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.
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).
Lululemon founder Chip Wilson has chipped in $100 million to prime the pump for finding a cure to the rare form of muscular dystrophy that has hampered him for the past 35 years. He’s got more than a monetary stake in the donation. The 67-year-old entrepreneur suffers from facioscapulohumeral muscular dystrophy type 2 (FSHD) and just launched Solve FSHD to find a cure.
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.
An interim analysis from Fulcrum Therapeutics Inc.’s phase II study of losmapimod for treating facioscapulohumeral muscular dystrophy (FSHD) produced data that pleased the CEO and displeased investors.
With a phase IIb readout coming in the third quarter of 2020, Cambridge, Mass.-based Fulcrum Therapeutics Inc. might be set up for a win in facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder for which there’s no treatment.