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).

Satellos Bioscience Inc. has released promising preliminary data showing SAT-3247 can improve skeletal muscle function in a mouse model of...

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).

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.