Satellos Bioscience Inc. recently presented preclinical data for the novel oral small-molecule AP2-associated protein kinase 1 (AAK1) inhibitor SAT-3247, being developed for the treatment of Duchenne muscular dystrophy and other muscle degenerative diseases, at the Muscular Dystrophy Association conference.
Solid Biosciences Inc. has announced a nonexclusive worldwide license and collaboration agreement with Armatus Bio Inc. for the use of Solid’s proprietary capsid AAV-SLB101 for the development and commercialization of Armatus’ vectorized RNAi candidate to treat facioscapulohumeral muscular dystrophy (FSHD).
Epic Bio (Epicrispr Biotechnologies Inc.) has announced that EPI-321 has been awarded U.S. orphan drug designation for the treatment of facioscapulohumeral muscular dystrophy (FSHD).
Arrowhead Pharmaceuticals Inc. has filed an application in New Zealand for clearance to initiate a phase I/II trial of ARO-DUX4, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for patients with facioscapulohumeral muscular dystrophy (FSHD).
Gene therapy developer Kate Therapeutics Inc. (KateTx), which is developing next-generation adeno-associated virus (AAV) vectors that target skeletal and cardiac muscle, has unveiled $51 million series A round and a licensing deal with Astellas Pharma Inc.
Facioscapulohumeral muscular dystrophy (FSHD) is a skeletal muscular dystrophy characterized by DNA hypomethylation of D4Z4 repeat units of a macrosatellite array found at the distal end of chromosome region 4q35, which causes a myotoxic expression of DUX4. Researchers from Epic-Bio presented the discovery of EPI-321, a novel gene therapy candidate for the treatment of FSHD.
Mirecule Inc. has entered into a strategic collaboration and exclusive license agreement with Sanofi SA to develop and commercialize a best-in-class antibody-RNA conjugate (ARC) for the treatment of facioscapulohumeral muscular dystrophy (FSHD).
Controlling the epigenetics of a patient, figuring out what genes are expressed and understanding their level of expression, is at the center of Epic Bio, a new company founded by Stanley Qi, co-inventor on the CRISPR patent held by the University of California.
Fulcrum Therapeutics Inc.’s phase IIb data with losmapimod in facioscapulohumeral muscular dystrophy (FSHD) brought renewed hope for patients in what historically has proved a challenging therapeutic space. Though the firm’s oral p38 mitogen-activated protein kinase inhibitor missed its primary biomarker endpoint – changes in DUX4-driven gene expression – other indicators of benefit in the study called ReDUX4 painted a bright picture.