NEW YORK, Jan. 9, 2020 /PRNewswire/ -- Tacitus Therapeutics, a clinical-stage company, has launched in collaboration with the Mount Sinai Health System to develop stem cell therapies initially targeting blood cancers and related clotting disorders. Their first therapy, HSC100, currently is being investigated in a Phase I clinical trial1.

Tacitus is building upon technology developed by and exclusively licensed from Mount Sinai. Based on research by scientific co-founders Ronald Hoffman, M.D., and Camelia Iancu-Rubin, Ph.D., the technology includes proprietary cell expansion, differentiation and engineering methods. Together, these methods manufacture healthy cells that overcome the limitations of traditional allogeneic, or donor, cell transplantations.

Blood cancers comprise about 10% of new cancer cases in the U.S. each year, and almost 60,000 people die from blood cancer complications annually. Most blood cancers start in the bone marrow, where blood is produced. A common therapy for such blood cancers is a hematopoietic stem cell (HSC) treatment or, as more commonly referred to, bone marrow transplantation. In this process, doctors infuse healthy HSCs into the patient's bloodstream, where they migrate to the bone marrow to grow or engraft.

HSCs for this process can be collected from bone marrow, circulating blood, or umbilical cord blood (CB) of healthy donors. While HSC transplants are common, significant barriers to success exist, including high levels of graft-versus-host disease, low numbers of healthy cells obtained from CB, and increased risk of bleeding due to delayed megakaryocyte, or platelet, engraftment.

Hoffman and Iancu-Rubin are pioneers of bone marrow cell therapy treatments, and development of this technology was enabled by the New York State Stem Cell Science program, NYSTEM. As a New York State Department of Health initiative, NYSTEM awarded a $1 million grant to Hoffman in 2010 that supported the original research underpinning this platform technology. In 2015, NYSTEM awarded Hoffman and Iancu-Rubin an $8 million grant to translate the technology from the laboratory into the clinic, where it is currently in clinical trial1.

Hoffman also serves as Director of the Myeloproliferative Disorders Research Program and Professor of Medicine (Hematology and Medical Oncology) and Iancu-Rubin is Associate Professor of Pathology at the Icahn School of Medicine and Director of the Cellular Therapy Laboratory at Mount Sinai Hospital.

"Promising discoveries by Mount Sinai scientific thought leaders may lead to new, essential cell-based therapies that will broadly benefit patients," said Erik Lium, Executive Vice President and Chief Commercial Innovation Officer, Mount Sinai Innovation Partners. "We're pleased to be collaborating with Tacitus to launch the next stage of development for these technologies."

"Tacitus is committed in its mission to advance next-generation cell therapies with curative potential," said Carter Cliff, CEO of Tacitus. "Based on our founders' solid foundation of research, we are translating these discoveries into broad clinical practice as we look to dramatically improve the standard of care for patients with life-threatening conditions."

About HSC100

HSC100 is an investigational therapy based on allogeneic hematopoietic stem cells (HSC) expanded from umbilical cord blood. HSC100 is being investigated currently in an open-label Phase I clinical trial1 in the United States for treatment of hematological malignancies. The success of unmanipulated cord blood as a source of stem cells has been hampered by the small number of stem cells present in a single cord, leading to delayed engraftment and frequent graft failure. Our proprietary technology includes the use of an epigenetic modifier, valproic acid, to expand the number and the quality of HSCs found in cord blood collections. For more information on HSC100 clinical trials, please visit http://www.clinicaltrials.gov.

1ClinicalTrials.gov identifier NCT03885947.

About Tacitus Therapeutics

Tacitus Therapeutics is a clinical-stage biotechnology company developing advanced medicines for treatment of blood cancers, immune disorders and other intractable disease conditions. Our mission is to pioneer best-in-class therapies using proprietary cell expansion, differentiation and engineering platform technologies that overcome the limitations of traditional cell transplantation. Initial targets include a lead clinical program (HSC100) investigating the treatment of blood cancers, followed by preclinical programs to address clotting disorders and other serious unmet medical needs. For additional information, please visit http://www.tacitustherapeutics.com.

About Mount Sinai Health System

The Mount Sinai Health System is New York City's largest integrated delivery system, encompassing eight hospitals, a leading medical school, and a vast network of ambulatory practices throughout the greater New York region. Mount Sinai's vision is to produce the safest care, the highest quality, the highest satisfaction, the best access and the best value of any health system in the nation. The Health System includes approximately 7,480 primary and specialty care physicians; 11 joint-venture ambulatory surgery centers; more than 410 ambulatory practices throughout the five boroughs of New York City, Westchester, Long Island, and Florida; and 31 affiliated community health centers. The Icahn School of Medicine is one of three medical schools that have earned distinction by multiple indicators: ranked in the top 20 by U.S. News & World Report's "Best Medical Schools", aligned with a U.S. News & World Report's "Honor Roll" Hospital, No. 12 in the nation for National Institutes of Health funding, and among the top 10 most innovative research institutions as ranked by the journal Nature in its Nature Innovation Index. This reflects a special level of excellence in education, clinical practice, and research. The Mount Sinai Hospital is ranked No. 14 on U.S. News & World Report's "Honor Roll" of top U.S. hospitals; it is one of the nation's top 20 hospitals in Cardiology/Heart Surgery, Diabetes/Endocrinology, Gastroenterology/GI Surgery, Geriatrics, Gynecology, Nephrology, Neurology/Neurosurgery, and Orthopedics in the 2019-2020 "Best Hospitals" issue. Mount Sinai's Kravis Children's Hospital also is ranked nationally in five out of ten pediatric specialties by U.S. News & World Report. The New York Eye and Ear Infirmary of Mount Sinai is ranked 12th nationally for Ophthalmology, Mount Sinai St. Luke's and Mount Sinai West are ranked 23rd nationally for Nephrology and 25th for Diabetes/Endocrinology, and Mount Sinai South Nassau is ranked 35th nationally for Urology. Mount Sinai Beth Israel, Mount Sinai St. Luke's, Mount Sinai West, and Mount Sinai South Nassau are ranked regionally. For more information, visit http://www.mountsinai.org or find Mount Sinai on Facebook, Twitter and YouTube.

About Mount Sinai Innovation Partners (MSIP)

MSIP is responsible for driving the real-world application and commercialization of Mount Sinai discoveries and inventions and the development of research partnerships with industry. Our aim is to translate discoveries and inventions into health care products and services that benefit patients and society. MSIP is accountable for the full spectrum of commercialization activities required to bring Mount Sinai inventions to life. These activities include evaluating, patenting, marketing and licensing new technologies building research, collaborations and partnerships with commercial and nonprofit entities, material transfer and confidentiality, coaching innovators to advance commercially relevant translational discoveries, and actively fostering an ecosystem of entrepreneurship within the Mount Sinai research and health system communities. For more information, please visit http://www.ip.mountsinai.orgor find MSIP onLinkedIn, Twitter, Facebook,Medium, and YouTube.

Media Contacts:

Mount Sinai Cynthia Cleto Mount Sinai Innovation Partners (646) 605-7359 cynthia.cleto@mmsm.edu

Tacitus TherapeuticsJoleen RauRau Communications(608) 209-0792232130@email4pr.com

SOURCE Tacitus Therapeutics

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Tacitus Therapeutics Launches in Collaboration with Mount Sinai to Develop Stem Cell Therapies for Life-Threatening Diseases - PRNewswire

Bone Marrow Stem Cells Comments Off on Tacitus Therapeutics Launches in Collaboration with Mount Sinai to Develop Stem Cell Therapies for Life-Threatening Diseases – PRNewswire | January 10th, 2020

What happened

Shares of CRISPR Therapeutics (NASDAQ:CRSP) rose over 113% last year, according to data provided by S&P Global Market Intelligence. The pharma stock built momentum throughout much of the year, but surged in October ahead of an important data presentation that ultimately lived up to the hype. That allowed the gene-editing stock to easily outperform the 28.8% gain of the S&P 500 in 2019.

The end-of-year rally was driven by promising clinical results for its lead drug candidate. The first two individuals, one with sickle cell disease (SCD) and one with transfusion-dependent beta thalassemia (TDT), dosed with CTX001 achieved functional cures after receiving an initial dose of the gene-editing product. The results need to be proven durable and replicated in a larger number of patients, but the update was about as good as investors could have hoped for at the current stage of development.

Image source: Getty Images.

Both SCD and TDT are caused by structural abnormalities in red blood cells. But these are one of the few cells in the human body that don't contain DNA. That means CRISPR Therapeutics has to harvest stem cells from the bone marrow of patients, apply gene editing to those extracted cells, and then inject the engineered stem cells back into patients (the ex vivo method). If the therapy works, then the engineered stem cells should produce functional red blood cells and potentially result in a cure.

In the early study, the ex vivo approach of CTX001 appeared to do just that. The TDT patient required an average of 16.5 blood transfusions per year in the two years before the clinical trial. Nine months after receiving the gene-editing treatment, the individual was transfusion independent (compared with an expected 12 transfusions) and expressed working copies of hemoglobin on 99.8% of red blood cells.

The SCD patient experienced an average of seven vaso-occlusive crises (painful blockages of blood vessels caused by abnormally shaped red blood cells) per year in the two years before the clinical trial. Four months after receiving the gene-editing treatment, the individual reported no vaso-occlusive crises (compared with an expectation for two such episodes) and expressed working copies of hemoglobin on 94.7% of red blood cells.

The early success of CTX001 bodes well for the ex vivo approach of CRISPR Therapeutics and its partner Vertex Pharmaceuticals(NASDAQ:VRTX), but investors should be careful not to extrapolate the results too broadly. Gene-editing tools that are applied inside the body (in vivo) face significantly steeper obstacles, such as the difficulty of delivering gene-editing payloads to specific tissue types inside the body. There's also the elephant in the room: Scientists are beginning to realize that current-generation CRISPR gene-editing tools don't work all that well.

Nonetheless, CRISPR Therapeutics is the top CRISPR-based gene-editing stock on the market. It has the cash, the partnerships, and the early results to back up its claim to that label.

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Here's Why CRISPR Therapeutics Stock Jumped 113.2% in 2019 - Motley Fool

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Miami, FL, Jan. 09, 2020 (GLOBE NEWSWIRE) -- The formal ceremony of the 2020 Stem Cell and Regenerative Medicine Action Awards will take place at a gala reception and dinner on January 23, during the 15th annual World Stem Cell Summit (WSCS) at the Hyatt Regency in Miami. Since 2005, the nonprofit Regenerative Medicine Foundation (RMF) (formerly Genetics Policy Institute) has recognized the stem cell and regenerative medicine community's leading innovators, leaders, and champions through its annual awards reception.

Bernard Siegel, Executive Director of Regenerative Medicine Foundation and founder of the World Stem Cell Summit, said, The 2020 Action Awards will recognize three important organizations that are positively impacting the emerging field of regenerative medicine. We will also honor a retired Major General, who has capped off his military and diplomatic career by promoting the cause of world peace through medicine. All of these distinguished honorees will be recognized for their devotion to improving health and developing cures through advocacy, innovation, leadership and inspiration. In addition, the wounded warrior veterans community of South Florida will also receive special recognition at the event.

Meet the 2020 Stem Cell & Regenerative Medicine Action Award Honorees:

Innovation Award: With the motto, We will not rest until we find a cure, the Cystic Fibrosis Foundation is geared towards the successful development and delivery of treatments, therapies and a cure for every person with cystic fibrosis. CF Foundation has added decades to the lives of people with the disease as a direct result of advances in treatment and care made possible through its innovative business model- venture philanthropy. The Foundation recently unveiled its Path to a Cure research agenda aimed at addressing the root genetic cause of the disease and is currently funding industry programs aimed at gene delivery with the goal of progressing into clinical studies in 2021.

Inspiration Award: Emily Whitehead Foundation is a nonprofit organization committed to raising funds to invest in the most promising pediatric cancer research. Tom and Kari Whitehead founded EWF in honor of their daughter Emily, the first child in the world to receive CAR T-cell therapy, training her own cells to fight cancer. Her inspiring story focused public attention on thepotential for cancer immunotherapy to transform cancer treatment,as well as the need to support lifesaving cancer immunotherapy research. The foundation provides support to pediatric cancer patients and promotes awareness of the disease through education and sharing other inspiring stories.

Advocacy Award: Gift of Life Marrow Registry was established in 1991 by Jay Feinberg and his family after Jay received a life-saving bone marrow transplant. Gift of Life is dedicated to saving lives and facilitating bone marrow and blood stem cell transplants for patients with leukemia, lymphoma, sickle cell and other diseases. In 2019, Gift of Life opened the worlds first apheresis center fully integrated within a registry, the Dr. Miriam and Sheldon G. Adelson Gift of Life-Be The Match Stem Cell Collection Center. With the collection center and rapidly expanding donor database, Gift of Life will launch a biobank to advance cellular therapies using allogeneically sourced cells in 2020.

Leadership Award: Ret. Major General Bernard Burn Loeffke, PhD (US Military) is a highly decorated Special Forces officer, diplomat and medical officer.He survived two helicopter crashes and was wounded in combat. After the Vietnam War, he served as the Army Attach at theU.S. Embassy in Moscow, first Defense Attach at the U.S Embassy in Beijing, a staff officer in theWhite House, and Director of the Commission onWhite House Fellows. His last command was Commanding General of Army South. After 35 years in the military, he became a medical officer traveling the world on relief missions to third and fourth world countries. Presently, at age 85, he champions the hydrocephalus and wounded warrior communities. He continues to serve as an inspiration and supporter of building peaceful international relations through medical partnerships and played a pivotal role as a keynote speaker at the inaugural 2019 World Stem Cell Summit CHINA.He is called the Peace General in Latin America. In China, he is simply known as The General, our Friend.

To learn more about past honorees and details for sponsoring or attending the upcoming 2020 Stem Cell and Regenerative Medicine Action Awards dinner, please visit, https://www.worldstemcellsummit.com/stem-cell-action-awards/

About the World Stem Cell Summit (WSCS)

Produced by the non-profit Regenerative Medicine Foundation (RMF), and in its 15th year, the World Stem Cell Summit will take place January 21-24, 2020, in Miami, Florida in partnership with Phacilitate Leaders World, as part of Advanced Therapies Week. The Summit is the most inclusive and expansive interdisciplinary, networking, and partnering meeting in the stem cell science and regenerative medicine field. With the overarching purpose of fostering translation of biomedical research, funding, and investments targeting cures, the Summit and co-located conferences serve a diverse ecosystem of stakeholders. For more information about the upcoming World Stem Cell Summit in Miami, please visit: http://www.worldstemcellsummit.com.

About the Regenerative Medicine Foundation (RMF)

The nonprofit Regenerative Medicine Foundation fosters strategic collaborations to accelerate the development of regenerative medicine to improve health and deliver cures. RMF unites the worlds leading researchers, medical centers, universities, labs, businesses, funders, policymakers, experts in law, regulation and ethics, medical philanthropies, and patient organizations. We maintain a trusted network of leaders and pursue our mission by producing our flagship World Stem Cell Summit series of conferences and public days, honoring leaders through the Stem Cell and Regenerative Medicine Action Awards, supporting our official journal partner STEM CELLS Translational Medicine (SCTM), promoting solution-focused policy initiatives both nationally and internationally and creating STEM/STEAM educational projects. For more information about RMF, please visit: http://www.regmedfoundation.org.

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Stem Cell and Regenerative Medicine Action Awards to be Presented at World Stem Cell Summit on January 23 at the Hyatt Regency Miami - GlobeNewswire

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LEUKOTAC (inolimomab) is available again in France, following the granting of cohort ATU for the treatment of graft-versus-host disease, corticosteroid-resistant or corticosteroid-dependent, with grade II-IV

Lyon, FRANCE, January 9, 2020, ElsaLys Biotech announced today that the cATU has been granted by the ANSM and its clinical experts, after evaluation of a dossier containing data on the quality, safety and efficacy of the drug based on its administration in several hundred patients included in clinical trials or treatedvia named patient Temporary Authorization for Use (ATU nominative) until November 2015. This authorization includes the implementation of a reinforced monitoring (defined in the Protocol for Therapeutic Use) of the efficacy and safety data obtained in patients treated within the framework of this cATU. Inolimomab treatment can only be considered if the patient cannot be included in an ongoing clinical trial.

"We have data that support the benefit of inolimomab treatment in patients with acute corticosteroid-resistant or corticosteroid-dependent graft-versus-host disease (Grades II-IV in Glucksberg classification)," said Dr. David LIENS, Chief Medical Officer, ElsaLys Biotech. "We are delighted with this decision by the ANSM, which allows us to, once again, make inolimomab (1 mg/mL, solution for infusion) available to hematologists in the therapeutic emergency which is this pathology".

"While we continue to work on the filing of marketing authorization applications (MAA) in Europe and in the US, this ATU demonstrates the therapeutic value of inolimomab in the management of acute graft-versus-host disease (aGvHD). The ATU program in France allows patients, whose survival is at stake, to have access to a therapeutic solution before marketing in Europe, in close collaboration with the competent authority, the ANSM. The implementation of this cATU is effective immediately" said Dr. Christine GUILLEN, CEO and co-founder of ElsaLys Biotech.

Considering the potential emergency situation of the indication, it is recommended that hematology specialists anticipate the administrative procedures by contacting the ATU Cell (by Tel: 0800 08 90 81 - Fax: 01 56 59 05 60 or by e-mail: atu-leukotac@pharma-blue.com) which is at their disposal for any further information or request for a Protocol for Therapeutic Use and collection of information.

About inolimomab (LEUKOTAC)

Inolimomab (LEUKOTAC) is an immunotherapy monoclonal antibody that targets the interleukin-2 receptor (IL-2), a chemical molecule named cytokine that contributes to the development and proliferation of some white blood cells including T-cells responsible for aGvHD. By linking specifically to the a chain of the receptor (CD25), inolimomab prevents IL-2 from binding on the surface of the donors over-active T-cells which blocks their multiplication.

The efficacy of inolimomab in aGvHD lies mainly in its specificity and its preferential affinity to the CD25 receptor found on the surface of T-lymphocytes.

About steroid-resistant aGvHD

Formerly called bone marrow transplant, Hematopoietic Stem Cell Transplantation (HSCT) is the last therapeutic option for patients with certain blood cancers or severe immunodeficiency. In practice, the treatment is designed to replace the diseased blood cells of the patient with the hematopoietic stem cells of a matching donor (allograft).

Once grafted, these stem cells will produce new healthy and functional blood cells, including white blood cells that will allow patients to bridge their immune deficiency or to eliminate surviving cancer cells.

If this technique has made considerable progress in 60 years, half of transplant recipients are still victims of complications: side effects of conditioning pretreatment (that aims to prevent transplant rejection), long-term susceptibility to infections and GvHD. In the latter case, the donors over-active T-cells turn against the patients tissues: mucous membranes, skin, gastro-intestinal tract, liver and lungs. The acute form appears just after the transplant, the chronic form occurring several months later (preceded or not by an aGvHD).

Affecting between 30 to 55% of patients, GvHD is the main complication of transplantation. To halt this autoimmune disease, physicians combine corticosteroids with other immunosuppressive agents. The fact remains that some 30 to 50% of aGvHD gradually become resistant or dependant to these first-line treatments. To date clinicians do not have any standard of treatment approved in Europe for these patients for whom there is a strong unmet medical need. Thus, in Europe, 4,000 children and adults die each year from their aGvHD.

About ELSALYS BIOTECH

ELSALYS BIOTECH is a clinical stage immuno-oncology company which designs and develops a new generation of therapeutic antibodies targeting tumors and their immune and/or vascular microenvironment.

To convert these novel targets into drug candidates, the Company is currently conducting 5 proprietary development programs including inolimomab (LEUKOTAC), an immunotherapy antibody that has recently demonstrated its clinical superiority in Phase 3 and that is closed to market approval in an orphan post-cancer disease with very poor prognosis: steroid-resistant acute Graft-versus-Host Disease.

Founded in 2013, ELSALYS BIOTECH is located in the heart of the European cluster LYON BIOPOLE. Its shareholders are TRANSGENE, SOFIMAC INNOVATION, joined in 2015 by IM EUROPE, a subsidiary of INSTITUT MERIEUX, and CREDIT AGRICOLE CREATION, and in 2018 by LABORATOIRES THEA.

Stay in touch with ElsaLys Biotech and receive directly our press releases by filling our contact form on http://www.elsalysbiotech.com

And follow us on Twitter: @ElsalysBiotech

Contacts

ELSALYS BIOTECHDr. Christine GUILLENCEO and Co-founder+33 (0)4 37 28 73 00guillen@elsalysbiotech.com

PRESSEATCG PARTNERS Marie PUVIEUX (France) +33 (0)6 10 54 36 72Cline VOISIN (UK/US) +33 (0)6 62 12 53 39presse@atcg-partners.com

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LEUKOTAC (inolimomab) is available again in France, following the granting of cohort ATU for the treatment of graft-versus-host disease,...

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Adipose Derived Stem Cell Therapy Market Report 2018-2026includes a comprehensive analysis of the present Market. The report starts with the basic Adipose Derived Stem Cell Therapy industry overview and then goes into each and every detail.

Adipose Derived Stem Cell Therapy Market Report contains in depth information major manufacturers, opportunities, challenges, and industry trends and their impact on the market forecast. Adipose Derived Stem Cell Therapy also provides data about the company and its operations. This report also provides information on the Pricing Strategy, Brand Strategy, Target Client, Distributors/Traders List offered by the company.

Adipose Derived Stem Cell Therapy Market competition by top manufacturers/players, with Adipose Derived Stem Cell Therapy sales volume, Price (USD/Unit), Revenue (Million USD) and Market Share for each manufacturer/player; the top players including: BioRestorative Therapies, Inc., Celltex Therapeutics Corporation, Antria, Inc., Cytori Therapeutics Inc., Intrexon Corporation, Mesoblast Ltd., iXCells Biotechnologies, Pluristem Therapeutics, Inc., Thermo Fisher Scientific, Inc., Tissue Genesis, Inc., Cyagen US Inc., Celprogen, Inc., and Lonza Group, among others.

Description:

Adipose derived stem cells (ADSCs) are stem cells derived from adipocytes, and can differentiate into variety of cell types. ADSCs have multipotency similar to bone marrow mesenchymal stem cells, thus ADSCs substitute for bone marrow as a source of stem cells. Numerous manual and automatic stem cell separation procedures are adopted in order to separate adipose stem cells (ASCs) from adipose tissue. Flow cytometry can also be used to isolate ADSCs from other stem cells within a cell solution.

Adipose derived stem cells are gaining appeal as a new cell source in regenerative medicine therapies as it can be differentiated into a variety of different cell lineages. These stem cells also have anti-apoptotic, anti-inflammatory, pro-angiogenic, immunomodulatory, and anti-scarring properties, which enhances its effectiveness.

Get Request Sample Copy of Research Report @ https://www.coherentmarketinsights.com/insight/request-sample/2357

Important Features that are under offer & key highlights of the report:

1) What all regional segmentation covered? Can the specific country of interest be added?Currently, the research report gives special attention and focus on the following regions:North America (U.S., Canada, Mexico), Europe (Germany, U.K., France, Italy, Russia, Spain etc), South America (Brazil, Argentina etc) & Middle East & Africa (Saudi Arabia, South Africa etc)** One country of specific interest can be included at no added cost. For inclusion of more regional segment quote may vary.

2) What all companies are currently profiled in the report?The report Contain the Major Key Players currently profiled in this market.** List of companies mentioned may vary in the final report subject to Name Change / Merger etc.

3) Can we add or profiled new company as per our need?Yes, we can add or profile new company as per client need in the report. Final confirmation to be provided by the research team depending upon the difficulty of the survey.** Data availability will be confirmed by research in case of a privately held company. Up to 3 players can be added at no added cost.

4) Can the inclusion of additional Segmentation / Market breakdown is possible?Yes, the inclusion of additional segmentation / Market breakdown is possible to subject to data availability and difficulty of the survey. However, a detailed requirement needs to be shared with our research before giving final confirmation to the client.** Depending upon the requirement the deliverable time and quote will vary.

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Adipose Derived Stem Cell Therapy Market Dynamics in the world mainly, the worldwide 2018-2026 Adipose Derived Stem Cell Therapy Market is analyzed across major global regions. CMI also provides customized specific regional and country-level reports for the following areas:

Region Segmentation:

North America (USA, Canada and Mexico)Europe (Germany, France, UK, Russia and Italy)Asia-Pacific (China, Japan, Korea, India and Southeast Asia)South America (Brazil, Argentina, Columbia etc.)Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa)

Key questions answered in the report:

1. What will the market growth rate of Adipose Derived Stem Cell Therapy market in 20262. What are the key factors driving the global Adipose Derived Stem Cell Therapy market3. Who are the key manufacturers in Adipose Derived Stem Cell Therapy market space?4. What are the market opportunities, market risk and market overview of the Adipose Derived Stem Cell Therapy market?5. What are sales, revenue, and price analysis by types and applications of Adipose Derived Stem Cell Therapy market?6. What are sales, revenue, and price analysis by regions of Adipose Derived Stem Cell Therapy industry?

Further in the report, the Adipose Derived Stem Cell Therapy market is examined for Sales, Revenue, Price and Gross Margin. These points are analyzed for companies, types, and regions. In continuation with this data, the sale price is for various types, applications and region is also included. The Adipose Derived Stem Cell Therapy industry consumption for major regions is given. Additionally, type wise and application wise figures are also provided in this report.

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In this study, the years considered to estimate the market size of 2018-2026 Adipose Derived Stem Cell Therapy Market are as follows:History Year: 2015-2017Base Year: 2017Estimated Year: 2018Forecast Year 2018 to 2026

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Adipose Derived Stem Cell Therapy Market Size, Analysis, Competitive Strategies and Forecasts, 2018-2026 - Primo Journal

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What happened

Shares of stocks across the oil and gas industry surged in December. These included shares of independent exploration and production companyNoble Energy(NYSE: NBL), and oilfield services companiesHalliburton(NYSE: HAL) andTechnipFMC(NYSE: FTI). The three companies' shares were up 19.7%, 16.6%, and 13.8%, respectively, according to data provided byS&P Global Market Intelligence.

This largely mirrored the performance of the oil and gas industry as a whole. Industrywide fundsSPDR Oil and Gas Exploration & Production ETFandSPDR Oil and Gas Equipment & Services ETFrose 16.4% and 18.1% for the month, respectively.

When oil prices go up, oil stocks outperform. Image source: Getty Images.

The price of crude oil was the major factor in the companies' December outperformance. After a rocky summer in which prices plunged, oil closed out the year with a sustained three-month rally. An early December OPEC production cut helped keep prices on the risein December. For the month, the international benchmark Brent Crude spot price rose 5.1% to finish the year at $67.77/barrel, while U.S. benchmark WTI Crude was up 9.2% to close out 2019 at $61.14/barrel.

While rising oil prices helped Noble Energy, it also had some good news from its gas operations. On December 31, the company announced that it had begun production at its aptly named Leviathan offshore field in Israel. Leviathan, which the company touts as "the largest natural gas field in the Eastern Mediterranean," is expected to initially produce1.2 billion cubic feet of natural gas per day, with further development projected.

While Noble was ramping up production in the Mediterranean, a slowdown in North American drilling activity was weighing down third quarter earnings at both Halliburton and TechnipFMC. However, Halliburton responded by promising aggressive cost cuts to ease what it sees as continued weakness in the sector.

TechnipFMC's December performance lagged its oilfield services peer Halliburton. Although both companies released poor Q3 2019 earnings reports in October, TechnipFMC's bottom line in particularfell way short of expectations. Management hopesto turn things around by splitting into two companies: an oil and gas engineering and construction company, and a more traditional oilfield services company. Investors may be waiting to buy in before the split occurs.

In the new year, as was true in December, one of the biggest issues that will affect share price growth for these companies -- and the industry generally -- is the price of oil.

In December, the U.S. Energy Information Administration (EIA) projected that Brent Crude spot prices would average $61/barrel in 2020, while WTI Crude would average $55.50/barrel. It cited a forecast of "rising global oil inventories, particularly in the first half of" 2020. Here's how that would compare to averages in recent years:

*Estimate as of 12/10/2019. Data source: U.S. Energy Information Administration. Chart by author.

Those projected average spot prices are lower than they've been for the last two years, and much lower than current prices. However, the EIA's estimate is just that, an estimate, and it could be way off. Nobody knows where oil prices are heading. News of the U.S. drone strike in the Middle East caused oil to briefly spike about 4%, before easing back down. However, the situation remains uncertain and could lead to higher prices -- which would likely benefit these three companies -- or not much of anything.

TechnipFMC hopes to make its split into two pure-play companies in the first half of this year, and until that dust settles, investors bullish on oil prices would be better off considering Halliburton or Noble. Investors who think oil prices are likely to sink probably want to steer clear.

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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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Why These 3 Oil and Gas Stocks Rose by Double Digits In December - Nasdaq

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DetailsCategory: Proteins and PeptidesPublished on Thursday, 09 January 2020 12:06Hits: 265

Anti-tumor effects demonstrated in vivo in preclinical melanoma immuno-oncology model

MENLO PARK, CA, USA I January 08, 2020 I CohBar, Inc. (NASDAQ: CWBR), a clinical stage biotechnology company developing mitochondria based therapeutics (MBTs) to treat chronic diseases and extend healthy lifespan, today announced the discovery of a series of novel mitochondrial peptide analogs with potent in vitro activity as selective inhibitors of C-X-C Chemokine Receptor Type 4 (CXCR4) and with preliminary in vivo efficacy in a mouse model of melanoma, including substantial reduction in tumor growth as compared to control animals. CXCR4 is a key regulatory receptor involved in tumor growth, invasion, angiogenesis, metastasis, and resistance to therapy.

This new discovery offers the potential to develop novel therapeutics for difficult-to-treat cancers, based on peptides encoded in the mitochondrial genome, said Ken Cundy, Ph.D., CohBars Chief Scientific Officer. Inhibition of this key regulatory pathway is potentially applicable to a wide range of cancers, as well as orphan indications where CXCR4 signaling is dysregulated.

Novel peptide analogs of a mitochondrially encoded peptide (MBT5) demonstrated potent and selective inhibition of human CXCR4 receptor in cell-based assays, with IC50 values in the low nanomolar concentration range. In a difficult-to-treat in vivo mouse model of melanoma, the B16F10 syngeneic tumor model, the combination of an analog of MBT5 administered subcutaneously with the chemotherapeutic temozolomide showed enhanced antitumor activity, reducing tumor growth after 11 days by 61% compared to control animals. The reduction in tumor growth produced by the combination exceeded the effect of either temozolomide used as a single agent, which reduced tumor growth by 38% compared to control, or the murine checkpoint inhibitor anti-PD-1 antibody, which had no effect on tumor growth in this model.

CohBar plans to further explore the efficacy of this new family of peptides in additional animal models with the goal of identifying a new clinical development MBT candidate.

These new data further expand our understanding of the broad regulatory influence exerted by mitochondria and the therapeutic potential of analogs of peptides encoded in mitochondrial DNA, said Steve Engle, CohBar CEO. We are just beginning to scratch the surface of this previously untapped field.

CXCR4 is overexpressed in more than 75% of cancers and high levels of the receptor are associated with poor survival prognosis. Inhibition of the CXCR4 receptor has been shown to mobilize immune cells, enhance the effects of chemotherapy and immunotherapy in various cancers, and reduce the development of metastatic tumors by blocking the ability of tumor cells to evade immune surveillance. CXCR4 also regulates the homing and retention of hematopoietic stem cells and malignant cells in the bone marrow.

Further details of these new studies will be available on the CohBar website at http://www.cohbar.com.

About CohBar

CohBar (NASDAQ: CWBR) is a clinical stage biotechnology company focused on the research and development of mitochondria based therapeutics, an emerging class of drugs for the treatment of chronic and age-related diseases. Mitochondria based therapeutics originate from the discovery by CohBars founders of a novel group of naturally occurring mitochondrial-derived peptides within the mitochondrial genome that regulate metabolism and cell death, and whose biological activity declines with age. To date, the company has discovered more than 100 mitochondrial-derived peptides. CohBars efforts focus on the development of these peptides into therapeutics that offer the potential to address a broad range of diseases, including nonalcoholic steatohepatitis (NASH), obesity, fibrotic diseases, cancer, type 2 diabetes, and cardiovascular and neurodegenerative diseases. The companys lead compound, CB4211, is in the phase 1b stage of a phase 1a/1b clinical trial that includes an evaluation of biological activity relevant to NASH and obesity.

For additional company information, please visit http://www.cohbar.com.

SOURCE: CohBar

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CohBar Discovers Novel Peptide Inhibitors of CXCR4, a Key Regulator of Tumor Growth and Metastasis | Proteins and Peptides | News Channels -...

Bone Marrow Stem Cells Comments Off on CohBar Discovers Novel Peptide Inhibitors of CXCR4, a Key Regulator of Tumor Growth and Metastasis | Proteins and Peptides | News Channels -… | January 10th, 2020

When a person's immune system is impaired by a genetic disease, a bone-marrow transplant can be a powerful therapeutic tool, but with a major downside: during the first few months the recipient's defenses against viruses are severely weakened. The slightest infection can lead to a hospital trip.

A still-experimental type of treatment known as T-cell therapy aims to assist during this vulnerable period -- the months during which the body is rebuilding its natural defenses. After two decades of clinical trials, the technology has been refined, and is being used to treat more and more patients, many of them children.

A boy named Johan is one of them.

Today he is a mischievous, smiling toddler with a thick shock of light-brown hair, who never tires, playfully tormenting the family's puppy, Henry. There is no sign of the three-year-long medical and emotional roller-coaster ride he and his family, who live in an affluent Washington suburb, have been on.

The first traumatic surprise came with the results of a pregnancy test: Johan was not planned.

"That was a huge shock. I cried," said his mother, 39-year-old Maren Chamorro.

She had known since childhood that she carried a gene that can be fatal in a child's first 10 years, chronic granulomatous disease (CGD). Her brother died of it at the age of seven. The inexorable laws of genetics meant that Maren had a one in four chance of transmitting it to her child.

For their first children, she and her husband Ricardo had chosen in-vitro fertilization, allowing the embryos to be genetically tested before implantation.

Their twins Thomas and Joanna were born -- both disease-free -- seven and a half years ago. But in Johan's case, a post-birth genetic test quickly confirmed the worst: he had CGD.

After conferring with experts at Children's National Hospital in Washington, the couple took one of the most important decisions of their lives: Johan would receive a bone-marrow transplant, a risky procedure but one that would give him a chance of a cure.

"Obviously, the fact that Maren had lost a sibling at a young age from the disease played a big role," Ricardo confided.

Bone marrow, the spongy tissue inside bones, serves as the body's "factory" for the production of blood cells -- both red and white.

Johan's white blood cells were incapable of fighting off bacteria and fungal infections. A simple bacterial infection, of negligible concern in a healthy child, could spread out of control in his young body.

Luckily, Johan's brother Thomas, six years old at the time, was a perfect match. In April 2018, doctors first "cleansed" Johan's marrow using chemotherapy. They then took a small amount of marrow from Thomas's hip bones using a long, thin needle.

From that sample they extracted "supercells," as Thomas calls them -- stem cells, which they reinjected into Johan's veins. Those cells would eventually settle in his bone marrow -- and begin producing normal white blood cells.

The second step was preventive cell therapy, under an experimental program led by immunologist Michael Keller at Children's National Hospital.

The part of the immune system that protects against bacteria can be rebuilt in only a matter of weeks; but for viruses, the natural process takes at least three months.

From Thomas's blood, doctors extracted specialized white blood cells -- T-cells -- that had already encountered six viruses.

Keller grew them for 10 days in an incubator, creating an army of hundreds of millions of those specialized T-cells. The result: a fluffy white substance contained in a small glass vial.

Those T-cells were then injected into Johan's veins, immediately conferring protection against the six viruses.

"He has his brother's immune system," said Keller, an assistant professor at Children's National.

Johan's mother confirmed as much: today, when Thomas and Johan catch a cold, they have the same symptoms, and for nearly the same amount of time.

"I think it's pretty cool to have immunity from your big brother," Maren Chamorro said.

This therapeutic approach -- boosting the body's immune system using cells from a donor or one's own genetically modified cells -- is known as immunotherapy.

Its main use so far has been against cancer, but Keller hopes it will soon become available against viruses for patients, like Johan, who suffer from depressed immune systems.

The chief obstacles to that happening are the complexity of the process and the costs, which can run to many thousands of dollars. These factors currently restrict the procedure to some 30 medical centers in the United States.

For Johan, a year and a half after his bone marrow transplant, everything points to a complete success.

"It's neat to see him processing things, and especially play outside in the mud," his mother said. "You know, what a gift!"

Her only concern now is the same as any mother would have -- that when her son does fall ill, others in the family might catch the same bug.

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The 'supercells' that cured an infant's grave genetic illness - Japan Today

Bone Marrow Stem Cells Comments Off on The ‘supercells’ that cured an infant’s grave genetic illness – Japan Today | January 10th, 2020

Daily life can be excruciatingly painful and tiring for a West Bridgford mum suffering with a lifelong inherited blood disease.

Oyesola Oni, 37, was born with sickle cell disease and has to have eight pints of blood transfused into her body every six weeks at Nottingham City Hospital.

Sickle cell can cause serious and potentially fatal complications such as organ damage, stroke, death to bone tissue and acute chest syndrome.

People with sickle cell disease produce "unusually shaped" red blood cells that can cause problems because they do not live as long as healthy blood cells. They can also block blood vessels.

If both parents have the gene that affects red blood cells, there's a one in four chance of each child they have being born with the disease.

I mainly get crisis pains in my ribs, legs, hips, my back and my lower abdomen. You cant describe the pain," said Oye, of West Bridgford.

"Its like something stabbing me, at other times its like something crushing my bones. Its excruciating."

The only cure for sickle cell disease is astem cell or bone marrow transplant,but they're not done very often because of the risks involved.

Her story comes as the NHS launch a call for more men to donate blood in 2020 because of a "serious imbalance" in the gender of new donors.

The mum has regular red cell transfusions for sickle cell disease after several years of her condition getting worse.

Having a secure supply of blood is particularly important for people like Mrs Oni, who receive many transfusions over their lives.

The mum to daughter Ade, 12, who does not have sickle cell, said people who donate the blood that she receives are "heroes that dont wear capes".

They give blood to someone they dont know its amazing, very selfless. Its an extraordinary thing to do and I hope more men start donating blood in the New Year," she added.

"Having the transfusions gives me so much more energy, keeps me out of hospital and allows me to spend more time with my family."

The mum said she hopes to return to work as in customer services thanks to the continued transfusions.

During 2019, 43 percent of the new donors at Nottingham Donor Centre were men.

Until the end of November, 1,203 women started donating blood in Nottingham but only 898 men.

The NHS said this is a concern because men have higher iron levels and only mens blood can be used for some transfusions and products.

Without more men starting to give blood, blood stocks will come under increasing pressure in future years, the NHS has warned.

Mike Stredder, the head of donor recruitment for NHS Blood and Transplant, added: All our donors are amazing. But we need more men to start donating blood in Nottingham during the New Year.

"Mens blood can be used in extraordinary, lifesaving ways, but we dont have enough new male donors coming forward.

"This is not about recruiting as many donors as possible its about getting the right gender mix.

If you cant find an appointment right away dont worry your blood will do extraordinary things if you donate in a few weeks instead."

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Mum's 'excruciating' battle with lifelong disease that requires regular blood transfusions - Nottinghamshire Live

Bone Marrow Stem Cells Comments Off on Mum’s ‘excruciating’ battle with lifelong disease that requires regular blood transfusions – Nottinghamshire Live | January 10th, 2020

MENLO PARK, Calif., Jan. 08, 2020 (GLOBE NEWSWIRE) -- CohBar, Inc. (NASDAQ: CWBR), a clinical stage biotechnology company developing mitochondria based therapeutics (MBTs) to treat chronic diseases and extend healthy lifespan, today announced the discovery of a series of novel mitochondrial peptide analogs with potent in vitro activity as selective inhibitors of C-X-C Chemokine Receptor Type 4 (CXCR4) and with preliminary in vivo efficacy in a mouse model of melanoma, including substantial reduction in tumor growth as compared to control animals. CXCR4 is a key regulatory receptor involved in tumor growth, invasion, angiogenesis, metastasis, and resistance to therapy.

This new discovery offers the potential to develop novel therapeutics for difficult-to-treat cancers, based on peptides encoded in the mitochondrial genome, said Ken Cundy, Ph.D., CohBars Chief Scientific Officer. Inhibition of this key regulatory pathway is potentially applicable to a wide range of cancers, as well as orphan indications where CXCR4 signaling is dysregulated.

Novel peptide analogs of a mitochondrially encoded peptide (MBT5) demonstrated potent and selective inhibition of human CXCR4 receptor in cell-based assays, with IC50 values in the low nanomolar concentration range. In a difficult-to-treat in vivo mouse model of melanoma, the B16F10 syngeneic tumor model, the combination of an analog of MBT5 administered subcutaneously with the chemotherapeutic temozolomide showed enhanced antitumor activity, reducing tumor growth after 11 days by 61% compared to control animals. The reduction in tumor growth produced by the combination exceeded the effect of either temozolomide used as a single agent, which reduced tumor growth by 38% compared to control, or the murine checkpoint inhibitor anti-PD-1 antibody, which had no effect on tumor growth in this model.

CohBar plans to further explore the efficacy of this new family of peptides in additional animal models with the goal of identifying a new clinical development MBT candidate.

These new data further expand our understanding of the broad regulatory influence exerted by mitochondria and the therapeutic potential of analogs of peptides encoded in mitochondrial DNA, said Steve Engle, CohBar CEO. We are just beginning to scratch the surface of this previously untapped field.

CXCR4 is overexpressed in more than 75% of cancers and high levels of the receptor are associated with poor survival prognosis. Inhibition of the CXCR4 receptor has been shown to mobilize immune cells, enhance the effects of chemotherapy and immunotherapy in various cancers, and reduce the development of metastatic tumors by blocking the ability of tumor cells to evade immune surveillance. CXCR4 also regulates the homing and retention of hematopoietic stem cells and malignant cells in the bone marrow.

Further details of these new studies will be available on the CohBar website at http://www.cohbar.com.

About CohBar

CohBar (NASDAQ: CWBR) is a clinical stage biotechnology company focused on the research and development of mitochondria based therapeutics, an emerging class of drugs for the treatment of chronic and age-related diseases. Mitochondria based therapeutics originate from the discovery by CohBars founders of a novel group of naturally occurring mitochondrial-derived peptides within the mitochondrial genome that regulate metabolism and cell death, and whose biological activity declines with age. To date, the company has discovered more than 100 mitochondrial-derived peptides. CohBars efforts focus on the development of these peptides into therapeutics that offer the potential to address a broad range of diseases, including nonalcoholic steatohepatitis (NASH), obesity, fibrotic diseases, cancer, type 2 diabetes, and cardiovascular and neurodegenerative diseases. The companys lead compound, CB4211, is in the phase 1b stage of a phase 1a/1b clinical trial that includes an evaluation of biological activity relevant to NASH and obesity.

For additional company information, please visit http://www.cohbar.com.

Forward-Looking Statements

This news release contains forward-looking statements which are not historical facts within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are based only on our current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, projections, anticipated events and other future conditions. In some cases you can identify these statements by forward-looking words such as believe, may, will, estimate, continue, anticipate, intend, could, should, would, project, plan, expect, goal, seek, future, likely or the negative or plural of these words or similar expressions. Examples of such forward-looking statements including but not limited to statements regarding the ability of mitochondrial peptide analogs to reduce tumor growth in mice; anticipated outcomes of research and clinical trials for our mitochondria based therapeutic (MBT) candidates; expectations regarding the growth of MBTs as a significant future class of drug products; and statements regarding anticipated therapeutic properties and potential of our mitochondrial peptide analogs and MBTs. You are cautioned that such statements are not guarantees of future performance and that actual results or developments may differ materially from those set forth in these forward looking statements. Factors that could cause actual results to differ materially from these forward-looking statements include: our ability to successfully advance drug discovery and development programs, including the delay or termination of ongoing clinical trials; our possible inability to mitigate the prevalence and/or persistence of the injection site reactions, receipt of unfavorable feedback from regulators regarding the safety or tolerability of CB4211 or the possibility of other developments affecting the viability of CB4211 as a clinical candidate or its commercial potential; results that are different from earlier data results including less favorable than and that may not support further clinical development; our ability to raise additional capital when necessary to continue our operations; our ability to recruit and retain key management and scientific personnel; and our ability to establish and maintain partnerships with corporate and industry partners. Additional assumptions, risks and uncertainties are described in detail in our registration statements, reports and other filings with the Securities and Exchange Commission and applicable Canadian securities regulators, which are available on our website, and at http://www.sec.gov or http://www.sedar.com.

You are cautioned that such statements are not guarantees of future performance and that our actual results may differ materially from those set forth in the forward-looking statements. The forward-looking statements and other information contained in this news release are made as of the date hereof and CohBar does not undertake any obligation to update publicly or revise any forward-looking statements or information, whether as a result of new information, future events or otherwise, unless so required by applicable securities laws. Nothing herein shall constitute an offer to sell or the solicitation of an offer to buy any securities.

Investor and Media Contact:Jordyn TaraziDirector of Investor RelationsCohBar, Inc.(650) 445-4441 Jordyn.tarazi@cohbar.com

Joyce AllaireLifeSci Advisors, LLC jallaire@lifesciadvisors.com

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CohBar Discovers Novel Peptide Inhibitors of CXCR4, a Key Regulator of Tumor Growth and Metastasis - Associated Press

Bone Marrow Stem Cells Comments Off on CohBar Discovers Novel Peptide Inhibitors of CXCR4, a Key Regulator of Tumor Growth and Metastasis – Associated Press | January 8th, 2020

CALQUENCE (acalabrutinib) is now available for adult patients with previously untreated and relapsed/refractory chronic lymphocytic leukemia

MISSISSAUGA, ON, Jan. 8, 2020 /CNW/ - AstraZeneca Canada today announced that Health Canada has approved Calquence (acalabrutinib), an oral Bruton's tyrosine kinase (BTK) inhibitor, for the treatment of adult patients with chronic lymphocytic leukemia (CLL), as monotherapy or in combination with obinutuzumab in the first-line setting, and as monotherapy for relapsed/refractory (r/r) disease.1

CLL is the most common type of leukemia in adults, accounting for 44 per cent of all cases in Canada.2 Morethan 2,200 people in Canada are diagnosed with the disease each year and more than 600 will die from it.3,4 Despite advancements in the treatment of CLL, there is still no cure for the disease and even after successful initial treatment, some patients may relapse, leaving them in need of further innovation.

"CLL is most often diagnosed when patients are more than 60 years old, at a time when they are already dealing with other health conditions related to aging and are trying to maintain the best quality of life," says Antonella Rizza, CEO of Lymphoma Canada. "Today's announcement offers Canadians living with CLL an important new option for this incurable but treatable disease."'

The Canadian approval was granted under Project Orbis, a new international health authority collaboration which provides a framework for simultaneous submission and review of oncology products among international partners.5Under this collaboration, Health Canada, the U.S. FDA, and the Australian Therapeutic Goods Administration (TGA) collectively reviewed the application for Calquence, making it the second treatment approved as part of the program and the first in hematology.

"In the last several years, we've been moving away from traditional chemotherapies to more targeted therapies for CLL." said Dr. Carolyn Owen, Alberta Health Services, Calgary. "Health Canada's approval of acalabrutinib provides a new effective and well tolerated treatment option for CLL patients and improves their treatment options."

The Health Canada approval of Calquence was based on positive interim data from two Phase III clinical trials, ELEVATE-TN and ASCEND.6,7The ELEVATE-TN trial evaluated the safety and efficacy of Calquence in combination with obinutuzumab, a CD20 monoclonal antibody, or Calquence alone versus chlorambucil, a chemotherapy, in combination with obinutuzumab in previously untreated patients with CLL. The ASCEND trial evaluated the efficacy of Calquence in previously treated patients with CLL.Together, the trials showed that Calquence in combination with obinutuzumab or as a monotherapy significantly reduced the relative risk of disease progression or death. Across both trials, the safety and tolerability of Calquence were consistent with its established profile.1

About chronic lymphocytic leukemia (CLL)Chronic lymphocytic leukemia is the most common type of leukemia in adults, which begins in the bone marrow, and progresses slowly.8 In CLL, too many blood stem cells in the bone marrow become abnormal lymphocytes and these abnormal cells have difficulty fighting infections.9 As the number of abnormal cells grows there is less room for healthy white blood cells, red blood cells and platelets.9This could result in anaemia, infection and bleeding.9B-cell receptor signalling through BTK is one of the essential growth pathways for CLL. Many people with CLL do not have any symptoms upon diagnosis, and the disease is often found in blood tests for unrelated health problems.10

AboutCalquenceCalquence(acalabrutinib; previously known as ACP-196) is a selective inhibitor of Bruton's tyrosine kinase (BTK).1Calquencebinds covalently to BTK, thereby inhibiting its activity, and has demonstrated this with minimal interactions with other immune cells in pre-clinical studies.1,6,7In B cells, BTK signaling results in activation of pathways necessary for B cell proliferation, trafficking, chemotaxis and adhesion.1 The recommended dose ofCalquenceis one 100mg capsule taken orally twice daily (approximately 12 hours apart), until disease progression or unacceptable toxicity.1Calquencemay be taken with or without food.1

About AstraZenecaAstraZeneca is a global, innovation-driven biopharmaceutical business with a primary focus on the discovery, development and commercialization of primary and specialty care medicines that transform lives. Our primary focus is on three important areas of healthcare: Cardiovascular and Metabolic disease; Oncology; and Respiratory, Inflammation and Autoimmunity. AstraZeneca operates in more than 100 countries and its innovative medicines are used by millions of patients worldwide. In Canada, we employ more than 675 employees across the country and our headquarters are located in Mississauga, Ontario. For more information, please visit the company's website at http://www.astrazeneca.ca.

References

SOURCE AstraZeneca Canada Inc.

For further information: AstraZeneca Corporate Communications, [emailprotected]; Hibaq Ali, Weber Shandwick Canada, [emailprotected] / tel: 416-642-7915

http://www.astrazeneca.ca

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New Treatment Approved in Canada for Most Common Type of Leukemia - Canada NewsWire

Bone Marrow Stem Cells Comments Off on New Treatment Approved in Canada for Most Common Type of Leukemia – Canada NewsWire | January 8th, 2020

(MENAFN - Fior Markets)

Spearheaded by researchers at Baylor College of Medicine divulges a contemporary mechanism that donates to adult bone conservation and restores and unfurls the possibility of advancing the therapeutic plan of action for enhancing bone healing.

Corresponding author Dr. Dongsu Park professor of molecular and human genetics said that adult bone repairs depend on the setting off of bone stem cells which yet remains deficiently distinguished. Bone stem cells have been discovered both in the bone marrow interior of the bone and also in the periosteum the exterior layer of the tissue that wraps the bone. Former studies have portrayed that these two communities of stem cell albeit they apportion various characteristics also have distinctive functions and particular regulatory processes.

Of the two periosteal steam cells are the minimalistcomprehended. It is known that they constitute a heterogeneous population ofcells that can bestow to bone density, molding and rupture restoration,however, scientists had not been able to discern between varied subtypes of thebone stem cell to scrutinize how their varied purposes are controlled.

In the present study Park and his colleagues advanced aprocedure to recognize varied subpopulations of periosteal stem cells expoundtheir benefaction to bone fracture restoration in animate mouse models andrecognize particular components that control their migration and multiplicationunder psychological circumstances.

The researchers found particular trademarks for periosteal stem cells in mouse models. The trademarks recognized a definite subset of stem cells that donates to long-lasting adult bone resurrection.

MENAFN07012020007010660ID1099519082

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Contemporary Bone Alleviates Mechanisms Have Prospective Therapeutic Applications - MENAFN.COM

Bone Marrow Stem Cells Comments Off on Contemporary Bone Alleviates Mechanisms Have Prospective Therapeutic Applications – MENAFN.COM | January 7th, 2020

Fourteen charities based in London have won a 1,000 festive boost thanks to nominations from the public.

The charities were nominated to win a share of 120,000 as part of specialist insurer Ecclesiasticals annual 12 days of giving Christmas campaign.

Anthony Nolan, which helps to save lives by matching individuals willing to donate their blood stem cells or bone marrow to people with blood cancer and blood disorders, and Shakespeare Schools Foundation, which helps thousands of young people from across the UK become better at teamwork, more confident and more ambitious (see notes for full list1), are among the local charities set to benefit from the money, following overwhelming public support in the area.

More than 120,000 people around the UK nominated a cause close to their heart, with over 5,000 charitable causes up and down the country receiving votes. The 120 winning charities were picked at random from those nominated.

The full list of the 120 charity winners is available to view online at http://www.ecclesiastical.com/12days

Thanking supporters in London, Mark Hews, group chief executive at Ecclesiastical, said: Here at Ecclesiastical, our core purpose is to contribute to the greater good of society, so charitable giving is at the heart of our business. We know that 1,000 can make a huge difference to the incredible work that charities do and were looking forward to seeing how this festive financial boost will change lives for the better.

Last year, Ecclesiastical launched its second Impact Report to celebrate some of the many good causes it has helped.

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London charities scoop share of 120000 festive financial boost - London Post

Bone Marrow Stem Cells Comments Off on London charities scoop share of 120000 festive financial boost – London Post | January 7th, 2020

SUZHOU, Chinaand SHANGHAI, Jan. 7, 2020 /PRNewswire/ -- Gracell Biotechnologies Co., Ltd. ("Gracell"), a clinical-stage immune cell therapy company, today announced the initiation of an investigational study of GC027, the first product candidate developed using TruUCAR to treat relapsed or refractory (R/R) T-cell malignancies.

T-cell acute lymphoblastic leukemia or T-ALL is an aggressive form of ALL, which affects white blood cells and the bone marrows ability to generate healthy blood cells. About 15-20% of people with ALL have T-ALL. While T-ALL is treatable by chemotherapy and stem cell transplant, around 75% of patients will relapse within two years[1]. T-cell lymphoblastic lymphoma (T-LBL) is another devastating T-cell malignancies. For patients who develop R/R T-ALL or T-LBL, there are few options for treatment.

Autologus CAR-T therapies rely on patients' own T cells, which have been affected by prior therapies; thus, cell quality as well as efficacy remains questionable. Allogenic CAR-T therapies made of healthy donors' T cells would be characterized as being of consistently good quality with the potential to improve efficacy. Unlike autologous CAR-T cells, allogeneic CAR-T cells can be made as off-the-shelf product which means patients do not have to wait for lengthy production time. Furthermore, the cost of production can be significantly lower. Allogenic CAR-T therapies also provide a vital treatment option for patients with viral infections and/or other conditions prohibiting access to autologous cell therapies.

TruUCARbased GC027 is designed to meet the above unmet needs. Its cells are made of T cells from healthy donors, genetically edited and inserted with chimeric antigen receptor (CAR) ex vivo, which can specifically bind to and eliminate target T malignant cells. Different from industry leaders' off-the-shelf CAR-T design, Gracell's proprietary and patented TruUCAR technology requires no co-administration of anti-CD52, a cytotoxic agent for ablating cancerous cells while inducing long term immune depletion in the patient.Instead, GC027 utilizes CRISPRgenome editing strategy that is expected to avoid graft-versus-host disease (GvHD) as well as graft rejection caused by the patients' immune system.

The prudent preclinical studies provide substantial evidence to trigger GC027 moving into a non-IND(investigational new drug)clinical trial to evaluate the safety, pharmacokinetics and pharmacodynamics of GC027 therapy in patients suffering from relapsed and refractory T lymphocyte malignancies.

TruUCAR is another technological breakthrough developed by Gracell following the recent announcement of FasTCAR technology and products. It enables producing off-the-shelf CAR-T cells from healthy MHC (major histocompatibility complex) mismatched donors with a large number of doses readily to be dispatched to patients in need.

"Launch of the investigational GC027 study as the first-of-its-kind therapy marks another significant milestone for Gracell," said Dr. William CAO, Founder and CEO of Gracell. "Once the concept is well-proved with solid evidence for safety and efficacy, we will immediately deploy development of a series of TruUCAR products for other medical unmet needs, including B cell malignancies."

About GC027

GC027 is an investigational, off-the-shelf CAR-T cell therapy for T cell malignancies, derived from healthy donors. The use of healthy donor's cells are preferential to a patient's own with potential to improve efficacy, reduce production time, and lower cost of goods.

About T-ALL

T lymphoblastic leukemia (T-ALL) is an aggressive form of T cell malignancies, with a diffuse invasion of bone marrow and peripheral blood. In 2015, ALL affected around 876,000 people globally and resulted in 110,000 deaths worldwide. T-ALL compromises about 15%-20% children and adults[1].Current standard therapies for T-ALL are chemotherapies and stem cell transplantation. A large portion of these patients will experience relapse within two years following treatment by conventional therapies.

About T-LBL

T lymphoblastic lymphoma (T-LBL) is an aggressive form of T cell malignancies, with rare lymphoproliferative neoplasm of mature T cells caused by infection with the retrovirus human T lymphotropic virus. T-LBL compromises about 2% of adult non-Hodgkin's lymphoma (NHL) and 30% of pediatric NHL patients[2]. Five-year overall survival is only 14% in adults.Although first-line treatment using cytotoxic combination chemotherapy can achieve 70% ORR, nearly 90% of patients relapse, often within months of completing chemotherapy.

About Gracell

Gracell Biotechnologies Co., Ltd. ("Gracell") is a clinical-stage biopharma company, committed to developing highly reliable and affordable cell gene therapies for cancer. Gracell is dedicated to resolving the remaining challenges in CAR-T, such as high production costs, lengthy manufacturing process, lack of off-the-shelf products, and inefficacy against solid tumors. Led by a group of world-class scientists, Gracell is advancing FasTCAR, TruUCAR (off-the-shelf CAR), Dual CAR and Enhanced CAR-T cell therapies for leukemia, lymphoma, myeloma, and solid tumors.

CONTACT:

[1]Pediatric hematologic Malignancies: T-cell acute lymphoblastic Leukemia, Hematology 2016

[2]Clinical Review: Adult T-cell Leukemia/lymphoma, Journal ofOncology Practice 2017

SOURCE Gracell

http://www.gracellbio.com

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Gracell Initiates Investigational Study of the Technological Breakthrough TruUCAR Therapy for Relapsed or Refractory T-cell Malignancies - PRNewswire

Bone Marrow Stem Cells Comments Off on Gracell Initiates Investigational Study of the Technological Breakthrough TruUCAR Therapy for Relapsed or Refractory T-cell Malignancies – PRNewswire | January 7th, 2020

NEW YORK, Jan. 07, 2020 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, announced today that Chaim Lebovits, President and Chief Executive Officer, will provide a corporate overview at the 2020 Biotech Showcase, being held on January 13-15, 2020 at the Hilton San Francisco Union Square in San Francisco, California.

Mr. Lebovits will also present at the 3rd Annual Neuroscience Innovation Forum, taking place on January 12, 2020, at the Marines Memorial Club in San Francisco. Additionally, Ralph Kern M.D., MHSc, BrainStorms Chief Operating Officer and Chief Medical Officer, will participate on aRare & Orphan Diseases Panel.

Meetings

BrainStorms senior management will also be hosting institutional investor and partnering meetings at the 2020 Biotech Showcase conference (https://goo.gl/SGFm62). Please use the Investor contact information provided below to schedule a meeting.

About NurOwn

NurOwn (autologous MSC-NTF cells) represent a promising investigational approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. NurOwn is currently being evaluated in a Phase 3 ALS randomized placebo-controlled trial and in a Phase 2 open-label multicenter trial in Progressive MS.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. For more information, visit BrainStorm's website at http://www.brainstorm-cell.com.

Safe-Harbor Statement

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

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BrainStorm Cell Therapeutics to Present at the 2020 Biotech Showcase and 3rd Annual Neuroscience Innovation Forum at JPM Week - Yahoo Finance

Bone Marrow Stem Cells Comments Off on BrainStorm Cell Therapeutics to Present at the 2020 Biotech Showcase and 3rd Annual Neuroscience Innovation Forum at JPM Week – Yahoo Finance | January 7th, 2020

Cynata Therapeutics (CYP) has received a research and development tax incentive refund of $1,891,795 for the 2018-2019 financial year.

This tax incentive refund increases the company's cash position which stood at $9.2 million at the end of the September quarter.

It also enables further resources to be invested towards Cynata's phase 2 clinical trial programs for the critical limb ischemia (CLI) (reduced blood flow) and osteoarthritis products.

This will be alongside the anticipated phase 2 trial for CYP-001 in graft-versus-host disease which will be conducted by Fujifilm.

CLI is an advanced stage of peripheral artery disease which is the narrowing of the arteries in the limbs, typically in the lower legs.

It results from severely impaired blood flow which can cause pain, tissue damage, and gangrene.

Around 25 per cent of CLI patients who are unable to undergo surgery to remove the affected area, often an amputation, will die within a year of diagnosis.

Cynata' Cymerus mesenchymal stem cells (MSCs) have been successfully tested in a mouse model of CLI.

Muscles on the ischaemic leg were injected with Cymerus MSCs or a control.

Over a four-week follow-up period, the return of blood flow was measured and in animals treated with Cymerus MSCs blood flow in the injured limb was significantly higher at every point compared to the control.

MSCs are an adult stem cell found in a wide range of human tissues including bone marrow, fat tissue and placenta.

They are multi-potent which means they can produce more than one type of cell, for example they can differentiate into cartilage cells, bone cells and fat cells.

MSCs have been shown to ease regeneration and effects on the immune system without relying on engraftment (when the transplanted cells start to grow and make healthy cells).

The research and development tax incentive is an important Australian Government program that encourages companies to engage in research and development benefiting Australia by providing a tax offset for eligible activities.

Cynata's share price is up a steady 4.82 per cent with shares trading for $1.20 apiece at 3:29 pm AEDT.

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Cynata Therapeutics (ASX:CYP) receives R&D tax incentive refund of more than $1.8M - The Market Herald

Bone Marrow Stem Cells Comments Off on Cynata Therapeutics (ASX:CYP) receives R&D tax incentive refund of more than $1.8M – The Market Herald | January 7th, 2020

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MCLEAN, United States (AFP) When a person's immune system is impaired by a genetic disease a bone marrow transplant can be a powerful therapeutic tool, but with a major downside during the first few months the recipient's defences against viruses are severely weakened. The slightest infection can lead to a hospital trip.

A still-experimental type of treatment known as T-cell therapy aims to assist during this vulnerable period the months during which the body is rebuilding its natural defences. After two decades of clinical trials, the technology has been refined and is being used to treat more and more patients, many of them children.

A boy named Johan is one of them.

Today he is a mischievous, smiling toddler, with a thick shock of light-brown hair, who never tires, playfully tormenting the family's puppy, Henry.

There is no sign of the three-year-long medical and emotional roller coaster ride he and his family, who live in an affluent Washington suburb, have been on.

The first traumatic surprise came with the results of a pregnancy test Johan was not planned.

That was a huge shock. I cried, said his mother, 39-year-old Maren Chamorro.

Risky procedure

She had known since childhood that she carried a gene that can be fatal in a child's first 10 years, chronic granulomatous disease (CGD).

Her brother died of it at the age of seven. The inexorable laws of genetics meant that Maren had a one in four chance of transmitting it to her child.

For their first children, she and her husband Ricardo had chosen invitro fertilisation, allowing the embryos to be genetically tested before implantation.

Their twins Thomas and Joanna were born both disease-free seven and a half years ago.

But in Johan's case, a post-birth genetic test quickly confirmed the worst: He had CGD.

After conferring with experts at Children's National Hospital in Washington, the couple took one of the most important decisions of their lives, Johan would receive a bone marrow transplant a risky procedure but one that would give him a chance of a cure.

Obviously, the fact that Maren had lost a sibling at a young age from the disease played a big role, Ricardo confided.

Bone marrow, the spongy tissue inside bones, serves as the body's factory for the production of blood cells both red and white.

His brother's immune system

Johan's white blood cells were incapable of fighting off bacteria and fungal infections. A simple bacterial infection, of negligible concern in a healthy child, could spread out of control in his young body.

Luckily, Johan's brother Thomas, six years old at the time, was a perfect match. In April 2018, doctors first cleansed Johan's marrow using chemotherapy. They then took a small amount of marrow from Thomas's hip bones using a long, thin needle.

From that sample they extracted supercells, as Thomas calls them stem cells, which they reinjected into Johan's veins. Those cells would eventually settle in his bone marrow and begin producing normal white blood cells.

The second step was preventive cell therapy, under an experimental programme led by immunologist Michael Keller at Children's National Hospital.

The part of the immune system that protects against bacteria can be rebuilt in only a matter of weeks; but for viruses, the natural process takes at least three months.

Hurdles remain

From Thomas's blood, doctors extracted specialised white blood cells T-cells that had already encountered six viruses.

Keller grew them for 10 days in an incubator, creating an army of hundreds of millions of those specialised T-cells. The result: A fluffy white substance contained in a small glass vial.

Those T-cells were then injected into Johan's veins, immediately conferring protection against the six viruses.

He has his brother's immune system, said Keller, an assistant professor at Children's National.

Johan's mother confirmed as much: Today, when Thomas and Johan catch a cold they have the same symptoms, and for nearly the same amount of time.

I think it's pretty cool to have immunity from your big brother, Maren Chamorro said.

This therapeutic approach boosting the body's immune system using cells from a donor or one's own genetically modified cells is known as immunotherapy.

Its main use so far has been against cancer, but Keller hopes it will soon become available against viruses for patients, like Johan, who suffer from depressed immune systems.

The chief obstacles to that happening are the complexity of the process and the costs, which can run to many thousands of dollars. These factors currently restrict the procedure to some 30 medical centres in the United States.

For Johan, a year and a half after his bone marrow transplant, everything points to a complete success.

It's neat to see him processing things, and especially play outside in the mud, his mother said.

You know, what a gift!

Her only concern now is the same as any mother would have that when her son does fall ill, others in the family might catch the same bug.

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The supercells' that cured an infants genetic illness - Jamaica Observer

Bone Marrow Stem Cells Comments Off on The supercells’ that cured an infants genetic illness – Jamaica Observer | January 5th, 2020

There was a head-scratching moment when Martin Swales answered his front door and a priest handed him a letter.

The mystery was quickly solved. It contained a thank you note from someone whose life Martin had saved.

He knew his bone marrow had been given to someone called Jan and imagined it was a woman in Britain.

In fact the recipient was dad Jan Zemek 4,500 miles away in the US.

And Martins gift of life has led to an extraordinary 30-year bond between the pair, who are like blood brothers.

Jan named his second daughter Martina in honour of his hero and Martin is godfather to his third girl.

Retired welder Martin, 58, of Guisborough, North Yorks, said: Donating bone marrow didnt just save Jans life, it changed mine as well.

The first time I met Jan, I put my arms around him and he hugged me back.

It felt natural, like I was welcoming my brother. It feels like our two families have become one.

They each have three grown-up children and have visited each other for baptisms, graduations, and weddings.

Martin recently went to Switzerland, where Jan lives with his family, to celebrate 30 years since the transplant and present his blood brother with a Walk of Fame plaque.

It includes the touching message: Stood strong, fought hard, and won. You are a survivor.

The mens amazing and heart-warming story dates from 1986 when Martin joined the Anthony Nolan stem cell register after an appeal to save two girls living in the North East.

He was not a match for the girls but in 1989 was called by the register because he could be for Jan.

Martin said: It was quite a shock because Id pretty much forgotten about the register. They told me I was a possible match for someone and what was involved. I said yes straight away. I wanted to help if I could.

Despite the discomfort, Martin gave bone marrow from his hip at a clinic in Harley Street that August. Doctors extracted it from inside his hip using a long needle. Today most donations are no more invasive than giving blood.

Martin spent two nights in hospital. He said: It doesnt take long but at the time I was suffering from sciatica so I think I found it a bit more painful than most. It was an uncomfortable journey home on the train.Anthony Nolan covered the cost of the trip.

Jan, a 27-year-old dad, was diagnosed with leukaemia in 1987. Initially doctors kept the news from him as no treatment was available in the Czech Republic, where he lived.

Jan said: I was diagnosed one year after the Chernobyl tragedy, weve never known if that radiation was to blame for my cancer. I suddenly grew very tired, nobody knew the reason.

I didnt know how sick I was because the doctors wouldnt tell me.

My wife, who was then my girlfriend, went to the same doctors and they told her, Dont marry this guy, dont have children with him. He is going to die in two years.

But Radka ignored their warning and insisted on marrying Jan in 1987.

His only hope was a bone marrow transplant. Weeks later he left for the US with his dad, who planned to be his donor.

Jan said: A few months earlier, I read in the paper the opera singer Jos Carreras was diagnosed with a similar blood disease and was going to the same US centre for a transplant.

They arrived with less than 40 in their pockets and discovered a transplant from his dad would give Jan only a 15 per cent chance of survival.

Instead doctors advised them to find a donor. It took two years and 10,000 to test potential donors before they found a perfect match in Martin.

By then Jan and Radka had become parents to their first daughter, Jana.

Jan needed to raise more than 100,000 to fund the transplant.

He said: It was such a huge amount of money to raise but when you are dying you have no choice.

There were 12 rival local radio stations but they all got together to run a joint appeal, which they broadcast at the same time. It was incredible.

Jan did a sponsored run, gave talks about his ordeal to church congregations to request donations, and wrote to celebrities, especially those with links to the Czech Republic.

Donald Trump s ex-wife Ivana gave 1,000, as did One Flew Over the Cuckoos Nest director Milos Forman. Jan said: The response was crazy. So many people donated 20 dollars or 50.

Martins bone marrow was flown to the Fred Hutchinson Cancer Research Center in Seattle, where Jan was waiting in an isolation room.

He had been blasted with chemo and radiotherapy so his immune system would not attack Martins transplanted cells.

Normally, under strict anonymity rules to protect donor and recipient, Martin and Jan would have been unable to contact each other for years.

But a priest from the North East of England working at the hospital recognised Martins address when the bag of bone marrow arrived.

He offered to take a photo of Jan, a thank you letter, and a Czech garnet stone to Martin when he returned home in 1990.

Martin said: I was stunned. I had no idea my bone marrow had travelled so far. Knowing Id helped a young father, just like me, brought home how important it was and how easily it could have been me waiting for a stranger to save my life.

I wrote straight back. The priest also brought a letter from a couple whose daughter was in the same hospital.

Her transplant didnt work. Sadly she died, but they wrote to thank me for saving Jan. Responding to them was much harder. How do you find the right words?

Martin and Jan kept in touch. When Jans second daughter was born in 1991, he and Radka named her after Martin.

Jan said: How do you repay someone who saved your life? Naming our daughter after Martin was our way of showing him we would never forget what he did for us.

Hes not just the man who saved my life. He is a nice guy. Thats why were so close.

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Jan, 59, and his family moved to Switzerland, where he landed a job with a sports marketing firm that works with World Athletics.

In 1992 his job brought him to Crystal Palace in South London and he spent a few days with Martin and family.

Jans youngest daughter Michaela was born in 1995 and he invited Martin and his family to Switzerland for the baptism and asked him to be godfather.

The two families continued to visit each other and holidayed together in the Czech capital Prague. When Jans eldest, Jana, was studying at Newcastle University, she regularly spent weekends with Martin and his wife Tracey.

Martin said: It meant so much to visit Jan for the 30 anniversary of his transplant earlier this year.

"They showed us the sights and we went up the mountains. It was brilliant. I could never have imagined this when I joined the stem cell register all those years ago.

He added: I hope Martin and I will be able to celebrate another anniversary together in ten years.

The Anthony Nolan register matches potential donors to patients needing stem cell transplants and does vital research. To join, donate or find out more, see anthonynolan.org .

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Bone marrow donor's amazing 30 year bond with man he saved - Mirror Online

Bone Marrow Stem Cells Comments Off on Bone marrow donor’s amazing 30 year bond with man he saved – Mirror Online | January 5th, 2020

IN the summer of 2019, a mother in Nashville, Tennessee in the United States, with a seemingly incurable genetic disorder finally found an end to her suffering by editing her genome.

Victoria Grays recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research gene therapy.

I have hoped for a cure since I was about 11, the 34-year-old said.

Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency.

Over several weeks, Grays blood was drawn so that doctors could get to the cause of her illness stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 pronounced Crisper a new tool informally known as a molecular scissors.

The genetically-edited cells were transfused back into Grays veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary, but theoretically, she has been cured.

This is one patient. This is early results. We need to see how it works out in other patients, said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

But these results are really exciting.

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease beta thalassemia.

She had previously needed 16 blood transfusions per year. Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified. But Crispr, invented in 2012, made gene editing more widely accessible.

It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

Its all developing very quickly, said French geneticist Emmanuelle Charpentier, one of Crisprs inventors and the co-founder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Gene cures

Crispr was the latest breakthrough in a year of great strides in gene therapy, a medical adventure that started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practising the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not, such as making normal red blood cells in Grays case or making tumour-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union.

They join several other gene therapies bringing the total to eight approved in recent years to treat certain cancers and an inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

Twenty-five, 30 years, thats the time it had to take, he said. It took a generation for gene therapy to become a reality. Now, its only going to go faster.

Just outside Washington, at the US National Institutes of Health (NIH), researchers are also celebrating a breakthrough period.

We have hit an inflection point, said US NIHs associate director for science policy Carrie Wolinetz.

These therapies are exorbitantly expensive, however, costing up to US$2 million (RM8.18 million) meaning patients face grueling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion and fighting a general infection.

You cannot do this in a community hospital close to home, said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to Massachusetts Institute of Technology (MIT) researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

In this Oct 10, 2018, photo, He speaks during an interview at his laboratory in Shenzhen, China. The scientist was recently sentenced to three years in prison for practicing medicine illegally and fined 3 million yuan (RM1.76 million). AP

Bioterrorism potential

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who dont necessarily share the medical ethics of Western medicine.

In 2018 in China, scientist He Jiankui triggered an international scandal and his excommunication from the scientific community when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA (deoxyribonucleic acid) of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV (human immunodeficiency virus), even though there was no specific reason to put them through the process.

That technology is not safe, said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr scissors often cut next to the targeted gene, causing unexpected mutations.

Its very easy to do if you dont care about the consequences, he added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species, e.g. malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesnt believe in the more dystopian scenarios predicted for gene therapy, including American biohackers injecting themselves with Crispr technology bought online.

Not everyone is a biologist or scientist, she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies crops?

Charpentier thinks that technology generally tends to be used for the better.

Im a bacteriologist -- weve been talking about bioterrorism for years, she said. Nothing has ever happened. AFP Relaxnews

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Gene editing breakthroughs that cured genetic diseases in 2019 - The Star Online

Bone Marrow Stem Cells Comments Off on Gene editing breakthroughs that cured genetic diseases in 2019 – The Star Online | January 5th, 2020

There is also another option on the table: a technology called haplo-identical, where they could use the stem cells from my brother, who is a 50 per cent match.

But it shouldnt have been this hard to find a match, and thats whyI started my campaign to sign more people up to the transplant list.I want to make a difference for other people who have to go through this.

If I dont make it, I want to leave a legacy that the children can look at when theyre older and know that Mummy did everything she could to fight this thing. There can only be one winner with this disease, and it needs to be me.

As told to Jessica Salter

Leukaemia Care is one of three charities supported by this years Telegraph Christmas Charity Appeal. Our others are Wooden Spoon, which works with the rugby community to raise money for disabled and disadvantaged children,and The Silver Line, a telephone support service for lonely elderly people. To donate,visit telegraph.co.uk/charity or call 0151 284 1927 before the end of January

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My agonising two-year wait for a stem-cell donor after being diagnosed with leukaemia - The Telegraph

Bone Marrow Stem Cells Comments Off on My agonising two-year wait for a stem-cell donor after being diagnosed with leukaemia – The Telegraph | January 4th, 2020