About Sickle Cell Disease Bioverativ and Sangamo Announce FDA Acceptance of IND Application for Gene-Edited Cell Therapy BIVV003 to Treat Sickle Cell Disease Business Wire WALTHAM, Mass. Ph.D., Vice President, Clinical Development at Bioverativ. Sickle cell disease (SCD) affects more than five million people worldwide, predominantly in sub-Saharan Africa. We believe the precision, efficiency and specificity of zinc finger nuclease technology differentiate BIVV003 from other genomic therapies in development.”. “Gene-edited cell therapy has the potential to provide patients living with sickle cell disease a lifelong treatment with a single administration. Sanofi's Bioverativ subsidiary's programs supplement GBT's existing pipeline and support the company's strategy to address sickle cell disease from multiple approaches. Bioverativ and Sangamo are developing BIVV003 as part of an exclusive worldwide collaboration to develop and commercialize gene-edited cell therapies for sickle cell … About BIVV003 and the Phase 1/2 Clinical Trial Bioverativ and Bicycle Therapeutics announced a research collaboration focused on the discovery, development and commercialization of innovative therapies for hemophilia and sickle cell disease. This press release contains forward-looking statements regarding Sangamo’s current expectations. Editor's note: This story was updated to include financial terms of the agreement with Sanofi. The company tapped Syros Pharmaceuticals and its gene control platform in December 2019 to develop new treatments for sickle cell and beta thalassemia. It was a landmark approval in a field where doctors focused on managing symptoms, such as infections, pain throughout the body and fatigue. or This IND enables Bioverativ to initiate a Phase 1/2 clinical trial to assess the safety, tolerability, and efficacy of BIVV003 in adults with sickle cell disease, and Bioverativ expects to open several clinical sites across the United States this year. The programs Global Blood Therapeutics licensed from Sanofi’s Bioverativ unit work differently than Oxbryta, which stops the polymerization, or sickling, of an abnormal type of hemoglobin found in people with sickle cell disease. About the Bioverativ and Sangamo Collaboration //--> RELATED: GBT picks up Roche’s dropped heart drug for its sickle cell disease pipeline in $127M deal. Bioverativ will also present preclinical data on BIVV003, a gene-edited cell therapy candidate for the treatment of people with sickle cell disease, which is … About Sickle Cell Anemia. GBT adds 2 Sanofi sickle cell meds to its pipeline in deal worth up to $353M, GBT chief blames COVID-19 for 'clear' slowdown in Oxbryta launch, but analysts are still impressed, GBT picks up Roche’s dropped heart drug for its sickle cell disease pipeline in $127M deal. The collaboration will seek to identify and develop Bicycles to treat rare blood disorders. This week’s clinical update looks at 3 clinical trials for the treatment of sickle cell disease (SCD), relapsed or refractory blood cancers and certain T cell cancers. Sickle cell disease is a group of disorders that affects hemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body. [CDATA[> Lancet 381: 142-51. The Bioverativ … As part of the clinical trial protocol, a patient’s HSCs are isolated from the blood and then undergo non-viral, ex vivo gene editing using ZFNs to modify the erythroid enhancer of the BCL11A gene, which is a key regulator of the level of fetal hemoglobin. Bioverativ is committed to actively working with the blood disorders community, and its hemophilia therapies when launched represented the first major advancements in hemophilia treatment in more than two decades. * bioverativ and sangamo announce fda acceptance of ind application for gene-edited cell therapy bivv003 to treat sickle cell disease GBT has the option to license any drugs that come out of the partnership for development and commercialization. The collaboration will seek to identify and develop Bicycles to treat rare blood disorders. Sickle cell illness is an inherited dysfunction through which hemoglobin, the blood protein that carries oxygen, abnormally types right into a crescent form. Bioverativ Inc Sickle Cell Disease Drug Product and Services. Bioverativ research and development executive vice-president Dr Tim Harris said: “This collaboration offers a unique opportunity to identify an entirely new therapeutic modality that may lead to meaningful new treatments and outcomes for people living with haemophilia and sickle cell disease. Research. All rights reserved. As Global Blood Therapeutics works to bring its sickle cell disease drug, Oxbryta, to younger patients, the company is bolstering its pipeline with a pair of early-stage programs from Sanofi. Join us June 22–23 as we discuss new waves of cancer targets, novel approaches to cell and gene therapies, advances in biopharma manufacturing, and more. [CDATA[// >
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