![]() ![]() Red blood cells in good health are spherical and flow through small blood channels to carry oxygen to all regions of the body. Sickle cell disease is a group of inherited red blood cell diseases that affect hemoglobin, the protein that transports oxygen throughout the body. Request a sample and discover the recent advances in sickle cell disease treatment drugs Sickle Cell Disease Competitive Landscape Report In June 2022, Precision BioSciences, Inc., a clinical-stage gene editing company developing ARCUS-based ex vivo allogeneic CAR T and in vivo gene editing therapies announced it has entered into an exclusive worldwide in vivo gene editing research and development collaboration and license agreement with Novartis Pharma AG.Forma Therapeutics is a clinical-stage biopharmaceutical company focused on transforming the lives of patients with sickle cell disease (SCD) and rare blood disorders. In October 2022, Novo Nordisk completed the acquisition of Forma Therapeutics Holdings, Inc.(GBT), a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments that provide hope to underserved patient communities starting with sickle cell disease (SCD). In October 2022, Pfizer announced the completion of its acquisition of Global Blood Therapeutics, Inc.In November 2022, C4X Discovery Holdings plc signed an exclusive worldwide licensing agreement with AstraZeneca worth up to $402 million, for its NRF2 Activator programme. ![]() Louis to advance a Phase I clinical trial that will evaluate the safety and feasibility of the Company's lead clinical candidate motixafortide to mobilize CD34+ hematopoietic stem cells (HSCs) for gene therapies in SCD. announced a collaboration with Washington University School of Medicine in St. If approved, lovo-cel will be bluebird bio's third ex-vivo gene therapy approved by the FDA for a rare genetic disease and its second FDA approval for an inherited hemoglobin disorder, building on more than a decade of leadership in gene therapy. ![]() The BLA includes a request for Priority Review, which, if granted, would shorten the FDA's review of the application to six months from the time of filing, versus a standard review timeline of 10 months. announced the submission of its Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for lovotibeglogene autotemcel (lovo-cel) gene therapy in patients with sickle cell disease (SCD) ages 12 and older who have a history of vaso-occlusive events (VOEs).
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