cells are produced via the Bone Therapeutics scalable manufacturing process. Following the CTA approval by regulatory authorities in Europe, the Company has initiated patient recruitment for the Phase IIb clinical trial with ALLOB in patients with difficult tibial fractures, using its optimized production process. ALLOB continues to be evaluated for other orthopedic indications including spinal fusion, osteotomy, maxillofacial and dental.

Bone Therapeutics cell therapy products are manufactured to the highest GMP (Good Manufacturing Practices) standards and are protected by a broad IP (Intellectual Property) portfolio covering ten patent families as well as knowhow. The Company is based in the BioPark in Gosselies, Belgium. Further information is available at http://www.bonetherapeutics.com.

For further information, please contact:

Bone Therapeutics SAMiguel Forte, MD, PhD, Chief Executive OfficerJean-Luc Vandebroek, Chief Financial OfficerTel: +32 (0)71 12 10 00investorrelations@bonetherapeutics.com

For Belgian Media and Investor Enquiries:BepublicCatherine HaquenneTel: +32 (0)497 75 63 56catherine@bepublic.be

International Media Enquiries:Image Box CommunicationsNeil Hunter / Michelle BoxallTel: +44 (0)20 8943 4685neil.hunter@ibcomms.agency / michelle@ibcomms.agency

For French Media and Investor Enquiries:NewCap Investor Relations & Financial CommunicationsPierre Laurent, Louis-Victor Delouvrier and Arthur RouillTel: +33 (0)1 44 71 94 94bone@newcap.eu

Certain statements, beliefs and opinions in this press release are forward-looking, which reflect the Company or, as appropriate, the Company directors current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its subsidiary undertakings or any such persons officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.

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Bone Therapeutics announces topline results from Phase III knee osteoarthritis study with its enhanced viscosupplement JTA - GlobeNewswire

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A WOMAN blamed stress for her three-month long period until doctors found out the true cause was devastating.

Bansri Dhokia, 30, from Ealing, West London, is now urging others to see their GP as soon as they are unwell.

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She thought at worst, her odd periods, fatigue and breathlessness would be down to anaemia or low thyroid function that could be treated with medication.

But the truth was far worse, and Bansri was diagnosed with a blood cancer.

Bansri was taken into hospital that night where she stayed for 12 weeks having intense treatment to save her life.

Speaking of her symptoms, which started in May 2020, Bansri said: I blamed it on being overworked.

With blood cancer, the symptoms are often quite vague and hard to diagnose.

I really noticed the fatigue first. I could sleep for 12 hours a night and still feel exhausted.

Then I started to get breathless all the time. There were activities like climbing stairs or walking down the road that I used to find easy but was suddenly finding more difficult.

Bansris heavy period, which had been ongoing for three months, was particularly unusual for her.

She made repeated trips to the doctor to find out what was wrong but kept being pushed back.

I just knew something wasn't right and repeatedly asked for blood tests, Banrsri said.

The first four blood tests between May and July came back clear and by the time she had a fifth on 21 July, she was starting to get fed up.

Busy with work, Bansri almost missed the appointment but luckily, her husband Amrit Sagoo encouraged her to go.

She said: I went for the blood test in the afternoon and that evening, I was brushing my teeth when I got a call to say the ambulance was coming to collect me.

They explained I needed to go to hospital right away. I thought it was just for a night and packed an overnight bag.

"I didnt know what was wrong and that I would end up staying in hospital for 12 weeks.

Tests at the Royal London hospital revealed Bansri had acute lymphoblastic leukaemia (ALL), a rare cancer affecting just 790 people in the UK each year, mostly children and young people.

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A problem in the bone marrow leads to insufficient important blood cells, causing symptoms of unusual bleeding, tiredness and muscle aches.

Almost seven in 10 will survive ALL for five years or longer after diagnosis, and four in ten in those aged 25 to 64.

Bansri said: I didn't know much about leukaemia. I was really scared for my life. I had no idea what the prognosis was. I just cried and I kept questioning why this was happening to me."

With lockdown restrictions still in place, Bansri had to tell her friends and family about her diagnosis over Zoom.

She said: It was the hardest thing I have ever had to do. I asked my sister to gather my family in the living room. We are very close and I could not look at her because I just couldn't deal with seeing the sadness in her face."

Bansri started chemotherapy straight away, because ALL is very aggressive and develops quickly.

She said: "It was so upsetting seeing pieces of my hair fall out on my pillow. I was growing it as we were planning to have Hindu and Sikh religious wedding ceremonies in 2020, after our civil wedding the year before.

"One day I just asked the nurse to shave my head, and in that moment, I felt really empowered."

But one of the hardest parts of the treatment - which she now needs therapy to recover from - is that she couldnt have visitors for the first eight weeks due to Covid.

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Bansri then needed a stem cell transplant to improve the chances that she would go into remission.

During the procedure, the patient has stem cells of a donor, sometimes a complete stranger, injected into their blood. The cells find their way to the bone marrow, helping it to start making normal cells again.

Most people who are white Europeans find a match from a related or unrelated donor on a large registry, but 400 UK patients don't find a suitable donor each year.

Bansri said: I knew immediately that being from an Indian background, there was a very low chance that I would find a match.

According to charities, donors are more likely to be white, and people from minority ethnic backgrounds are more likely to have rarer tissue types, making it harder to find patients from these backgrounds a matching donor.

That was quite scary because I knew how important it was to have a donor to save my life, Bansri said.

Luckily one of Bansris two siblings was a match, and the transplant took place in February 2021.

Bansri said: My recovery is going well so far but a stem cell transplant comes with many side effects, which are lifelong.

I have a long road to go but I take it day by day. Each month I get through is a success."

Bansri is vulnerable to infections because the transplant made her immune system weaker, and so she and her husband are still having to shield.

Bansri is urging people to join the stem cell donor register, particularly those in Asian communities.

HOW YOU CAN HELP SAVE A LIFE

REGISTERING to be a blood stem cell donor is easy.

Even if you can't donate to your relative, you might be ableto become a donor for someone else. You can do this by contacting one of the UK registers.

There are different donor registersin the UK.These work with each otherand with international registersto match donors with people who need stem cells.

You can sign up with:

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She said: People often have a misconception that, when you join the donor registry, you're giving something up, for example, in a kidney transplant, you do give up your kidney, and it's a longer recovery time.

My sibling was in hospital for a few hours on the day and didn't have any side effects afterwards.

In my community, cancer is a bit of a taboo subject and people dont speak about it so I think there is a lack of awareness of the importance of signing up to be on the register.

Bansri is also taking part in the Leukaemia Cares Spot Leukaemia campaign, which urges the general public to understand and recognise the signs.

She said: I want to see more Asian people talking about it because its not the fault of the person - its just bad luck.

If youre experiencing any of the symptoms, contact your GP and ask for a blood test. Early diagnosis saves lives.

Symptoms of acute lymphoblastic leukaemia

The NHS says most of the symptoms of ALL are caused by a lack of healthy blood cells. They include:

In some cases, the affected cells can spread from your bloodstream into your central nervous system. This can cause neurological symptoms (related to the brain and nervous system), including:

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I blamed stress for my three-month period but it turned out to be much more sinister... - The Sun

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T-cell leukemia is an uncommon form of cancer that causes a type of white blood cells known as T cells to grow uncontrollably in a persons bone marrow. Adult T-cell leukemia (ATL) is one form of the condition and results from a viral infection.

This information comes from the American Cancer Society.

Experts call the virus that causes ATL human T-cell leukemia virus type-1 (HTLV-1). This virus can also cause a type of lymphoma that begins in the immune system rather than the bone marrow. Doctors refer to the two conditions collectively as adult T-cell leukemia/lymphoma (ATLL).

This article looks at the types, symptoms, and causes of ATL. It also considers the treatment options and survival rates for those with the condition.

T-cell leukemia is a relatively rare form of cancer. Like other types of leukemia, it affects blood-forming cells in the bone marrow. These are cells that will go on to become blood cells but that are still in their early, or immature, form.

In addition to plasma, blood consists of red blood cells, white blood cells, and platelets. These cells go through several stages of development before becoming mature.

T-cell leukemia causes an abnormality in a specific type of immature white blood cells, known as T lymphocytes or T cells. T cells protect the body from infection. If these cells become cancerous, they divide and grow uncontrollably.

ATL is a type of T-cell leukemia caused by HTLV-1. The condition is rare in the United States, but it is more common in Japan, parts of Africa, South America, the Middle East, and the Caribbean.

Most people who contract HTLV-1 do not go on to develop any serious disease. HTLV-1 affects around 10 million people globally, but only 25% of them develop symptomatic ATLL.

There are four subtypes of ATLL:

Learn about the differences between leukemia and lymphoma here.

Symptoms of ATL can vary depending on the subtype a person has. People with the smoldering subtype may not have noticeable symptoms or may only develop a few skin lesions.

People with more aggressive forms of ATL may experience:

Hypercalcemia can be serious. It causes symptoms such as:

Children who develop ATLL often experience the acute or lymphomatous subtypes. Many develop an enlarged thymus, an organ that resides in front of the trachea, or windpipe. This can cause breathing problems.

To diagnose ATLL, a doctor will need to take samples of blood, bone marrow, or tissue. This may involve:

A healthcare professional can take blood from a vein in the arm, whereas bone or bone marrow usually comes from a hip bone.

A specialist will then examine the samples for signs of ATLL using various methods. They may use a microscope to examine them closely, or machines that test the samples proteins and DNA.

It is possible for ATL to go into remission. This means doctors cannot detect ATL in the body, and a person has no symptoms. Remission can be permanent or temporary.

However, ATL is often aggressive. The acute, lymphoma, and unfavorable chronic subtypes are more difficult to treat, while the favorable chronic and smoldering subtypes have a better prognosis.

For slow-growing forms of ATL, doctors may adopt a watch and wait approach to see whether the condition progresses. If the symptoms are mild and do not progress, a person may not need treatment or may not require it for some time.

Around 25% of cases of chronic or smoldering ATLL ultimately progress to the acute form. Anyone with acute ATLL typically undergoes treatment.

First-line treatment for ATL is antiviral therapy. Those with the lymphoma subtype seem to respond better to chemotherapy. Depending on the circumstances, a doctor may recommend one or both approaches.

Some individuals may also receive a stem cell transplantation from a donor, although this combined treatment is still under evaluation for its effectiveness.

Clinical trials are also testing the potential of immunomodulating drugs for ATL treatment.

ATL has a short overall survival rate, even with prompt treatment. This is because ATL is resistant to chemotherapy.

Data from 20002009 show that ATL patients who underwent intensive chemotherapy followed by stem cell transplantation had average survival times of:

Anyone who has completed treatment for ATLL will receive continuous health monitoring as part of their follow-up care. As the time spent in remission increases, these appointments become less frequent.

ATL occurs due to an infection with HTLV-1, a virus that belongs to the same class of viruses as HIV.

Similarly to HIV, HTLV-1 spreads through contact with bodily fluids. It can transmit through:

There is no cure or vaccine for HTLV-1. There is also no consistent method of screening for HTLV-1 worldwide and no way for doctors to predict who will go on to develop ATL. For this reason, preventing its spread is vital for preventing ATL.

However, because most people with HTLV-1 experience no symptoms, this can be challenging. Few studies have looked at the best ways of preventing HTLV-1 transmission.

Approaches that may help include:

Individuals should seek guidance from a doctor if they have any concerning symptoms, such as new rashes, skin lesions, or persistent fatigue.

They should also contact a doctor if they have come into contact with HTLV-1 at any point.

Where possible, schedule regular doctor visits. These give doctors an opportunity to perform physical examinations and obtain blood tests, as necessary. These may pick up on early signs of illness.

Some people with the slow-growing subtypes of T-cell leukemia have no symptoms. For these individuals, it is essential to visit a doctor as soon as any symptoms appear, because this could be a sign of the disease progressing.

ATL is a type of leukemia that affects the bodys T cells, which play a crucial role in the immune system. An HTLV-1 infection causes ATL.

ATL is often aggressive and difficult to treat. Those with a slow-growing subtype, such as smoldering ATL, usually have a better prognosis. The treatment may involve chemotherapy, antiviral drugs, or stem cell transplantation.

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DUBLIN--(BUSINESS WIRE)--The "Hematopoietic Stem Cell Transplantation (HSCT) Market - Size, Share, Outlook, and Opportunity Analysis, 2019 - 2027" report has been added to ResearchAndMarkets.com's offering.

The global hematopoietic stem cell transplantation market is expected to witness significant growth during the forecast period owing to the increasing prevalence of leukemia and lymphoma. According to Center for Disease Control and Prevention (CDC), in the U.S., around 45,360 people were diagnosed with leukemia in 2013, leading to 23,549 fatalities (13,625 men and 9,924 women). According to the same source the condition is more prevalent among men than women. Leukemia accounts for around 3% of all new cancer cases.

Hematopoietic stem cell transplantation is a procedure in which multipotent hematopoietic stem cells sourced from peripheral blood cells, bone marrow, or umbilical cord blood are transplanted into the patient. Hematopoietic stem cell transplantation is commonly used in the treatment of lymphoma (Hodgkin, Non-Hodgkin), leukemia, multiple myeloma, thalassemia, sickle cell anemia, and osteoporosis. It includes two transplantation sources; 1) autologous, that uses stem cells from the patient's own body, 2) and allogeneic that sources stem cells from a donor's body. According to World Health Organization (WHO), over 50,000 hematopoietic stem cell transplantation procedures are carried out globally, every year and this number is expected to increase over the years.

Company Profiles

Key features of the study:

Key Topics Covered:

1. Research Objectives and Assumptions

2. Market Overview

3. Market Dynamics, Regulations, and Trends Analysis

4. Impact Analysis of COVID-19

5. Global Hematopoietic Stem Cell Transplantation (HSCT) Market, By Transplant Type, 2016 - 2027, (US$ Million)

6. Global Hematopoietic Stem Cell Transplantation (HSCT) Market, By Indication, 2016 - 2027, (US$ Million)

7. Global Hematopoietic Stem Cell Transplantation (HSCT) Market, By Application, 2016 - 2027, (US$ Million)

8. Global Hematopoietic Stem Cell Transplantation (HSCT) Market, By Region, 2016 - 2027, (US$ Million)

9. Competitive Landscape

10. Section

For more information about this report visit https://www.researchandmarkets.com/r/jtneqg

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Global Hematopoietic Stem Cell Transplantation Market to 2027 - Size, Share, Outlook, and Opportunity Analysis - ResearchAndMarkets.com - Business...

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John Davy of Hancock marks two birthdays. The first isNov. 16, the day he was born in 1941. The second is Jan. 6,the day in 2014 when he received a lifesaving stem-cell transplantthanks to a complete stranger.

Now, John and his wife Sandhy Kale have become advocates for Be the Match, the national stem cell registry that found John his rare genetic match.

How often in the world do you get to save someones life? Davy asked. You fantasize about it sometimes. Heres an opportunity for anyone between 18 and 44 to do just that.

Davy said he began feeling abnormally tired sometime in 2013. One day, he walked to the mailbox, only a few hundred feet away from his front door, and had to stop several times on his way back to his house.

I said, Thats not me. Theres something off here, Davy said.

Davy went in to the hospital for some testing, and after a few false starts looking at his heart and running stress tests, doctors performed a Complete Blood Count, or CBC.

My blood count was so low, it wouldnt support life, he said.

Thats when Davy received his diagnosis. Myelodysplastic syndrome, or MDS, a form of blood cancer.

My first thought was, OK, what are we going to do about this? Davy said. Thats when the doctor told me there was no cure.

MDS cannot be cured through usual chemotherapy or radiation treatments. However, it can be treated with bone marrow or, as with Davy, the transplant of stem cells.

After receiving a second opinion, and speaking with a doctor experienced with stem cell transplants, Davy went on the national stem cell registry, known as Be the Match.

He was told he might have to wait upwards of a year before finding his match. But Davy got lucky in only three months, a viable donor joined the registry.

Davy knows little about the man who saved his life. He was 30 years old at the time, and a member of the United States military. Be the Match allows donors and patients to connect, if both sides are interested, but while John said he would love to shake the mans hand, his donor has wished to remain anonymous.

If I could speak to him, I would thank him profusely. For someone to be that generous, to donate to someone that hes never met, is astounding, Davy said.

Joining the Be the Match registry is as simple as swabbing a cheek.

Your genetic profile goes into the system, and, if donors are found to be a match to any patients waiting for transplants, only then are they called to go through the donation process.

There are two ways to donate stem cells. In either case, the donor will first undergo two injections to increase the production of their stem cells. In the first type of donation procedure, liquid bone marrow is extracted using a needle while the donor is under anesthesia. But the much more common way to donate used about 80 percent of the time is through a blood donation.

Similar to the process for donating plasma, the donor has blood drawn, it is cycled by a machine to remove only the stem cells, and the remaining blood is returned to the donor.

The recipient of the stem cells has to undergo a process to suppress their immune system, and the donated stem cells are given to the patient.

Because the immune system has to be repressed to accept the new cells, there is danger in the procedure, and even those who successfully accept the new stem cells can experience side effects of graft-verses-host reactions.

There is no guarantee, Kale said. This is a chance. You can take it if you want. Even if it buys you four or five years, you might get to see your kids graduating, your grandkids grow up. It was worth it to us.

And for Davy, they said, there was no other option. He accepted the risk, and said hes one of the lucky ones he had one minor reaction resulting in a rash across his chest, but overall, since his transplant, he has been able to resume a normal life. Today, seven years later, he is on no medications, and has no restrictions for how he can live his life.

It is that new lease on life that Davy said convinced himself and Sandhy that they had to become involved with Be the Match on a level besides being a recipient of their services. The two are now advocates for the system, traveling to drives to tell their story, and Davy acts as a support person for patients who may be recipients of transplants, telling them about what to expect in the process.

Its crucial, Davy said, to get as many people on the registry as possible. Because matches work on how genetically compatible two people are, people of similar ethnic backgrounds are more likely to match, and your ethnicity greatly impacts the likelihood of finding a good match.

White patients are the most likely to find a match within the system, at a rate of 79 percent. Native Americans have a 60 percent chance, Hispanic people a 48 percent, Asian 47 percent, and Black people only 29 percent.

Thats why Sandhy and I try to get as many people involved as we can, Davy said. The more people in the registry, the better chance you have.

Be the Match currently has a donor drive scheduled for Aug. 14 from 10 a.m. to 2 p.m. at E. Paul Community Center at 61 South Street in Troy. To join the registry you must be between the ages of 18 and 44 and be in good general health, and committed to donating to anyone in need. If you cannot attend the physical drive, a free cheek swab kit will be mailed to you. If you are interested in a kit, text TroyFD to 61474.

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Hancock man advocates for national bone marrow and stem cell registry that saved his life - Monadnock Ledger Transcript

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