Jaguar Gene Therapy LLC’s IND application for JAG-201, a gene therapy for a genetic form of autism spectrum disorder (ASD) and Phelan-McDermid syndrome (PMS; 22q13.3 deletion syndrome), has been cleared by the FDA.
Vivet Therapeutics SAS has been awarded financing of €4.9 million (US$5.3 million) from the French government to advance the development of a gene therapy for the treatment of the neurodegenerative disease cerebrotendinous xanthomatosis (CTX).
Newly approved gene therapies targeting sickle cell disease will be the first focus of the U.S. Centers for Medicare & Medicaid Services’ (CMS) Cell and Gene Therapy Access Model, the agency said Jan. 30.
Solid Biosciences Inc.’s SGT-003 has been granted orphan drug designation by the FDA. The company’s next-generation Duchenne muscular dystrophy gene therapy candidate was also granted fast track designation last month.
Beam Therapeutics Inc. has offered a progress update on its genetic disease franchise. BEAM-302, the company’s priority genetic disease program, is a potential treatment for α1-antitrypsin deficiency (AATD).
Kriya Therapeutics Inc. has announced its gene therapy program for thyroid eye disease (TED), KRIYA-586. It is designed to be a one-time, adeno-associated virus (AAV) gene therapy that drives durable expression of a monoclonal antibody blocking the insulin-like growth factor 1 receptor (IGF-1R).
Fractyl Health Inc. has nominated RJVA-001 as the first clinical type 2 diabetes candidate from its Rejuva gene therapy platform, which is designed to deliver locally administered genetic medicines to the pancreas.
RSS
