With the U.S. FDA giving the green light to Takeda Pharmaceutical Co. Ltd.’s Adzynma for treating a rare blood clotting disorder caused by a deficiency in the ADAMTS13 enzyme, the company has won two approvals in two days after the FDA approved fruquintinib a day earlier.
With the U.S. FDA giving the green light to Takeda Pharmaceutical Co. Ltd.’s Adzynma for treating a rare blood clotting disorder caused by a deficiency in the ADAMTS13 enzyme, the company has won two approvals in two days after the FDA approved fruquintinib a day earlier.
Sirius Therapeutics Inc. has submitted an application in Australia to begin a first-in-human trial of SRSD-107 for the prevention and treatment of thromboembolic disorders.
Among the avalanche of abstracts for the upcoming American Society of Hematology (ASH) annual conference released early on Nov. 2 were data from Arcellx Inc.’s phase I study of CART-ddBCMA in patients with relapsed or refractory multiple myeloma. The results caught investors’ attention as the company’s stock (NASDAQ:ACLX) jumped 18.5% upward to close at $40.77 per share.
Had it been asked to, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee would have voted Oct. 31 to recommend approval of Vertex Pharmaceutical Inc. and Crispr Therapeutics AG’s exagamglogene autotemcel, or exa-cel, as a one-time transformative treatment for severe sickle cell disease in individuals 12 and older.
It’s just a discussion next week of Vertex Pharmaceuticals Inc. and Crispr Therapeutics AG’s sickle cell disease (SCD) therapy, with no vote expected from the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee. The FDA isn’t questioning the drug’s efficacy as much as it is concerned about the effects of off-target editing.
Generation Bio Co. has announced a breakthrough in its nonviral genetic medicine platform with the development of a proprietary, novel DNA called immune-quiet DNA (iqDNA).
Research at Purdue Research Foundation has led to the identification of SYK kinase inhibitors reported to be useful for the treatment of glucose-6-phosphate dehydrogenase deficiency, sickle cell disease, α- and β-thalassemia.
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