Early findings from the phase 1/2a TEM-GBM study presented at the 2022 AACR Annual Meeting displayed potential of temferon to affect the tumor microenvironment of glioblastoma.

Immune system activation and tumor microenvironment alteration were effects observed in patients with glioblastoma treatment with temferon, a genetically modified Tie2-expressing monocyte (TEM) targeting interferon-2 (IFN2), according to early findings of the phase 1/2a TEM-GBM study (NCT03866109) presented in a poster at the American Association for Cancer Research (AACR) 2022 Annual Meeting.

These results provide the initial evidence for on-target activity of Temferon in GBM, said Bernard Gentner, MD, study coauthor and the leader of the translational stem cell and leukemia research unit at San Raffaele Telethon Institute for Gene Therapy in Milan, Italy.

Temferon is an investigational advanced therapy consisting of autologous CD34+-enriched hematopoietic stem and progenitor cells exposed to transduction with a lentiviral vector, driving myeloid-specific IFN2 expression. Genetically modified TEMs target IFN2 expression in the GBM tumor microenvironment.

In order to guarantee stable delivery of genetically engineered TEMs into the tumor, we transduce hematopoietic stem and progenitor cells with a lentiviral vector carrying the IFNa2 transgene transcriptionally regulated by the Tie2 promoter and by post transcriptional elements that guarantee that the transgene is expressed only in myeloid cells that are recruited into the tumor, Gentner said.

TEM-GBM is an open-label, dose-escalation study evaluating the safety and efficacy of Temferon in up to 21 newly diagnosed patients with GBM harboring an unmethylated MGMT promoter. Following surgical resection, up to 15 patients were assigned to 1 of 3 escalating doses of Temferon and 1 of 2 different conditioning regimens in part A of the trial. In Part B, 6 more patients will receive a single dose of Temferon at the recommended phase 2 dose.

Following completion of radiotherapy, patients received a conditioning regimen consisting of carmustine (BCNU) and thiotepa (Tepadina) in cohorts 1 to 4 or busulfan (Busulfex) and thiotepa in cohort 5 prior to administration of Temferon.

In-patient monitoring occurs until hematological recovery, then patients will undergo regular follow-up for up to 720 days. At that point, patients are invited to participate in a long term follow-up study for an additional 6 years.

Eligible adults aged 18 to 70 years must have an ECOG performance score of 0 to 1, a Karnofsky performance score greater than 70%, and adequate cardiac, renal, hepatic, and pulmonary function. Patients with active autoimmune disease or who have received any oral or parenteral chemotherapy or immunotherapy within 2 years of screening are excluded.

The primary end points of the study are Temferon engraftment over the first 90 days, proportion of patients achieving hematologic recovery 30 days after autologous stem cell transplantation, and short-term tolerability of Temferon as defined by stable blood counts, absence of cytopenias, absence of significant organ toxicities greater than grade 2, and absence of Replication Competent Lentivirus.

By the October 15, 2021, data cutoff, the median follow-up was 267 days (range: 60-749). Patients in cohorts 1 to 3 received a dose 0.5-2.0 x 106/kg Temferon with an average vector copy number of 0.70 and a transduction efficiency of 54%. Those in cohorts 4 and 5 received 2.0 x 106/kg Temferon with an average vector copy number of 0.77 and a transduction efficiency of 49%.

Investigators observed increasing proportions of Temferon-derived differentiated cells, as determined by the presence of vector genomes in the DNA of peripheral blood and bone marrow cells, reaching up to 30% at 1 month in the highest treatment cohort (2.0 x 106/kg). Those differentiated cells persisted at lower levels for up to 18 months.

All patients showed in vivo Temferon engraftment, Gentner said. Engraftment was highest at 1 month, and in many patients resembled pretty much the input fraction. Engraftment then decreased, stabilizing at 3 to 6 months around 10%.

Despite the significant proportion of engineered cells, only very low-medium concentrations of interferon alpha were detected in the plasma and in the cerebral spinal fluid, indicating a tight regulation of the vector expression.

Gentner added that Temferon did not delay hemopoietic recovery, and neutrophil and platelet engraftment were similar to standard autologous stem cell procedure.

Investigators did not detect any dose limiting toxicities. Gentner said that, so far, adverse events have been related to progression or the transplant procedure, not to the IFN2 itself.

Gentner B, Finocchiaro G, Farina F, et al. Genetically modified Tie-2 expressing monocytes target IFN-2 to the glioblastoma tumor microenvironment (TME): Preliminary data from the TEM-GBM Phase 1/2a study. Poster presented at: 2022 AACR Annual Meeting; April 8-13, 2022; New Orleans, LA. Abstract 5213.

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Evidence Shows Novel Temferon May Have Activity in Glioblastoma - Cancer Network

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April 9 (Reuters) - The first all-private team of astronauts ever launched to the International Space Station (ISS) were welcomed aboard the orbiting research platform on Saturday to begin a weeklong science mission hailed as a milestone in commercial spaceflight.

Their arrival came about 21 hours after the four-man team representing Houston-based startup company Axiom Space Inc lifted off on Friday from NASA's Kennedy Space Center, riding atop a SpaceX-launched Falcon 9 rocket.

The Crew Dragon capsule lofted into orbit by the rocket docked with the ISS at about 8:30 a.m. EDT (1230 GMT) on Saturday as the two space vehicles were flying roughly 250 miles (420 km) above the central Atlantic Ocean, a live webcast of the coupling from the National Aeronautics and Space Administration showed.

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The final approach was delayed for about 45 minutes by a technical glitch with a video feed used to monitor the capsule's rendezvous with the ISS, but it otherwise proceeded smoothly.

The multinational Axiom team, planning to spend eight days in orbit, was led by retired Spanish-born NASA astronaut Michael Lopez-Alegria, 63, the company's vice president for business development.

His second-in-command was Larry Connor, a real estate and technology entrepreneur and aerobatics aviator from Ohio designated as the mission pilot. Connor is in his 70s, but the company did not provide his precise age.

Rounding out the Ax-1 crew were investor-philanthropist and former Israeli fighter pilot Eytan Stibbe, 64, and Canadian businessman and philanthropist Mark Pathy, 52, both serving as mission specialists.

With docking achieved, it took nearly two hours for the sealed passageway between the space station and crew capsule to be pressurized and checked for leaks before hatches were opened to allow the newly arrived astronauts to come aboard the ISS.

The Ax-1 team was welcomed by all seven of the regular, government-paid crew members already occupying the space station: three American astronauts, a German astronaut from the European Space Agency and three Russian cosmonauts.

The NASA webcast showed the four smiling Axiom astronauts, dressed in navy blue flight suits, floating headfirst, one by one, through the portal into the space station, warmly greeted with hugs and handshakes by the ISS crew.

Lopez-Alegria later pinned astronaut wings onto the uniforms of the three spaceflight rookies of his Axiom team -- Connor, Stibbe and Pathy -- during a brief welcome ceremony.

Stibbe is now the second Israeli to fly to space, after Ilan Ramon, who perished with six NASA crewmates in the 2003 space shuttle Columbia disaster.

SCIENCE FOCUSED

The new arrivals brought with them two dozen science and biomedical experiments to conduct aboard ISS, including research on brain health, cardiac stem cells, cancer and aging, as well as a technology demonstration to produce optics using the surface tension of fluids in microgravity.

The mission, a collaboration among Axiom, Elon Musk's rocket company SpaceX and NASA, has been touted by all three as a major step in the expansion of space-based commercial activities collectively referred to by insiders as the low-Earth orbit economy, or "LEO economy" for short. read more

NASA officials say the trend will help the U.S. space agency focus more of its resources on big-science exploration, including its Artemis program to send humans back to the moon and ultimately to Mars.

While the space station has hosted civilian visitors from time to time, the Ax-1 mission marks the first all-commercial team of astronauts sent to ISS for its intended purpose as an orbiting research laboratory.

The Axiom mission also stands as SpaceX's sixth human spaceflight in nearly two years, following four NASA astronaut missions to the space station and the Inspiration 4 launch in September that sent an all-civilian crew into orbit for the first time. That flight did not dock with the ISS.

Axiom executives say their astronaut ventures and plans to build a private space station in Earth orbit go far beyond the astro-tourism services offered to wealthy thrill-seekers by such companies as Blue Origin and Virgin Galactic (SPCE.N), owned respectively by billionaire entrepreneurs Jeff Bezos and Richard Branson.

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Reporting by Steve Gorman in Los Angeles; Editing by Angus MacSwan, Daniel Wallis and Jonathan Oatis

Our Standards: The Thomson Reuters Trust Principles.

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Space station's first all-private astronaut team welcomed aboard orbiting platform - Reuters

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Courtesy of Pavlo Gonchar/SOPA Images/LightRocket via Getty Images

Drug pricing watchdog ICER, the Institute for Clinical and Economic Review, issued a draft report on bluebird bios gene therapy betibeglogene autotemcel for beta-thalassemia. Despite the proposed price tag of $2.1 million, ICERs not-yet-finalized report supports the therapys cost-effectiveness. This is good news for the recently beleaguered company.

Gene therapies are typically designed to cure a disease by replacing or fixing a damaged gene. Bluebirds therapy, which is listed under the brand name Zynteglo, had been approved in Europe and the UK, where its price is around $1.7 million (U.S.). However, the company pulled the therapy off the market in Europe over what it called a hostile pricing and reimbursement environment.

On April 5, bluebird bio announced it was beginning a comprehensive restructuring in hopes of cutting $160 million in costs over the next two years. It planned to re-focus on near-term catalysts, which include Zynteglo in the U.S., gene therapy for cerebral adrenoleukodystrophy (eli-cel) and a potential biologics license application (BLA) for lovotibeglogene autotemcel (lovo-cel) gene therapy for sickle cell disease. The BLA application is planned for 2023, while the U.S. regulatory decisions are expected this year. The PDUFA date for Zynteglo is Aug.19, 2022, and Sept. 16, 2022, for eli-cel.

As part of the restructuring, the company is cutting its workforce by about 30%.

ICER recommendations arent binding, but they have influence. If ICER says a drug is overpriced, it provides ammunition for payers, such as Medicare and insurers, to push back against proposed prices.

Gene therapies are very expensive. For example,Novartis Zolgensma, the one-time gene therapy onasemnogene abeparvovec for spinal muscular atrophy (SMA), is generally viewed as the most expensive drug with a price tag of $2.1 million. On the other hand, as an apparent cure for a disease that kills children by the age of two, it is very rare. The argument for these therapies, aside from their curative potential for otherwise incurable diseases, is that over the life of the patient, they are cost-effective.

Novartis and Spark Therapeuticss gene therapy Luxturna (voretigene neparvovec) runs about $850,000 per patient in the U.S. The therapy is for inherited retinal dystrophy with RPE65 mutations. It is typically diagnosed in childhood and eventually causes almost total blindness, and the therapy is essentially a cure.

Beta thalassemia is a genetic disease that impairs the ability of red blood cells to manufacture hemoglobin, the molecule in the body that carries oxygen. There are about 40,000 newly diagnosed cases in children each year around the world. People with the most severe form of it develop life-threatening anemia around four to six months of age and have to receive monthly blood transfusions and other treatments, such as iron-chelating drugs. The only other potential cure is hematopoietic stem cell transplantation (HSCT) but requires a donor with a matching human leukocyte antigen (HLA) profile within the appropriate age range.

Bluebirds Zynteglo appears to be another option for a cure, although how long the therapys effects last is something of an open question. The ICER report noted the uncertainties, but concluded that the evidence suggests that beti-cel provides net health benefits to patients with TDT.

The ICER report indicated, per Managed Healthcare Executive, that "patients could be treated without reaching the potential budget impact threshold at three prices (about $1.85 million, $2.11 million and $2.38 million per course of treatment). This analysis was done at several prices to document the percentage of patients who could be treated without crossing a potential budget impact threshold that is aligned with the overall growth in the U.S. economy.

In Phase III trials, 89% of patients who received the therapy became transfusion independent, and in Phase I/II and III trials, those patients remained transfusion-free for at least 42 months. In general, side effects were mild and no deaths were reported. In December 2021, bluebird presented data at the American Society of Hematology meeting from a long-term study (LTF-303) that showed adult and pediatric patients with beta-thalassemia who required regular red blood cell transfusions can produce normal or near-normal levels of total hemoglobin and remain transfusion-free with stable iron markers up to seven years after receiving beti-cel.

A 2017 study published in Blood found that on average, beta-thalassemia patients required 17 transfusions per year, 23 days apart. Mean total healthcare costs for the patients were $128,062, plus or minus $62,260 per year. Total costs were primarily driven by chelation and transfusion costs.

Although the severity of the disease varies, a 2009 study found that people with beta-thalassemia major often die from cardiac complications of iron overload by 30 years of age," making bluebird's new therapy, if it is successful, vital for these patients.

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Drug Price Watchdog Calls Bluebird Bio's $2.1 Million Gene Therapy Cost-Effective - BioSpace

Cardiac Stem Cells Comments Off on Drug Price Watchdog Calls Bluebird Bio’s $2.1 Million Gene Therapy Cost-Effective – BioSpace | April 15th, 2022

The addition of the innate cell engager AMF13 to preactivated and expanded natural killer (NK) cells may represent an effective treatment for pretreated patients with advanced lymphoma, according to findings from a phase 1/2 study (NCT04074746) that were presented during the 2022 AACR Annual Meeting. 1

Results showed that patients experienced a median overall response rate (ORR) of 89.5% (n = 17/19). Overall, 10 patients experienced complete responses (CRs) and 7 experienced partial responses (PRs).2

Lead author Yago Nieto, MD, PhD, a professor of medicine in the Department of Stem Cell Transplantation and Cellular Therapy at the University of Texas MD Anderson Cancer Center, in Houston, discussed the findings during a press conference during the meeting. He said the study team was pleasantly surprised by the quality of tumor responses in patients with resistant lymphomas.

This is the first clinical trial using off the shelf cord blood-derived cytokine-induced memory-likeex vivoexpanded NK cells precomplexed with the innate cell engager AMF13 construct to treat patients with CD30-positive relapsed/refractory Hodgkin lymphoma, he said. We saw very encouraging activity in this population of very heavily pretreated patients.

The current standard of care for relapsed CD30-positive lymphomas is brentuximab vedotin (Adcetris), an antibody-drug conjugate that delivers a toxic cytoskeleton destabilizing agent to cells expressing CD30. However, not all these lymphomas respond to brentuximab vedotin. When that treatment fails, those tumors then become extremely resistant to killing and patients are left with very few effective therapeutic options.

To address the problem, investigators enrolled 22 patients with relapsed or refractory CD30+ lymphoma into this single-center phase 1/2 trial, 20 of whom were diagnosed with Hodgkin lymphoma (HL). All had active progressive disease at enrollment, and none received bridging therapy. Patients were heavily pretreated, with a median of 7 (range, 1-14) prior lines of therapy. Nine underwent autologous stem cell transplantation (SCT) and 5 received allogeneic SCT.

Eligible patients had relapsed/refractory CD30-positive classical HL, B-cell non-Hodgkin lymphoma, anaplastic large-cell lymphoma, or peripheral T-cell lymphoma that was refractory or intolerant to brentuximab vedotin. They needed to have an ECOG performance status of 2 or below, and adequate renal, hepatic, pulmonary, and cardiac function.

The median age was 40 years (range, 20-75). Most patients were white (68.2%) and male 68.1%).

Patients receive 2 cycles of fludarabine/cyclophosphamide, followed by AFM13-CB NK cells at 3 dose levelsDL1 (106NK/gg), DL2 (107NK/kg), and DL3 (108NK/kg)on day 0 plus 3 weekly intravenous infusions of 200 mg AFM13, a CD30/CD16A bispecific antibody. Nineteen patients completed both planned cycles of treatment.

Nieto and colleagues isolated NK cells from cord blood, then used a mixture of cytokines to activate the cells into a memory-like state, making them more persistent and effective. They then expanded the cells in culture and complexed them with AFM13.

At a median follow-up of 11 months, progression-free survival (PFS) and overall survival (OS) rates across all 3 dose levels were 52% and 81%, respectively. Across all dose levels, 53% of patients experienced CR and 37% had PR. Eleven percent had progressive disease.

Expansion of NK cells occurred immediately after infusion and persisted for 3 weeks.

Investigators established DL3 as the recommend phase 2 dose (RP2D). All 13 (100%) patients treated at this dose level responded to therapy, including eight CRs (62%).Five of those patients were in CR after cycle 1, and 3 additional patients converted from PR to CR after cycle 2, Nieto added.

The median PFS was 67% and the median OS was 93% in the RP2D population.

Investigators did not record any cytokine release syndrome or graft vs host disease (GVHD), or neurotoxicity. Our preliminary results show an excellent tolerability profile, Nieto said.

There was no instance of infusion-related reactions (IRRs) associated with AFM13-NK cells across 40 infusions. There was 1 instance of grade 3 IRR and 4 grade 2 IRRs in 108 infusions of AFM13 alone. Investigators observed no dose limiting toxicities.

Never before in mankind have we seen this approach, really leading to pretty staggering results, Timothy Yap, MBBS, PhD, FRCP, a medical oncologist and associate director of translational research in the Institute for Personalized Cancer Therapy at the University of Texas MD Anderson Cancer Center, said. Everyone can see for themselves how impressive these results are. In addition to that, the actual tolerability profile is truly excellent with no instances of cytokine release syndrome, no neurotoxicity, no GVHD. Truly, truly impressive.

References

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Adding Bispecific Antibody to Natural Killer Cells May Be Effective in Heavily Pretreated Lymphoma - http://www.oncnursingnews.com/

Cardiac Stem Cells Comments Off on Adding Bispecific Antibody to Natural Killer Cells May Be Effective in Heavily Pretreated Lymphoma – www.oncnursingnews.com/ | April 15th, 2022

The use of human bone marrow mesenchymal stem cells (hBMSCs) to regenerate and repair bone tissue defects is a complex research field of bone tissue engineering; nevertheless, it is a hot topic. One of the biggest problems is the limited survival and osteogenic capacity of the transplanted cells within the host tissue. Even for hBMSCs with their low immunogenicity, the body will still cause a local immune-inflammatory response directed against the allogeneic cells and thereby reduce the activity of the transplanted cells. Even in the case of successful transplantation, the lack of vascularization at the transplantation site makes it difficult for the transplanted cells to exchange nutrients and metabolic wastes that ultimately affects bone regeneration. In this study, we covalently modified alginate with RGD and QK peptides that were injected subcutaneously into immunocompetent mice. Histological analysis, as well as ELISA techniques, proved that this method is able to provide bioactive stem cell transplant beds containing functionalized biomaterials and vascularized surrounding tissues. Inflammation-related factors, such as IL-2, IL-6, TNF-, and IFN-, around the cell graft beds decreased with time and were lowest at the second week. Then, the hBMSCs were injected into the cell transplantation beds intended to form vascularized bonelike tissues that were evaluated by micro-computed tomography (Micro CT), histological, and immunohistochemical analyses. The results showed that the expression of osteogenesis-related proteins RUNX2, COL1A1, and OPN, as well as the expression of angiogenic factor vWF and cartilage-related protein COL2A1 were significantly upregulated in the hBMSC-derived osteogenic tissue. These results suggest that the stem cell transplantation strategy by constructing bioactive cell transplant beds is effective to enhance the bone regeneration capacity of hBMSCs and holds great potential in bone tissue engineering.

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Strategy of Stem Cell Transplantation for Bone Regeneration with Functionalized Biomaterials and Vascularized Tissues in Immunocompetent Mice -...

Bone Marrow Stem Cells Comments Off on Strategy of Stem Cell Transplantation for Bone Regeneration with Functionalized Biomaterials and Vascularized Tissues in Immunocompetent Mice -… | April 3rd, 2022

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A breakthrough made by Australian researchers might change the way doctors treat a broken or missing bone. Turns out stem cells can turn into bone if certain conditions are met.

Normally, bone can be made only of mesenchymal stem cells (MSCs) which are biologically found in the bone marrow.

Extracting them from there is a difficult and painful procedure while doing so at scale is beyond tricky.

But this could change any moment now after Australian researchers found that stem cells can be converted into bone when a certain type of sound waves are used.

Tests had previously shown that low frequency vibrations were great at inducing cell differentiation but the process took over a week and the results were mixed at best.

Nobody had bothered to look into high frequency sound waves until now. RMIT researchers took a microchip capable of dispersing sound waves in the Mhz range and turned it at MSCs in silicon oil on a culture plate.

The team noticed that after exposing the cells to 10MhZ signals for 10 minutes daily for five days, the markers indicating the bone conversion appeared.

We can use the sound waves to apply just the right amount of pressure in the right places to the stem cells, to trigger the change process, said Leslie Yeo, co-lead researcher on the study. Our device is cheap and simple to use, so could easily be upscaled for treating large numbers of cells simultaneously vital for effective tissue engineering.

This discovery, detailed in the journalSmall, eliminates the need of drugs to make stem cells behave this way. Moreover, the MSCs can be pulled from a variety of places, like fast tissue, not just bone marrow.

By injecting them into the body in case of an injury or disease, they can start working on a new bone faster and more efficient.

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Stem Cells Turn Into Bone When Sound Waves Are Near - TechTheLead

Bone Marrow Stem Cells Comments Off on Stem Cells Turn Into Bone When Sound Waves Are Near – TechTheLead | April 3rd, 2022

Jasper Therapeutics

REDWOOD CITY, Calif., March 31, 2022 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (NASDAQ: JSPR), a biotechnology company focused on hematopoietic cell transplant therapies, today announced that updated data from the Companys ongoing study of JSP191 as single agent conditioning prior to allogeneic hematopoietic stem cell (HSC) re-transplant in patients with severe combined immunodeficiency (SCID) has been accepted for presentation as a late-breaking poster at the 2022 Clinical Immunology Society (CIS) Annual Meeting, to be held in Charlotte, North Carolina from March 31 to April 3, 2022.

Title: Update: Single-Agent Conditioning with Anti-CD117 Antibody JSP191 Shows Donor Engraftment, Nave Lymphocyte Production, and Clinical Benefit in Patients with Severe Combined Immunodeficiency (SCID)Date and Time: Friday, April 1, 2022, 1:00-2:00 p.m. ET

This updated data indicates that JSP191 at 0.6mg/kg can deplete blood stem cells, leading to long-term donor cell engraftment, immune reconstitution which positively affects the clinical status of SCID patients who suffer from poor T cell and negligible B cell immunity because they failed their first transplant, said Wendy Pang, MD, Ph.D., Senior Vice President of Research and Translational Medicine of Jasper Therapeutics. This population of SCID patients is largely without treatment options and rely on supportive therapies like life long IVIG to provide some level of immune protection. JSP191 based conditioning may provide these patients with the best chance of a safe and successful transplant and reconstituted immune system.

CIS attendees are the primary caregivers for the immune deficient patient population, we are pleased to be able to present this data at the 2022 CIS annual meeting, Ronald Martell, CEO of Jasper. We believe that with our successful clinical efforts, we are one step closer, and uniquely positioned to deliver a targeted non-genotoxic conditioning agent to patients with SCID.

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About JSP191

JSP191 is a humanized monoclonal antibody in clinical development as a conditioning agent that blocks stem cell factor receptor signaling leading to clearance of hematopoietic stem cells from bone marrow, creating an empty space for donor or genetically modified transplanted stem cells to engraft. To date, JSP191 has been evaluated in more than 100 healthy volunteers and patients. Three clinical trials for myelodysplastic syndromes (MDS)/acute myeloid leukemia (AML), severe combined immunodeficiency (SCID) and Fanconi anemia are currently enrolling. The Company plans a new study of JSP191 as a second-line therapeutic in lower risk MDS patients in 2022 as well as to a pivotal study in MDS/AML transplant in early 2023. Enrollment in additional studies are planned in patients with sickle cell disease, chronic granulomatous disease and GATA2 MDS who are undergoing hematopoietic cell transplantation.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The company is advancing two potentially groundbreaking programs. JSP191, an anti-CD117 monoclonal antibody, is in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow in patients undergoing hematopoietic cell transplantation. It is designed to enable safer and more effective curative allogeneic hematopoietic cell transplants and gene therapies. In parallel, Jasper Therapeutics is advancing its preclinical mRNA engineered hematopoietic stem cell (eHSC) platform, which is designed to overcome key limitations of allogeneic and autologous gene-edited stem cell grafts. Both innovative programs have the potential to transform the field and expand hematopoietic stem cell therapy cures to a greater number of patients with life-threatening cancers, genetic diseases and autoimmune diseases than is possible today. For more information, please visit us at jaspertherapeutics.com.

Forward-Looking Statements

Certain statements included in this press release that are not historical facts are forward-looking statements for purposes of the safe harbor provisions under the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements are sometimes accompanied by words such as believe, may, will, estimate, continue, anticipate, intend, expect, should, would, plan, predict, potential, seem, seek, future, outlook and similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These forward-looking statements include, but are not limited to, statements regarding the potential long-term benefits of hematopoietic stem cells (HSC) engraftment following targeted single-agent JSP191 conditioning in the treatment of severe combined immunodeficiency (SCID) and Jaspers ability to potentially deliver a targeted non-genotoxic conditioning agent to patients with SCID. These statements are based on various assumptions, whether or not identified in this press release, and on the current expectations of Jasper and are not predictions of actual performance. These forward-looking statements are provided for illustrative purposes only and are not intended to serve as, and must not be relied on by an investor as, a guarantee, an assurance, a prediction or a definitive statement of fact or probability. Actual events and circumstances are difficult or impossible to predict and will differ from assumptions. Many actual events and circumstances are beyond the control of Jasper. These forward-looking statements are subject to a number of risks and uncertainties, including general economic, political and business conditions; the risk that the potential product candidates that Jasper develops may not progress through clinical development or receive required regulatory approvals within expected timelines or at all; risks relating to uncertainty regarding the regulatory pathway for Jaspers product candidates; the risk that clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release; the risk that Jasper will be unable to successfully market or gain market acceptance of its product candidates; the risk that Jaspers product candidates may not be beneficial to patients or successfully commercialized; patients willingness to try new therapies and the willingness of physicians to prescribe these therapies; the effects of competition on Jaspers business; the risk that third parties on which Jasper depends for laboratory, clinical development, manufacturing and other critical services will fail to perform satisfactorily; the risk that Jaspers business, operations, clinical development plans and timelines, and supply chain could be adversely affected by the effects of health epidemics, including the ongoing COVID-19 pandemic; the risk that Jasper will be unable to obtain and maintain sufficient intellectual property protection for its investigational products or will infringe the intellectual property protection of others; and other risks and uncertainties indicated from time to time in Jaspers filings with the SEC. If any of these risks materialize or Jaspers assumptions prove incorrect, actual results could differ materially from the results implied by these forward-looking statements. While Jasper may elect to update these forward-looking statements at some point in the future, Jasper specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Jaspers assessments of any date subsequent to the date of this press release. Accordingly, undue reliance should not be placed upon the forward-looking statements.

Contacts:John Mullaly (investors)LifeSci Advisors617-429-3548jmullaly@lifesciadvisors.com

Jeet Mahal (investors)Jasper Therapeutics650-549-1403jmahal@jaspertherapeutics.com

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Jasper Therapeutics to Present Updated Data on JSP191 Conditioning in SCID Patients at the 2022 Clinical Immunology Society Annual Meeting - Yahoo...

Bone Marrow Stem Cells Comments Off on Jasper Therapeutics to Present Updated Data on JSP191 Conditioning in SCID Patients at the 2022 Clinical Immunology Society Annual Meeting – Yahoo… | April 3rd, 2022

This article was originally published here

Bioengineered. 2022 Apr;13(4):8382-8395. doi: 10.1080/21655979.2022.2036891.

ABSTRACT

The exosomes (Exo) had always been considered as transport vectors for microRNA (miRNA). An increasing number of data had clarified the influence of Exo on the cell progression of non-small cell lung cancer (NSCLC). Nevertheless, its specific mechanism had not yet been verified. This work was to explore the potential mechanism of Exo-derived miR-631 targeting and regulating E2F family of transcription factor 2 (E2F2) to repress the malignant behavior of NSCLC cells. Test of microRNA (miR)-631 and E2F2 in NSCLC was performed. BMSCs-Exo that altered miR-631 was co-cultured with NSCLC cells. Detection of the cloning and progression of NSCLC cells was performed. Testification of the targeting of miR-631 with E2F2 was conducted. In vivo experiments were performed to verify the results in vitro. In short, elevation of miR-631 Exo repressed the advancement and phosphatidylinositol 3-kinase/Akt activation of NSCLC cells, while silence of miR-631 was in the opposite. In terms of mechanism, miR-631 exerted the influence via targeting E2F2. The coincident results were obtained in animal models. In brief, BMSC-Exo mediated E2F2 via delivering miR-631 to NSCLC cells to modulate the malignant behavior of NSCLC.

PMID:35353027 | DOI:10.1080/21655979.2022.2036891

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MicroRNA-631 deriving from bone marrow mesenchymal stem cell exosomes facilitates the malignant behavior of non-small cell lung cancer via modulating...

Bone Marrow Stem Cells Comments Off on MicroRNA-631 deriving from bone marrow mesenchymal stem cell exosomes facilitates the malignant behavior of non-small cell lung cancer via modulating… | April 3rd, 2022

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The market elements are analyzed inside and out in the outline segment. This part is an unquestionable requirement perused for anybody settling on information driven choices. It talks about how Rheumatoid Arthritis Stem Cell Therapy functions, as well as market size and volume. The report is coordinated in straightforward organizations and incorporates outlines, tables, and charts to inspect the information and uncover the secret example in the numbers. Besides, the report incorporates verifiable deals and income data as well as guage designs for the following not many years.

The development and limiting elements are given their own fragment to help our clients in observing the Rheumatoid Arthritis Stem Cell Therapy touch spots and problem areas. The ends attracted this part depend on trustworthy and more significant position sources. Our specialists have utilized an assortment of market projection ways to deal with furnish our clients with reliable outcomes.

The Rheumatoid Arthritis Stem Cell Therapy is isolated into different groupings in the division segment. The fragment is an inside and out assessment of every classification, which is grouped by its qualities and expansiveness. Weve recorded every one of the measurements along with subjective clarifications to assist clients with appreciating the expected broadness of each class before very long. To dispose of errors in current realities and discoveries, the report utilizes an assortment of measurable methodologies. Moreover, an assortment of pattern projection approaches are utilized to uncover future development angles and prospects.

By Product Type (Allogeneic Mesenchymal Stem Cells, Bone Marrow Transplant and Adipose Tissue Stem Cells)

By End-User (Hospitals, Ambulatory Surgical Centers and Specialty Clinics)

By Region (North America, Europe, Asia Pacific, Latin America, and Middle East & Africa)

Mesoblast Ltd., Roslin Cells, Regeneus Ltd, ReNeuron Group plc, International Stem Cell Corporation, TiGenix and others

Promising Regions & Countries Mentioned In The Rheumatoid Arthritis Stem Cell Therapy Report:

The local review area inspects all potential market scenes in specific areas before very long. Its an exhaustive assessment of the Rheumatoid Arthritis Stem Cell Therapy possible districts. The examination additionally remembers a contextual investigation for significant market members to help shoppers distinguish and understand powerful techniques in the overall Rheumatoid Arthritis Stem Cell Therapy , as well as likely boundaries. Our master experts checked the data and endeavored to protect the most ideal degree of exactness.

Segmentation Overview:

By Product Type (Allogeneic Mesenchymal Stem Cells, Bone Marrow Transplant and Adipose Tissue Stem Cells)

By End-User (Hospitals, Ambulatory Surgical Centers and Specialty Clinics)

By Region (North America, Europe, Asia Pacific, Latin America, and Middle East & Africa)

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openings both developed and arising regions.

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Rheumatoid Arthritis Stem Cell Therapy Market Assessment, With Major Top Companies Analysis, Geographic Analysis, Growing Opportunities Data By...

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Abstract:

Background:

Objective:To observe the effect of H2O2 induced oxidative stress on autophagy and apoptosis of human bone marrow mesenchymal stem cells (hBMSCs).

Method: The hBMSCs were separated and cultured by density gradient centrifugation combined with adherence method. They were divided into blank group (with medium only), 3-MA (autophagy inhibitor) pretreatment group (with 2 ml of 5 mM 3-MA medium), H2O2 Intervention group (add 2ml medium containing 0.05mM H2O2), H2O2+3-MA treatment group (add 2ml medium containing 5mM 3-MA, then add 2ml medium containing 0.05mM H2O2). DCFH-DA staining was used to detect cellular reactive oxygen species (ROS) levels,and CCK-8 analysis was used to detect the effects of different concentrations (0,50,100,200,400mol/L) of H2O2 on the proliferation of hBMSCs; Monodansylcadaverine(MDC) Fluorescent amine probe staining, Lysosome Red Fluorescent Probe (Lyso-Tracker Red) staining to observe the level of autophagy; Immunofluorescence staining to detect the expression of LC3A/B; Flow cytometry (Annexin V/PI) to detect cell apoptosis Circumstances; Protein chip detection of autophagy-related proteins; Western blot detection of Beclin1, mTOR, p-mTOR, LC3A/B, and Cleaved caspase-3 protein expression.

Result: After treating hBMSCs with different concentrations of H2O2 (0,50,100,200,400mol) for 24h ,48h, and 72h, with the increase of H2O2 concentration, the cell proliferation ability decreased; while with the extension of time, the cell proliferation ability increased not significantly; 50mol cell proliferation ability is the strongest. Compared with the blank group and 3-MA group, the H2O2 intervention group increased the level of intracellular ROS, increased autophagosomes, and significantly decreased the apoptosis rate; up-regulated Beclin1, mTOR, LC3A/B and Cleaved caspase-3 protein expression, and down-regulated p-mTOR Protein expression level. Compared with the autophagy inhibitor 3-MA group, the H2O2+3-MA group increased the level of intracellular ROS, increased autophagosomes, and did not significantly increase the apoptosis rate; up-regulated the protein expression of Beclin1, mTOR, LC3A/B and Cleaved caspase-3 Down-regulate the expression of p-mTOR protein.

Conclusion: H2O2 can induce hMSCs to produce oxidative stress response. Under oxidative stress conditions, hMSCs can promote protective autophagy and reduce cell apoptosis or the level of apoptosis caused by excessive autophagy.

See more here:
Effect of oxidative stress-induced autophagy on proliferation and apoptosis of hMSCs - Newswise

Bone Marrow Stem Cells Comments Off on Effect of oxidative stress-induced autophagy on proliferation and apoptosis of hMSCs – Newswise | April 3rd, 2022

The latest study of the Personalized Cell Therapy MarketStatistics2022Report providesan elaborative analysis of the market size, industry share, growth, development, and competitive landscape. The report also provides a comprehensive analysis of the sales volume, revenue, gross margin, and price growth in the Personalized Cell TherapyMarket. Many key points covered in the report, include recent development in the global market, such as mergers and acquisitions, SWOT analysis, competitive landscape, industry trends, and company profiles.

Leading Key Players Covered in the GlobalPersonalized Cell Therapy Market Research Report:

Novartis AG, Vericel Corporation, Bellicum Pharmaceuticals, MolMed SpA, Cytori Therapeutics Inc, Gilead Sciences, Inc, Celgene Corporation, Bluebird Bio, Aurora Biopharma Inc, Saneron CCEL TherapeuticsInc, Kuur Therapeutics, MediGene AG, Sangamo Therapeutics

Get a Sample PDF of the Report @ https://www.alexareports.com/report-sample/2856089

Market Segment by Types:

By Cell Type, Hematopoietic Stem Cell, Skeletal Muscle Stem Cell/Mesenchymal Stem Cells/Lymphocytes, By Technique, Platelet Transfusions/Bone Marrow Transplantation/Packed Red Cell Transfusions/Organ Transplantation

Market Segment by Applications:

Cardiovascular Diseases, Neurological Disorders, Inflammatory Diseases, Diabetes, Cancer

Market Segment by Regions:

Table of Contents

Section 1 Personalized Cell Therapy Market Overview

Section 2 Global Personalized Cell Therapy Market Key Players Share

Section 3 Key PlayersPersonalized Cell Therapy Business Introduction

Section 4 Global Personalized Cell Therapy Market Segmentation (By Region)

Section 5 Global Personalized Cell Therapy Market Segmentation (by Product Type)

Section 6 Global Personalized Cell Therapy Market Segmentation (by Application)

Section 7 Global Personalized Cell Therapy Market Segmentation (by Channel)

Section 8 Personalized Cell Therapy Market Forecast 2021-2026

Section 9 Personalized Cell Therapy Application and Client Analysis

Section 10 Personalized Cell Therapy Manufacturing Cost of Analysis

Section 11 Conclusion

Section 12 Methodology and Data Source

If any customization or requirements in the research study, please let us know Alexa Reportsoffer the report as you want.

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Personalized Cell Therapy Market Size by Applications, Company Profiles, Product Types, Revenue and Forecast to 2026 ChattTenn Sports - ChattTenn...

Bone Marrow Stem Cells Comments Off on Personalized Cell Therapy Market Size by Applications, Company Profiles, Product Types, Revenue and Forecast to 2026 ChattTenn Sports – ChattTenn… | April 3rd, 2022

The SARS-CoV-2 virus can infect specialized pacemaker cells that maintain the hearts rhythmic beat, setting off a self-destruction process within the cells, according to a preclinical study co-led by researchers at Weill Cornell Medicine, NewYork-Presbyterian and NYU Grossman School of Medicine. The findings offer a possible explanation for the heart arrhythmias that are commonly observed in patients with SARS-CoV-2 infection.

In the study, reported March 8 in Circulation Research, the researchers used an animal model as well as human stem cell-derived pacemaker cells to show that SARS-CoV-2 can readily infect pacemaker cells and trigger a process called ferroptosis, in which the cells self-destruct but also produce reactive oxygen molecules that can impact nearby cells.

This is a surprising and apparently unique vulnerability of these cellswe looked at a variety of other human cell types that can be infected by SARS-CoV-2, including even heart muscle cells, but found signs of ferroptosis only in the pacemaker cells, said study co-senior author Dr. Shuibing Chen, the Kilts Family Professor of Surgery and a professor of chemical biology in surgery and of chemical biology in biochemistry at Weill Cornell Medicine.

Arrhythmias including too-quick (tachycardia) and too-slow (bradycardia) heart rhythms have been noted among many COVID-19 patients, and multiple studies have linked these abnormal rhythms to worse COVID-19 outcomes. How SARS-CoV-2 infection could cause such arrhythmias has been unclear, though.

In the new study, the researchers, including co-senior author Dr. Benjamin tenOever of NYU Grossman School of Medicine, examined golden hamstersone of the only lab animals that reliably develops COVID-19-like signs from SARS-CoV-2 infectionand found evidence that following nasal exposure the virus can infect the cells of the natural cardiac pacemaker unit, known as the sinoatrial node.

To study SARS-CoV-2s effects on pacemaker cells in more detail and with human cells, the researchers used advanced stem cell techniques to induce human embryonic stem cells to mature into cells closely resembling sinoatrial node cells. They showed that these induced human pacemaker cells express the receptor ACE2 and other factors SARS-CoV-2 uses to get into cells and are readily infected by SARS-CoV-2. The researchers also observed large increases in inflammatory immune gene activity in the infected cells.

The teams most surprising finding, however, was that the pacemaker cells, in response to the stress of infection, showed clear signs of a cellular self-destruct process called ferroptosis, which involves accumulation of iron and the runaway production of cell-destroying reactive oxygen molecules. The scientists were able to reverse these signs in the cells using compounds that are known to bind iron and inhibit ferroptosis.

This finding suggests that some of the cardiac arrhythmias detected in COVID-19 patients could be caused by ferroptosis damage to the sinoatrial node, said co-senior author Dr. Robert Schwartz, an associate professor of medicine in the Division of Gastroenterology and Hepatology at Weill Cornell Medicine and a hepatologist at NewYork-Presbyterian/Weill Cornell Medical Center.

Although in principle COVID-19 patients could be treated with ferroptosis inhibitors specifically to protect sinoatrial node cells, antiviral drugs that block the effects of SARS-CoV-2 infection in all cell types would be preferable, the researchers said.

The researchers plan to continue to use their cell and animal models to investigate sinoatrial node damage in COVID-19and beyond.

There are other human sinoatrial arrhythmia syndromes we could model with our platform, said co-senior author Dr. Todd Evans, the Peter I. Pressman M.D. Professor of Surgery and associate dean for research at Weill Cornell Medicine. And, although physicians currently can use an artificial electronic pacemaker to replace the function of a damaged sinoatrial node, theres the potential here to use sinoatrial cells such as weve developed as an alternative, cell-based pacemaker therapy.

Many Weill Cornell Medicine physicians and scientists maintain relationships and collaborate with external organizations to foster scientific innovation and provide expert guidance. The institution makes these disclosurespublic to ensure transparency. For this information, see profiles for Dr. Todd Evans, and Dr. Robert Schwartz.

Originally posted here:
Are COVID-19-Linked Arrhythmias Caused by Viral Damage to the Heart's Pacemaker Cells? - Weill Cornell Medicine Newsroom

Cardiac Stem Cells Comments Off on Are COVID-19-Linked Arrhythmias Caused by Viral Damage to the Heart’s Pacemaker Cells? – Weill Cornell Medicine Newsroom | April 3rd, 2022

Evidence Rating Level:1 (Excellent)

Study Rundown:Duchenne muscular dystrophy (DMD) is an X-linked genetic disorder characterized by progressive muscle degeneration leading to significant reduction in life expectancy. Males with DMD have an estimated life expectancy of 22 years with heart and respiratory muscles affected in later disease stages. In this phase 2 trial, a formulation of allogenic cardiosphere-derived cells (CAP-1002) was evaluated against placebo in patients with DMD. CAP-1002 is, in essence, a concentrate of cardiac stem cells with potential disease-modifying properties such as regenerative abilities. Participants (n=20) were randomized 1:1 to receive either CAP-1002 or placebo every three months for four total infusions. Primary outcome was upper limb function measured by a scale of 0-6 (PUL). CAP-1002 was shown to slow PUL decline by 71% compared to placebo or by an absolute difference of 2.6 points. CAP-1002 was generally well-tolerated with only one severe adverse hypersensitivity reaction leading to withdrawal from the trial. Limitations of this study include the small sample size. Nonetheless, this study provides promising preliminary results for a potential disease-modifying therapy in DMD.

Click to read the study in the Lancet

Relevant Reading:Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study.

In-Depth [randomized controlled trial]:HOPE-2 was a randomized-controlled phase 2 clinical trial to assess to safety and efficacy of intravenous CAP-1002 for the treatment of Duchenne muscular dystrophy (DMD). The study enrolled patients aged 10 and older with genetically confirmed DMD. Participants had to score between 2-5 on the Performance of Upper Limb (PUL) scale with 0 being no useful function of hands and 6 being maximum overhead reach without compensation. 20 participants were assigned 1:1 to either CAP-1002 (n=8) or placebo (n=12) infusion every 3 months for a total of four infusions. Mean age of the enrolled male participants was 14 in both groups. Primary outcome was the upper limb function on the PUL scale. Patients who received CAP-1002 had a greater change in PUL score from baseline after 12 months compared to placebo (percentile difference 36.2, 95% CI 12.7-59.7). On the PUL scale, the placebo group had a mean change of -3.4 points from baseline, while the CAP-1002 had a -0.8 point change (difference of 2.6 points). This can also be interpreted as a 71% slowing of loss of function in the CAP-1002 group. Three patients in the CAP-1002 group had infusion-related hypersensitivity reactions, one leading to discontinuation. No other adverse events were seen in the two groups.

2022 2 Minute Medicine, Inc. All rights reserved. No works may be reproduced without expressed written consent from 2 Minute Medicine, Inc. Inquire about licensing here. No article should be construed as medical advice and is not intended as such by the authors or by 2 Minute Medicine, Inc.

Originally posted here:
#VisualAbstract: Cardiosphere-derived cell therapy slows disease progression in Duchenne muscular dystrophy - Physician's Weekly

Cardiac Stem Cells Comments Off on #VisualAbstract: Cardiosphere-derived cell therapy slows disease progression in Duchenne muscular dystrophy – Physician’s Weekly | April 3rd, 2022

The following is management's discussion and analysis ("MD&A") of certainsignificant factors that have affected our financial position and operatingresults during the periods included in the accompanying financial statements, aswell as information relating to the plans of our current management. This reportincludes forward-looking statements. Generally, the words "believes,""anticipates," "may," "will," "should," "expect," "intend," "estimate,""continue," and similar expressions or the negative thereof or comparableterminology are intended to identify forward-looking statements. Such statementsare subject to certain risks and uncertainties, including the matters set forthin this report or other reports or documents we file with the Securities andExchange Commission from time to time, which could cause actual results oroutcomes to differ materially from those projected. Undue reliance should not beplaced on these forward-looking statements which speak only as of the datehereof. We undertake no obligation to update these forward-looking statements.

The following discussion and analysis should be read in conjunction with ourfinancial statements and the related notes thereto and other financialinformation contained elsewhere in this Form 10-K

Our Ability To Continue as a Going Concern

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Index

Biotechnology Product Candidates

GENERAL AMERICAN CAPITAL PARTNERS

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Index

Results of Operations Overview

Comparison of Years Ended December 31, 2021 and December 31, 2020

Cost of sales consists of the costs associated with the production of MyoCathand test kits, product costs, labor for production and training and lab andbanking costs consistent with products and services provided.

Cost of sales was $52,030 in the year ended December 31, 2021 compared to$64,117 in the year ended December 31, 2020. The decrease is due to the decreasein revenues.

Research and development expenses were $0 in 2021 remaining the same as $0 in2020.

Selling, General and Administrative

Gain (loss) on settlement of debt

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In valuing our common stock, our Board of Directors considered a number offactors, including, but not limited to:

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Index

Options outstanding at December 31, 2021 110,643,884 $ 0.0247

Options exercisable at December 31, 2021 93,491,384 $ 0.0256

Available for grant at December 31, 2021 34,168,070

Average Number Weighted Average

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Index

Our primary sources of revenue are from the sale of test kits and equipment,training services, patient treatments, laboratory services and cell banking.

Patient treatments and laboratory services revenue are recognized when thoseservices have been completed or satisfied.

Research and Development Costs

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Depreciation is computed using the straight-line method over the assets'expected useful lives or the term of the lease, for assets under capital leases.

Cash and cash equivalents include cash on hand, deposits in banks withmaturities of three months or less, and all highly liquid investments which areunrestricted as to withdrawal or use, and which have original maturities ofthree months or less.

We allocate the proceeds received from equity financing and the attached optionsand warrants issued, based on their relative fair values, at the time ofissuance. The amount allocated to the options and warrants is recorded asadditional paid in capital.

Selling, General and Administrative

Our opinion is that inflation has not had, and is not expected to have, amaterial effect on our operations.

Liquidity and Capital Resources

In 2021, we continued to finance our operational cash needs with cash generatedfrom financing activities.

Economic Injury Disaster Loan (EIDL)

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Net cash provided by investing activities was $0 for the year ended December 31,2021.

Existing Capital Resources and Future Capital Requirements

As of December 31, 2021, we had $8,016,314 in outstanding debt, net of debtdiscount of $273,216.

Off-Balance Sheet Arrangements

Recent Accounting Pronouncements

Refer to Note 1. Organization and Summary of Significant Accounting Policies inthe notes to our financial statements for a discussion of recent accountingpronouncements.

Edgar Online, source Glimpses

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-- Yescarta is First CAR T-cell Therapy to Receive NCCN Treatment Guideline Category 1 Recommendation --

Kite, a Gilead Company (Nasdaq: GILD), today announced the U.S. Food and Drug Administration (FDA) has approved Yescarta (axicabtagene ciloleucel) CAR T-cell therapy for adult patients with large B-cell lymphoma that is refractory to first-line chemoimmunotherapy or that relapses within 12 months of first-line chemoimmunotherapy. Yescarta demonstrated a clinically meaningful and statistically significant improvement in event-free survival (EFS; hazard ratio 0.398; P

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220401005519/en/

Earlier this month, the National Comprehensive Cancer Network (NCCN) updated its Clinical Practice Guidelines in Oncology for B-cell Lymphomas to include Yescarta for "Relapsed disease

Christi Shaw, Chief Executive Officer of Kite : "Kite started with a very bold goal: creating the hope of survival through cell therapy. Today's FDA approval brings that hope to more patients by enabling the power of CAR T-cell therapy to be used earlier in the treatment journey. This milestone has been years in the making. On behalf of the entire Kite community, we would like to thank the patients and physicians who have been on this journey with us. You are what drives us every day to explore the full potential of cell therapy."

CAR T-cell therapies are individually made starting from a patient's own white blood cells, called T-cells. The cells are removed through a process similar to donating blood and sent to Kite's specialized manufacturing facilities where they are engineered to target the patient's cancer, expanded, and then returned to the hospital for infusion back into the patient. Referring physicians and patients can immediately begin accessing Yescarta CAR T-cell therapy for this new FDA-approved indication through Kite's 112 authorized treatment centers across the U.S.

Frederick L. Locke, MD, ZUMA-7 Principal Investigator and Co-Leader of the Immuno-Oncology Program at Moffitt Cancer Center, Tampa, Florida : "Today's approval marks an exciting new standard of care. The ZUMA-7 trial enabled us to look at the broader picture of what happens to patients after a decision is made to follow a particular treatment path. What we found was that axi-cel resulted in three times as many patients receiving treatment with curative intent (CAR T-cell therapy), and an overall better outcome for patients than the previous standard of care. Additionally, we have now amassed significant experience with CAR T-cell therapy to better manage or prevent side-effects, making this treatment more accessible for older patients and those with medical conditions for whom the standard of care might be difficult."

SOC therapy for this patient population has historically been a multi-step process expected to end with a stem cell transplant. The process starts with chemoimmunotherapy, and if a patient responds to and can tolerate further treatment, they move on to high-dose chemotherapy (HDT) followed by a stem cell transplant (ASCT).

Jason Westin, MD, MS, FACP, ZUMA-7 Principal Investigator, Director, Lymphoma Clinical Research, and Associate Professor, Department of Lymphoma/Myeloma at The University of Texas MD Anderson Cancer Center : "Definitive clinical trial results such as these do not come along often and should drive a paradigm shift in how patients with relapsed or refractory LBCL are treated moving forward. Patients who do not respond to or relapse after initial treatment should quickly be referred to a CAR T-cell therapy authorized treatment center for evaluation."

Kite CAR T-cell therapy products are widely covered by commercial and government insurance programs in the U.S. Kite has also invested in expansion of manufacturing capacity ahead of today's FDA decision to support patient access.

Lee Greenberger, PhD, Chief Scientific Officer of The Leukemia & Lymphoma Society (LLS): "LLS was an early supporter of CAR T-cell therapy research, and to be able to see this innovative advance become available as an earlier line of treatment is truly remarkable. Current standard of care is a difficult process for patients, and no one knows at the start who will make it to stem cell transplant. With today's FDA decision, patients will have earlier access to this potentially curative treatment."

Yescarta was initially approved by the FDA in 2017 based on the ZUMA-1 trial for a smaller population of LBCL patients who failed two or more lines of therapy. The ZUMA-1 trial has recently reported durable 5-year survival results, with Yescarta showing 42.6% of study patients alive at 5 years and that 92% of those patients alive at 5 years have needed no additional cancer treatment at this important milestone.

As the only company dedicated exclusively to the research, development, commercialization, and manufacturing of cell therapy on a global scale, Kite has all functions critical to cell therapy vertically integrated. This structure enables the continual refinement and support of the highly specialized and complex end-to-end processes needed to support and improve upon patient outcomes with CAR T-cell therapy.

About ZUMA-7 Study

The FDA approval of Yescarta CAR T-cell therapy for adult patients with large B-cell lymphoma (LBCL) that is refractory to first-line chemoimmunotherapy or that relapses within 12 months of first-line chemoimmunotherapy is based on results from the ZUMA-7 study. Patients had not yet received treatment for relapsed or refractory lymphoma and were potential candidates for autologous stem cell transplant (ASCT). Results were presented in a Plenary session at the American Society of Hematology's (ASH) Annual Meeting & Exposition in December 2021 and simultaneously published in the New England Journal of Medicine (NEJM).

ZUMA-7 is a randomized, open-label, global, multicenter, Phase 3 study evaluating the safety and efficacy of Yescarta versus current standard of care (SOC) for second-line therapy (platinum-based salvage combination chemoimmunotherapy regimen followed by high-dose therapy [HDT] and ASCT in those who respond to salvage chemotherapy) in adult patients with relapsed or refractory LBCL within 12 months of first-line therapy. In the study, 359 patients in 77 centers around the world were randomized (1:1) to receive a single infusion of Yescarta or current SOC second-line therapy. The primary endpoint is event-free survival (EFS) as determined by blinded central review and defined as the time from randomization to the earliest date of disease progression per Lugano Classification, commencement of new lymphoma therapy, or death from any cause. Key secondary endpoints include objective response rate (ORR) and overall survival (OS). Additional secondary endpoints include patient reported outcomes (PROs) and safety.

Yescarta demonstrated a 2.5-fold increase in patients who were alive at two years and did not experience cancer progression or require the need for additional cancer treatment (40.5% vs. 16.3%) and a four-fold greater median EFS (8.3 mo. vs. 2.0 mo.) compared to SOC (hazard ratio 0.398; 95% CI: 0.308-0.514, P

Nearly three times as many patients randomized to Yescarta ultimately received the definitive CAR T-cell therapy treatment (94%) versus those randomized to SOC (35%) who received on-protocol HDT+ASCT. More patients responded to Yescarta (ORR: 83% vs. 50%, odds ratio: 5.31 [95% CI: 3.1-8.9; P

Fifty-five percent of patients in the SOC arm subsequently received CD19-directed CAR T-cell therapy off study.

In the study, Yescarta had a safety profile that was consistent with previous studies. Among the 168 Yescarta-treated patients evaluable for safety, Grade 3 cytokine release syndrome (CRS) and neurologic events were observed in 7% and 25% of patients, respectively. In the SOC arm, 83% of patients had high grade events, mostly cytopenias (low blood counts).

The Yescarta U.S. Prescribing Information has a BOXED WARNING for the risks of CRS and neurologic toxicities, and Yescarta is approved with a Risk Evaluation and Mitigation Strategy (REMS) due to these risks; see below for Important Safety Information.

About LBCL

Globally, LBCL is the most common type of non-Hodgkin lymphoma (NHL). In the United States, more than 18,000 people are diagnosed with LBCL each year. About 30-40% of patients with LBCL will need second-line treatment, as their cancer will either relapse (return) or become refractory (not respond) to initial treatment.

About Yescarta

Please see full Prescribing Information , including BOXED WARNING and Medication Guide.

YESCARTA is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of:

Limitations of Use : YESCARTA is not indicated for the treatment of patients with primary central nervous system lymphoma.

U.S. IMPORTANT SAFETY INFORMATION

BOXED WARNING: CYTOKINE RELEASE SYNDROME AND NEUROLOGIC TOXICITIES

CYTOKINE RELEASE SYNDROME (CRS)

CRS, including fatal or life-threatening reactions, occurred. CRS occurred in 90% (379/422) of patients with non-Hodgkin lymphoma (NHL), including Grade 3 in 9%. CRS occurred in 93% (256/276) of patients with large B-cell lymphoma (LBCL), including Grade 3 in 9%. Among patients with LBCL who died after receiving YESCARTA, 4 had ongoing CRS events at the time of death. For patients with LBCL in ZUMA-1, the median time to onset of CRS was 2 days following infusion (range: 1-12 days) and the median duration was 7 days (range: 2-58 days). For patients with LBCL in ZUMA-7, the median time to onset of CRS was 3 days following infusion (range: 1-10 days) and the median duration was 7 days (range: 2-43 days). CRS occurred in 84% (123/146) of patients with indolent non-Hodgkin lymphoma (iNHL) in ZUMA-5, including Grade 3 in 8%. Among patients with iNHL who died after receiving YESCARTA, 1 patient had an ongoing CRS event at the time of death. The median time to onset of CRS was 4 days (range: 1-20 days) and median duration was 6 days (range: 1-27 days) for patients with iNHL.

Key manifestations of CRS ( 10%) in all patients combined included fever (85%), hypotension (40%), tachycardia (32%), chills (22%), hypoxia (20%), headache (15%), and fatigue (12%). Serious events that may be associated with CRS include cardiac arrhythmias (including atrial fibrillation and ventricular tachycardia), renal insufficiency, cardiac failure, respiratory failure, cardiac arrest, capillary leak syndrome, multi-organ failure, and hemophagocytic lymphohistiocytosis/macrophage activation syndrome.

The impact of tocilizumab and/or corticosteroids on the incidence and severity of CRS was assessed in 2 subsequent cohorts of LBCL patients in ZUMA-1. Among patients who received tocilizumab and/or corticosteroids for ongoing Grade 1 events, CRS occurred in 93% (38/41), including 2% (1/41) with Grade 3 CRS; no patients experienced a Grade 4 or 5 event. The median time to onset of CRS was 2 days (range: 1-8 days) and the median duration of CRS was 7 days (range: 2-16 days). Prophylactic treatment with corticosteroids was administered to a cohort of 39 patients for 3 days beginning on the day of infusion of YESCARTA. Thirty-one of the 39 patients (79%) developed CRS and were managed with tocilizumab and/or therapeutic doses of corticosteroids with no patients developing Grade 3 CRS. The median time to onset of CRS was 5 days (range: 1-15 days) and the median duration of CRS was 4 days (range: 1-10 days). Although there is no known mechanistic explanation, consider the risk and benefits of prophylactic corticosteroids in the context of pre-existing comorbidities for the individual patient and the potential for the risk of Grade 4 and prolonged neurologic toxicities.

Ensure that 2 doses of tocilizumab are available prior to YESCARTA infusion. Monitor patients for signs and symptoms of CRS at least daily for 7 days at the certified healthcare facility, and for 4 weeks thereafter. Counsel patients to seek immediate medical attention should signs or symptoms of CRS occur at any time. At the first sign of CRS, institute treatment with supportive care, tocilizumab, or tocilizumab and corticosteroids as indicated.

NEUROLOGIC TOXICITIES

Neurologic toxicities (including immune effector cell-associated neurotoxicity syndrome) that were fatal or life-threatening occurred. Neurologic toxicities occurred in 78% (330/422) of all patients with NHL receiving YESCARTA, including Grade 3 in 25%. Neurologic toxicities occurred in 87% (94/108) of patients with LBCL in ZUMA-1, including Grade 3 in 31% and in 74% (124/168) of patients in ZUMA-7 including Grade 3 in 25%. The median time to onset was 4 days (range: 1-43 days) and the median duration was 17 days for patients with LBCL in ZUMA-1. The median time to onset for neurologic toxicity was 5 days (range:1- 133 days) and median duration was 15 days in patients with LBCL in ZUMA-7. Neurologic toxicities occurred in 77% (112/146) of patients with iNHL, including Grade 3 in 21%. The median time to onset was 6 days (range: 1-79 days) and the median duration was 16 days. Ninety-eight percent of all neurologic toxicities in patients with LBCL and 99% of all neurologic toxicities in patients with iNHL occurred within the first 8 weeks of YESCARTA infusion. Neurologic toxicities occurred within the first 7 days of infusion for 87% of affected patients with LBCL and 74% of affected patients with iNHL.

The most common neurologic toxicities ( 10%) in all patients combined included encephalopathy (50%), headache (43%), tremor (29%), dizziness (21%), aphasia (17%), delirium (15%), and insomnia (10%). Prolonged encephalopathy lasting up to 173 days was noted. Serious events, including aphasia, leukoencephalopathy, dysarthria, lethargy, and seizures occurred. Fatal and serious cases of cerebral edema and encephalopathy, including late-onset encephalopathy, have occurred.

The impact of tocilizumab and/or corticosteroids on the incidence and severity of neurologic toxicities was assessed in 2 subsequent cohorts of LBCL patients in ZUMA-1. Among patients who received corticosteroids at the onset of Grade 1 toxicities, neurologic toxicities occurred in 78% (32/41) and 20% (8/41) had Grade 3 neurologic toxicities; no patients experienced a Grade 4 or 5 event. The median time to onset of neurologic toxicities was 6 days (range: 1-93 days) with a median duration of 8 days (range: 1-144 days). Prophylactic treatment with corticosteroids was administered to a cohort of 39 patients for 3 days beginning on the day of infusion of YESCARTA. Of those patients, 85% (33/39) developed neurologic toxicities, 8% (3/39) developed Grade 3, and 5% (2/39) developed Grade 4 neurologic toxicities. The median time to onset of neurologic toxicities was 6 days (range: 1-274 days) with a median duration of 12 days (range: 1-107 days). Prophylactic corticosteroids for management of CRS and neurologic toxicities may result in higher grade of neurologic toxicities or prolongation of neurologic toxicities, delay the onset and decrease the duration of CRS.

Monitor patients for signs and symptoms of neurologic toxicities at least daily for 7 days at the certified healthcare facility, and for 4 weeks thereafter, and treat promptly.

REMS

Because of the risk of CRS and neurologic toxicities, YESCARTA is available only through a restricted program called the YESCARTA and TECARTUS REMS Program which requires that: Healthcare facilities that dispense and administer YESCARTA must be enrolled and comply with the REMS requirements and must have on-site, immediate access to a minimum of 2 doses of tocilizumab for each patient for infusion within 2 hours after YESCARTA infusion, if needed for treatment of CRS. Certified healthcare facilities must ensure that healthcare providers who prescribe, dispense, or administer YESCARTA are trained about the management of CRS and neurologic toxicities. Further information is available at http://www.YescartaTecartusREMS.com or 1-844-454-KITE (5483).

HYPERSENSITIVITY REACTIONS

Allergic reactions, including serious hypersensitivity reactions or anaphylaxis, may occur with the infusion of YESCARTA.

SERIOUS INFECTIONS

Severe or life-threatening infections occurred. Infections (all grades) occurred in 45% of patients with NHL. Grade 3 infections occurred in 17% of patients, including Grade 3 infections with an unspecified pathogen in 12%, bacterial infections in 5%, viral infections in 3%, and fungal infections in 1%. YESCARTA should not be administered to patients with clinically significant active systemic infections. Monitor patients for signs and symptoms of infection before and after infusion and treat appropriately. Administer prophylactic antimicrobials according to local guidelines.

Febrile neutropenia was observed in 36% of all patients with NHL and may be concurrent with CRS. In the event of febrile neutropenia, evaluate for infection and manage with broad-spectrum antibiotics, fluids, and other supportive care as medically indicated.

In immunosuppressed patients, including those who have received YESCARTA, life-threatening and fatal opportunistic infections including disseminated fungal infections (e.g., candida sepsis and aspergillus infections) and viral reactivation (e.g., human herpes virus-6 [HHV-6] encephalitis and JC virus progressive multifocal leukoencephalopathy [PML]) have been reported. The possibility of HHV-6 encephalitis and PML should be considered in immunosuppressed patients with neurologic events and appropriate diagnostic evaluations should be performed.

Hepatitis B virus (HBV) reactivation, in some cases resulting in fulminant hepatitis, hepatic failure, and death, can occur in patients treated with drugs directed against B cells, including YESCARTA. Perform screening for HBV, HCV, and HIV in accordance with clinical guidelines before collection of cells for manufacturing.

PROLONGED CYTOPENIAS

Patients may exhibit cytopenias for several weeks following lymphodepleting chemotherapy and YESCARTA infusion. Grade 3 cytopenias not resolved by Day 30 following YESCARTA infusion occurred in 39% of all patients with NHL and included neutropenia (33%), thrombocytopenia (13%), and anemia (8%). Monitor blood counts after infusion.

HYPOGAMMAGLOBULINEMIA B-cell aplasia and hypogammaglobulinemia can occur. Hypogammaglobulinemia was reported as an adverse reaction in 14% of all patients with NHL. Monitor immunoglobulin levels after treatment and manage using infection precautions, antibiotic prophylaxis, and immunoglobulin replacement. The safety of immunization with live viral vaccines during or following YESCARTA treatment has not been studied. Vaccination with live virus vaccines is not recommended for at least 6 weeks prior to the start of lymphodepleting chemotherapy, during YESCARTA treatment, and until immune recovery following treatment.

SECONDARY MALIGNANCIES

Secondary malignancies may develop. Monitor life-long for secondary malignancies. In the event that one occurs, contact Kite at 1-844-454-KITE (5483) to obtain instructions on patient samples to collect for testing.

EFFECTS ON ABILITY TO DRIVE AND USE MACHINES

Due to the potential for neurologic events, including altered mental status or seizures, patients are at risk for altered or decreased consciousness or coordination in the 8 weeks following YESCARTA infusion. Advise patients to refrain from driving and engaging in hazardous occupations or activities, such as operating heavy or potentially dangerous machinery, during this initial period.

ADVERSE REACTIONS

The most common non-laboratory adverse reactions (incidence 20%) in patients with LBCL in ZUMA-7 included fever, CRS, fatigue, hypotension, encephalopathy, tachycardia, diarrhea, headache, musculoskeletal pain, nausea, febrile neutropenia, chills, cough, infection with unspecified pathogen, dizziness, tremor, decreased appetite, edema, hypoxia, abdominal pain, aphasia, constipation, and vomiting.

The most common adverse reactions (incidence 20%) in patients with LBCL in ZUMA-1 included CRS, fever, hypotension, encephalopathy, tachycardia, fatigue, headache, decreased appetite, chills, diarrhea, febrile neutropenia, infections with pathogen unspecified, nausea, hypoxia, tremor, cough, vomiting, dizziness, constipation, and cardiac arrhythmias.

The most common non-laboratory adverse reactions (incidence 20%) in patients with iNHL in ZUMA-5 included fever, CRS, hypotension, encephalopathy, fatigue, headache, infections with pathogen unspecified, tachycardia, febrile neutropenia, musculoskeletal pain, nausea, tremor, chills, diarrhea, constipation, decreased appetite, cough, vomiting, hypoxia, arrhythmia, and dizziness.

About Kite

Kite, a Gilead Company, is a global biopharmaceutical company based in Santa Monica, California, with manufacturing operations in North America and Europe. Kite's singular focus is cell therapy to treat and potentially cure cancer. As the cell therapy leader, Kite has more approved CAR T indications to help more patients than any other company. For more information on Kite, please visit http://www.kitepharma.com .

About Gilead Sciences

Gilead Sciences, Inc. is a biopharmaceutical company that has pursued and achieved breakthroughs in medicine for more than three decades, with the goal of creating a healthier world for all people. The company is committed to advancing innovative medicines to prevent and treat life-threatening diseases, including HIV, viral hepatitis and cancer. Gilead operates in more than 35 countries worldwide, with headquarters in Foster City, California.

Forward Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other factors, including the possibility of unfavorable results from ongoing or additional clinical trials involving Yescarta; Kite's ability to initiate, progress or complete clinical trials within currently anticipated timelines or at all, including those involving Yescarta; Kite's ability to receive regulatory approvals in a timely manner or at all, including additional regulatory approvals of Yescarta, and the risk that any such approvals may be subject to significant limitations on use; the risk that physicians may not see the benefits of prescribing Yescarta; and any assumptions underlying any of the foregoing. These and other risks, uncertainties and other factors are described in detail in Gilead's Annual Report on Form 10-K for the year ended December 31, 2021, as filed with the U.S. Securities and Exchange Commission. These risks, uncertainties and other factors could cause actual results to differ materially from those referred to in the forward-looking statements. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. The reader is cautioned that any such forward-looking statements are not guarantees of future performance and involve risks and uncertainties and is cautioned not to place undue reliance on these forward-looking statements. All forward-looking statements are based on information currently available to Kite and Gilead, and Kite and Gilead assume no obligation and disclaim any intent to update any such forward-looking statements.

U.S. Prescribing Information for Yescarta including BOXED WARNING , is available at http://www.kitepharma.com and http://www.gilead.com .

Kite, the Kite logo, Yescarta, Tecartus, XLP and GILEAD are trademarks of Gilead Sciences, Inc. or its related companies.

For more information on Kite, please visit the company's website at http://www.kitepharma.com . Follow Kite on social media on Twitter ( @KitePharma ) and LinkedIn .

View source version on businesswire.com: https://www.businesswire.com/news/home/20220401005519/en/

Jacquie Ross, Investors investor_relations@gilead.com

Mary Lynn Carver, Media mcarver@kitepharma.com

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Mavacamten Demonstrated Significant Reduction in Need for Septal Reduction Therapy in Symptomatic Obstructive HCM Patients in Phase 3 VALOR Trial -...

Cardiac Stem Cells Comments Off on Mavacamten Demonstrated Significant Reduction in Need for Septal Reduction Therapy in Symptomatic Obstructive HCM Patients in Phase 3 VALOR Trial -… | April 3rd, 2022

According to The Insight Partners new research study on Epithelial Cell Culture Media Market Forecast to 2027 COVID-19 Impact and Global Analysis by Product Type and End User, the market is expected to reach US$ 303,040.33 thousand by 2028 from US$ 128,155.95 thousand in 2020; it is estimated to grow at a CAGR of 11.4% from 2021 to 2028.

Certain age-related diseases, abnormalities, and trauma damage the tissues and organs. Regenerative medicines have the potential to replace or heal tissues and organs, along with normalizing congenital defects. In the last decade of the century, tissue engineering techniques have emerged impressively, and they are now being employed in broader areas of regenerative medicine. Thus, it has now become possible to use these techniques in the development of clinical therapies for the maintenance, repair, replacement, and enhancement of biological functions. Further, the regenerative medicines developed using cell-based models can potentially assist researchers in the early intervention of degenerative diseases and traumatic injuries.

Download sample PDF Copy of Epithelial Cell Culture Media Market study at: https://www.theinsightpartners.com/sample/TIPRE00022539/

PromoCell GmbH; Merck KGaA; ATCC; AXOL Bioscience Ltd.; Thermo Fisher Scientific, Inc.; Bio-Techne Corporation; Celprogen, Inc.; Lonza Group AG; HiMedia Laboratories; and Cell Biologics, Inc. are among the leading companies operating in the epithelial cell culture media market.

Geographically, the epithelial cell culture media market is segmented into North America, Europe, Asia Pacific (APAC), the Middle East and Africa (MEA), and South and Central America (SCAM). North America held the largest market share in 2020. In 2020, the US held the largest share of the market in North America. The market growth in North America is attributed to the key driving factors such as the presence of various market players and increasing demand for cell culture products from biopharmaceutical and biotechnology companies.

Human amniotic epithelial cells (hAECs) from placental tissues have gained substantial attention in the field of regenerative medicine owing to their proliferative capacity, easy access, multilineage differentiation potential, and safety. These are perinatal stem cells that have embryonic stem cell-like properties and the capability to be induced to differentiate. Thus, a growing focus on bringing advancements in regenerative medicine is likely to boost the adoption of epithelial cell cultures, thereby bolstering the demand for the respective culture.

Inquiry Before Buying on epithelial cell culture media market at: https://www.theinsightpartners.com/inquiry/TIPRE00022539/

Below is the list of the growth strategies done by the players operating in the epithelial cell culture media market:

In May-21 Bio-Techne has released MimEX GI, a new product line for generating 3-dimensional (3-D) gastrointestinal tissue on a 2-D surface.

In Sep-2020 Axol Bioscience and Censo Biotechnologies Announce Merger. The newentitywould become a global leader in the iPSC-based neuroscience, immune cell, and cardiac simulation industries for drug development and screening.

The report segments the epithelial cell culture media market as follows:

By Product Type

Human Mammary Epithelial CellsBronchia/Trachea Epithelial CellsRenal Epithelial CellsOthers

By End User

Biopharmaceutical CompaniesAcademic and Research Laboratories

Interested in Purchasing epithelial cell culture media market Report? Click here @ https://www.theinsightpartners.com/buy/TIPRE00022539/

About Us:

The Insight Partners is a one stop industry research provider of actionable intelligence. We help our clients in getting solutions to their research requirements through our syndicated and consulting research services. We are a specialist in Technology, Healthcare, Manufacturing, Automotive and Defense, Food & beverage, Chemical and Materials, Semiconductors etc.

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Epithelial Cell Culture Media Market to exceed USD 303040.33 thousand by 2028 says, The Insight Partners - Digital Journal

Cardiac Stem Cells Comments Off on Epithelial Cell Culture Media Market to exceed USD 303040.33 thousand by 2028 says, The Insight Partners – Digital Journal | April 3rd, 2022

Few of us know what they are or exactly how they work. But many of us have heard about the healing powers of stem cells, as well as the controversy surrounding them. Stem cells are well-debated and highly complex with promises ranging from fixing damaged knees to regenerating receding hairlines.

Cleveland Clinic is a non-profit academic medical center. Advertising on our site helps support our mission. We do not endorse non-Cleveland Clinic products or services.Policy

But what are stem cells? And, whats all the fuss all about?

Director of the Center for Regenerative Medicine and Surgery, Amy Lightner, MD, shares the differences between stem cell types, how stem cells can be used and when to be cautious of claims that might be too good to be true.

When most of us think of stem cells, we probably recall images of Dolly the cloned sheep. While its true that Dolly was born of stem cells, her place in science history is just one of many advancements in the field.

In fact, there are many different types of stem cells, each of which has different responsibilities and abilities. What unifies them is their ability to regenerate into new cells.

Regenerative medicine is an emerging field that uses innovative treatments to help regenerate or heal cell function thats lost due to aging, disease or injury, Dr. Lightner explains. The way we achieve this is by using stem cells in large quantities, targeted to a certain area, that the body uses to promote healing.

Adult stem cells are the only type of stem cells that are currently approved for medical use in the United States by the U.S. Food and Drug Administration (FDA). The term adult stem cells is a little confusing because theyre actually found in infants, children and adults. These cells live in a variety of tissue in our bodies including bone marrow, muscles, your brain, your intestines and more.

Think of adult stem cells as a little army of cells that can regenerate themselves into new cells to maintain and repair the tissue or muscle where theyre found. The catch with adult stem cells is that they cant become different types of cells (for example, blood stem cells can only become new blood cells, not skin or brain cells).

Unlike adult stem cells, embryonic stem cells have many more possibilities. Harvested during an embryos blastocyst stage (about five or six days after an embryo has been fertilized in a lab), embryonic stem cells have the potential to become any type of cell (called pluripotent cells). For these reasons, embryonic stem cells are the type of stem cells that generate the controversy most people associate with the topic.

Stem cell therapy has been around since the 1970s, when the first adult bone marrow cells were used to treat blood disease. A bone marrow transplant allows a recipient whose bone marrow cells have been damaged by chemotherapy or disease to receive healthy bone marrow stem cells from a donor.

Those stem cells have the potential to mature within the blood system into different immune cells that recognize and fight off different types of blood cancer. And they also have the ability to heal, says Betty Hamilton, MD, Department of Hematology and Medical Oncology.

Bone marrow transplants are currently used to treat diseases including:

While you may have heard about the use of stem cell therapy for knees, back pain, arthritis, hair loss, diabetes and more, no other types of stem cell therapy beyond bone marrow transplants have yet been approved by the FDA. But thousands of clinical trials are available ranging from treatments for Crohns disease to multiple sclerosis and more. The common link between all these trials is the ability of the stem cells to reduce inflammation and repair damage to your body.

Dr. Hamilton and Dr. Lightner agree that were only just beginning to scratch the surface of stem cell therapy. In recent years, during the height of the COVID-19 pandemic, many clinical trials were underway to explore whether stem cells could be used to help treat the damaged lungs in people severely affected by the disease.

I think potential is the perfect word to describe stem cells, says Dr. Hamilton. We know they have these anti-inflammatory and regenerative properties where they can provide a significant improvement to someone suffering from a certain disease. There are so many diseases where inflammation happens, and something needs to be repaired, and so any help the immune system can get provides a lot of potential.

Scientists are also researching whether adult stem cells can turn into pluripotent stem cells, which would allow the cells to change into any cell type without involving the use of embryonic stem cells.

While the potential for stem cell therapy is great, doctors caution that were not quite there yet.

I always tell patients that ask about stem cell therapy clinics or traveling overseas for stem cell therapy treatment that if its not something that is a clinical trial with FDA oversight, then they have no real way of knowing whats being given to them, advises Dr. Lightner.

This means more harm can come than good if you dont know exactly whats being given to you. Or, in some cases, youre just spending thousands of dollars for what ends up being saline, Dr. Lightner says.

The best way to know that youre receiving sound medical treatment is to make sure the one youre considering is approved by the FDA on its Clinical Trials database.

Dr. Lightner cautions against treatments that sound too good to be true. While stem cell therapy has helped improve and save millions of lives, its best to know what exactly youre signing up for by seeking out a qualified medical provider offering an FDA-approved clinical trial.

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Is Stem Cell Therapy Right for You? - Health Essentials from Cleveland Clinic

Skin Stem Cells Comments Off on Is Stem Cell Therapy Right for You? – Health Essentials from Cleveland Clinic | April 3rd, 2022

This spring, streamlineand save time and moneywith these seasonal tips and tricks

USE THE CODE 'SPRINGSKIN' FOR 10% OFF ATSCENTUALS.COM, ONLY UNTIL JUNE 21, 2022

The weather is getting warmer, the days a little longer, and flowers are starting to bloom. As the seasons change, so should our skin care routines. Vancouver-based Scentuals is celebrating the start of spring with seven easy ways to make the change.

Good skin starts from the inside out. Drink enough water, exercise regularly, eat healthy balanced meals, take your vitamins, and prioritize quality sleep.

Your skin care products have expiry dates too. Using products past their shelf life makes them less effectiveor entirely ineffectiveand can cause skin irritation.

ScentualsRefresh and gently balance your skin with an alcohol-free mist. Better yet, try one that smells like roses. Made with floral waters, witch hazel and hyaluronic acid, Scentuals Rose Facial Mist helps soothe and refine your skin.

Say goodbye to dull winter skin and hello to renewed healthy radiance. The Scentuals Radiance Facial Scrub gently exfoliates with ground apricot seeds, while cucumber extract calms skin irritation and reduces redness.

ScentualsSwap out thick cleansers and creams for lighter, fast-absorbing options. The Radiance Facial Cleanser and Cream by Scentuals have you covered.

ScentualsBrightening vitamin C leaves your skin with a healthy glow and promotes collagen production. Try the award-winning Scentuals Vitamin C Serum, effectively formulated with vitamin C, hyaluronic acid and plant stem cells.

Defend your skin against harmful UV rays by wearing sunscreen daily. Additional benefits to daily SPF include preventing sunburns, skin cancer and premature aging.

CREATED BY VANCOUVER MAGAZINE, IN PARTNERSHIP WITH SCENTUALS

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7 Easy Ways to Transition Your Skin Care Routine for Spring - Vancouver Magazine

Skin Stem Cells Comments Off on 7 Easy Ways to Transition Your Skin Care Routine for Spring – Vancouver Magazine | April 3rd, 2022

Cell and gene therapies are poised to have a major impact on the landscape of modern medicine, carrying the potential to treat an array of different diseases with unmet clinical need.

However, the number of approved, clinically adopted cell and gene therapies is mere compared to the amount that are currently in development. A major barrier for the translation of such therapies is the safe integration of therapeutic genes into the human genome. The insertion of therapeutic genes bears the risk of off target effects, or integration of the gene into an unintended location.

A number of different strategies have been proposed to mitigate this effect. The most recent body of work comes from a collaboration between Harvards Wyss Institute for Biologically Inspired Engineering, Harvard Medical School (HMS) and the ETH Zurich in Switzerland.

Published in Cell Report Methods, the research focused on identifying safe spots in the genome. These locations, known as genomic safe harbors (GSHs), are areas in the genome that meet the following criteria: they can be accessed easily by genome-editing strategies, are within a safe distance from genes that possess functional properties and permit expression of a therapeutic gene, only once it has landed in the harbor. A simple analogy is deciding which harbor to dock a boat there are many considerations, and these depend on the type of boat you are sailing, the weather conditions and ease of access.

The research team adopted computational strategies that enabled the identification of 2,000 predicted GSHs. From this initial identification, they successfully validated two of the sites both in vitro and in vivo using reporter proteins.

Technology Networks interviewed the studys first author, Dr. Erik Aznauryan, research fellow in the laboratory of Professor George Church at Harvard Medical School. Aznauryan dives into further detail on the history of GSH research, the methods adopted to validate the GSH sites and the potential applications of this research.

Molly Campbell (MC): Can you talk about the history of genomic safe harbor research, and how they were discovered?

Erik Aznauryan (EA): Three genomic sites were empirically identified in previous studies to support stable expression of genes of interest in human cells: AAVS1, CCR5 and hRosa26. All these examples were established without any a-priori safety assessment of the genomic loci they reside in.

Attempts have been made to identify human GSH sites that would satisfy various safety criteria, thus avoiding the disadvantages of existing sites. One approach developed by Sadelain and colleagues used lentiviral transduction of beta-globin and green fluorescence protein genes into induced pluripotent stem cells (iPSCs), followed by the assessment of the integration sites in terms of their linear distance from various coding and regulatory elements in the genome, such as cancer genes, miRNAs and ultraconserved regions.

They discovered one lentiviral integration site that satisfied all of the proposed criteria, demonstrating sustainable expression upon erythroid differentiation of iPSCs. However, global transcriptome profile alterations of cells with transgenes integrated into this site were not assessed. A similar approach by Weiss and colleagues used lentiviral integrations in Chinese hamster ovary (CHO) cells to identify sites supporting long-term protein expression for biotechnological applications (e.g., recombinant monoclonal antibody production). Although this study led to the evaluation of multiple sites for durable, high-level transgene expression in CHO cells, no extrapolation to human genomic sites was carried out.

Another study aimed at identifying GSHs through bioinformatic search of mCreI sites regions targeted by monomerized version of I-CreI homing endonuclease found and characterized in green algae as capable to make targeted staggered double-strand DNA breaks residing in loci that satisfy GSH criteria. Like previous work, several stably expressing sites were identified and proposed for synthetic biology applications in humans. However, local and global gene expression profiling following integration events in these sites have not been conducted.

All these potential GSH sites possess a shared limitation of being narrowed by lentiviral- or mCreI-based integration mechanisms. Additionally, safety assessments of some of these identified sites, as well as previously established AAVS1, CCR5 and Rosa26, were carried out by evaluating the differential gene expression of genes located solely in the vicinity of these integration sites, without observing global transcriptomic changes following integration.

A more comprehensive bioinformatic-guided and genome-wide search of GSH sites based on established criteria, followed by experimental assessment of transgene expression durability in various cell types and safety assessment using global transcriptome profiling would, thus, lead to the identification of a more reliable and clinically useful genomic region.

MC: If GSHs do not encode proteins, or RNAs with functions in gene expression, or other cellular processes what is their function in the genome?

EA: In addition to protein coding, functional RNA coding, regulatory and structural regions of the human genome, other less well understood and inactive DNA regions exist.

A large proportion of the human genome seems to have evolved in the presence of a variety of integrating viruses which, as they inserted their DNA into the eukaryotic genome over the course of million years, lead to an establishment of vast non-coding elements that we continue to carry to this day. Furthermore, partial duplications of functional human genes have resulted in the formation of inactive pseudogenes, which occupy space in the genome yet are not known to bear cellular functions.

Finally, functional roles of some non-coding portions of the human genome are not well understood yet. Our search of safe harbors was conducted using existing annotation of the human genome, and as more components of it are deciphered the identification of genomic regions safe for gene insertion will become more informed.

MC: Are you able to discuss why some regions of the genome were previously regarded as GSHs but are now recognized as non-GSHs?

EA: In the absence of other alternatives, AAVS1, CCR5 and hRosa26 sites were historically called GSHs, as they supported the expression of genes of interest in a variety of cell types and were suitable for use in a research setting.

Their caveats (mainly, location within introns of functional genes, closely surrounded by other known protein coding genes as well as oncogenes) however prevent them from being used for clinical applications. Therefore, in our paper we dont call them GSHs, and refer to our newly discovered sites as GSHs.

MC: You thoroughly scanned the genome to identify candidate loci for further study as potential GSHs. Can you discuss some of the technological methods you adopted here, and why?

EA: We used several publicly available databases to identify genomic coordinates of structural, regulatory and coding components of the human genome according to the GSH criteria we outlined in the beginning of our study (outside genes, oncogenes, lncRNAs etc.,). We used these coordinates and bioinformatic tools such as command lines bedtools to exclude these genomic elements as well as areas adjacent to them. This left us with genomic regions putative GSHs from which we could then experimentally validate by inserting reporter and therapeutic genes into them followed by transcriptomic analysis of GSH-integrated vs non-integrated cells.

MC: You narrowed down your search to test five, and then two GSHs. Can you expand on your choice of reporter gene when assessing two GSHs in cell lines?

EA: Oftentimes in research you go with what is available or what is of the most interest to the lab you are currently working in.

Our case was not an exception, and we initially (up until the T cell work) used the mRuby reporter gene as it was widely available and extensively utilized and validated in our lab at ETH Zurich back then.

When I moved to the Wyss Institute at Harvard, I began collaborating with Dr. Denitsa Milanova, who was interested in testing these sites in the context of skin gene therapy particularly the treatment of junctional epidermolysis bullosa caused by mutations in various anchor proteins connecting different layers of skin, among which is the LAMB3 gene. For this reason, we decided to express this gene in human dermal fibroblasts, together with green fluorescent protein to have a visualizable confirmation of expression. We hope we would be able to translate this study into clinics.

MC: Can you describe examples of how GSHs can be utilized in potential therapeutics?

EA: Current cell therapy approaches rely on random insertion of genes of interest into the human genome. This can be associated with potential side effects including cancerous transformation of therapeutic cells as well as eventual silencing of the inserted gene.

We hope that current cell therapies will eventually transition to therapeutic gene insertions precisely into our GSHs, which will alleviate both described concerns. Specific areas of implementation may involve safer engineering of T cells for cancer treatment: insertion of genes encoding receptors targeting tumor cells or cytokines capable of enhancing anti-tumor response.

Additionally, these sites can be used for the engineering of skin cells for therapeutic (as discussed earlier with the LAMB3 example) as well as anti-aging applications, such as expression of genes that result in youthful skin phenotype.

Finally, given the robustness of gene expression from our identified sites, they can be used for industry-scale bio-manufacturing: high-yield production of proteins of interest in human cell lines for subsequent extraction and therapeutic applications (e.g., production of clotting factors for patients with hemophilias).

MC: Are there any limitations to the research at this stage?

EA: A primary limitation to this study is the low frequency of genomic integration events using CRISPR-based knock-in tools. This means that cells in which the gene of interest successfully integrated into the GSH must be pulled out of the vastly larger population of cells without this integration.

These isolated cells would then be expanded to generate homogenous population of gene-bearing cells. Such pipeline is not ideal for a clinical setting and improvements in gene integration efficiencies are needed to help this technology easier translate into clinics.

Our lab is currently working on developing genome engineering tools which would eventually allow to integrate large genes into GSHs with high precision and efficiency.

MC: What impact might this study have on the cell and gene therapy development space?

EA: This study will hopefully lead to many researchers in the field testing our sites, validating them in other therapeutically relevant cell types and eventually using them in research as well as in clinics as more reliable, durable and safe alternatives to current viral based random gene insertion methods.

Additionally, since in our work we shared all putative GSHs identified by our computational pipeline, we hope researchers will attempt to test sites we havent validated yet by implementing the GSH evaluation pipeline that we outlined in the paper. This will lead to identification of more GSHs with perhaps even better properties for clinical translation or bio-manufacturing.

MC: What are your next steps in advancing this work?

We hope to one day translate our successful in vitro skin results and start using these GSHs in an in vivo context.

Additionally, we are looking forward to improving integration efficiencies into our GSHs, which would further support clinical transition of our sites.

Finally, we will evaluate the usability of our GSHs for large-scale production of therapeutically relevant proteins, thus ameliorating the pipeline of manufacturing of biologics.

Dr. Erik Aznauryan was speaking to Molly Campbell, Senior Science Writer for Technology Networks.

Link:
Sailing the Genome in Search of Safe Harbors - Technology Networks

Skin Stem Cells Comments Off on Sailing the Genome in Search of Safe Harbors – Technology Networks | April 3rd, 2022

image:zebrafish notochord nuclei at 15-somite stage. Grey: nuclear DNA (DAPI). Color: histone H3K9me3 view more

Credit: Phong Nguyen, Franka Rang & Kim de Luca. Copryight Hubrecht Institute.

How is the activity of genes regulated by the packaging of DNA? To answer this question, a technique to measure both gene expression and DNA packaging at the same time was developed by Franka Rang and Kim de Luca, researchers from the group of Jop Kind (group leader at the Hubrecht Institute and Oncode Investigator). This method, EpiDamID, determines the location of modified proteins around which the DNA is wrapped. It is important to gather information about these modifications, because they influence the accessibility of DNA, thereby affecting the gene activity. EpiDamID is therefore valuable for research into the early development of organisms. The results of the study are published in Molecular Cell on April 1st 2022.

In order to fit DNA into the nucleus of a cell, it is tightly packed around nuclear proteins: histones. Depending on the tightness of this winding, the DNA can be (in)accessible to other proteins. This therefore determines whether the process of gene expression, translation of DNA into RNA and eventually into proteins, can take place.

DNA packaging determine gene activity

The tightness of DNA winding around histones is regulated by the addition of molecular groups, so-called post-translational modifications (PTMs), to the histones. For example, if certain molecules are added to the histones, the DNA winding is loosened. This makes the DNA more accessible for certain proteins and causes the genes in this part of the DNA to become active, or expressed. Furthermore, proteins that are crucial for gene expression can directly recognize and bind the PTMs. This enables transcription: the process of DNA copying.

The regulation of gene expression, for instance through PTMs, is also known as epigenetic regulation. Since all cells in a body have the same DNA, regulation of gene expression is needed to (de)activate specific functions in individual cells. For instance, heart muscle cells have different functions than skin cells, thus require different genes to be expressed.

Analysis of single cells using EpiDamID

To understand how PTMs affect gene expression, first authors Franka Rang and Kim de Luca designed a new method to determine the location of the modifications. Using this approach, called EpiDamID, researchers can analyze single cells, whereas previous methods were only able to measure a large group of cells. Analysis on such a small scale results in knowledge on how DNA winding differs per cell, rather than information on the average DNA winding of many cells.

EpiDamID is based on DamID, a technique which is used to determine the binding location of certain DNA-binding proteins. Using EpiDamID, the binding location of specific PTMs on histone proteins can be detected in single cells. Compared to others, a great advantage of this technique is that researchers need very limited material. Furthermore, EpiDamID can be used in combination with other methods, such as microscopy, to study regulation of gene expression on different levels.

Future prospects

Following the development of this technique, the Kind group will focus on the role of PTMs from the point of view of developmental biology. Because single cells are analyzed using EpiDamID, only a limited amount of material is needed to generate enough data. This allows researchers to study the early development of organisms from its first cell divisions, when the embryo consists of only a few cells.

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Publication

Rang, F. J.*, de Luca, K. L.*, de Vries, S. S., Valdes-Quezada, C., Boele, E., Nguyen, P. D., Guerreiro, I., Sato, Y., Kimura, H., Bakkers, J. & Kind, J. Single-cell profiling of transcriptome and histone modifications with EpiDamID. Molecular Cell, 2022.

*Authors contributed equally

Jop Kind is group leader at the Hubrecht Institute for Developmental Biology and Stem Cell Research and Oncode Investigator.

About the Hubrecht Institute

The Hubrecht Institute is a research institute focused on developmental and stem cell biology. It encompasses 21 research groups that perform fundamental and multidisciplinary research, both in healthy systems and disease models. The Hubrecht Institute is a research institute of the Royal Netherlands Academy of Arts and Sciences (KNAW), situated on Utrecht Science Park. Since 2008, the institute is affiliated with the UMC Utrecht, advancing the translation of research to the clinic. The Hubrecht Institute has a partnership with the European Molecular Biology Laboratory (EMBL). For more information, visit http://www.hubrecht.eu.

Experimental study

Cells

Single-cell profiling of transcriptome and histone modifications with EpiDamID

1-Apr-2022

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