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.
Modifying a patient’s DNA is no longer just for science fiction novels. The CRISPR gene editing technique developed by Jennifer Doudna and Emmanuelle Charpentier only took 10 years to reach the market as Casgevy (exagamglogene autotemcel/exa-cel, Vertex Pharmaceuticals Inc.), treating congenital pathologies such as β-thalassemia and severe sickle cell disease. But science does not stop.
Iaso Biotherapeutics Co. Ltd. has established new collaborations with Umoja Biopharma Inc. for the development and commercialization of novel ex vivo and in vivo cell and gene therapies. These collaborations seek to advance off-the-shelf cell and gene therapies with applications in oncology and immunology.
Voyager Therapeutics Inc. has entered into a strategic collaboration and capsid license agreement with Novartis Pharma AG, a subsidiary of Novartis AG, to advance potential gene therapies for Huntington’s disease and spinal muscular atrophy (SMA).
Modifying a patient’s DNA is no longer just for science fiction novels. The CRISPR gene editing technique developed by Jennifer Doudna and Emmanuelle Charpentier only took 10 years to reach the market as Casgevy (exagamglogene autotemcel/exa-cel, Vertex Pharmaceuticals Inc.), treating congenital pathologies such as β-thalassemia and severe sickle cell disease (SCD). But science does not stop.
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