HONG KONG – Belgium-based Bone Therapeutics SA has inked development and commercialization deals for its bone cell therapy platform in Chinese and Southeast Asian markets.
Scribe Therapeutics Inc., another CRISPR-based genome editing firm out of the Jennifer Doudna stable, launched with $20 million in series A funding and a deal with Biogen Inc. in amyotrophic lateral sclerosis (ALS), which brings in another $15 million up front and up to $400 million in development and commercial milestones.
Both Solid Biosciences Inc. and Pfizer Inc. got lifts to their Duchenne muscular dystrophy (DMD) programs from the FDA as the agency released a clinical hold and awarded a fast track designation to their respective adeno-associated viral (AAV) programs.
PERTH, Australia – Antisense Therapeutics Ltd. saw its stock bounce 26% following the news that the FDA has granted rare pediatric disease designation for ATL-1102 for the treatment of Duchenne muscular dystrophy (DMD), following submission of phase II data that showed its immunomodulatory therapy met primary disease progression endpoints.
Positive top-line data from Blueprint Medicines Corp.’s phase I and II trials of Ayvakit (avapritinib) for treating advanced systemic mastocytosis (SM) are pushing the company to submit an sNDA before 2020 ends.
Researchers at the University of California at San Diego have used RNA-targeted CRISPR to reverse symptoms in an animal model of myotonic dystrophy type 1 (DM1). They reported their findings in the Sept. 14, 2020, issue of Nature Biomedical Engineering.
In the more than two years since Casma Therapeutics Inc. raised its series A and completed its new $50 million series B, the company has advanced its agonist program for treating muscular dystrophy and identified new targets.
DUBLIN – Ipsen SA is on track for an NDA filing for palovarotene in fibrodysplasia ossificans progressiva (FOP), an ultra-rare disease characterized by the gradual replacement of skeletal muscle and connective tissue with bone, following an interim analysis of phase III data which indicates that the drug may have a substantial effect on the disease process.
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.
Shares of Sweden-based Hansa Biopharma AB (NASDAQ:HNSA) jumped 32.7% to SEK53.90 (US$5.81) on July 2 as Sarepta Therapeutics Inc. became the first partner to license its lead asset, imlifidase, to enable gene therapies to treat muscular dystrophy patients harboring neutralizing antibodies to the adeno-associated virus (AAV) vectors the medicines employ.
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