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.
Shares of Entera Bio Ltd. (NASDAQ:ENTX), a lightly traded Israeli drug delivery specialist, fell 27% on May 21 after the company said an ongoing phase II trial of its oral parathyroid hormone (PTH) candidate in osteoporosis patients found the two lowest doses tested demonstrated "suboptimal increases" in P1NP, an important biomarker of bone formation.
HONG KONG – South Korea’s Daewon Pharmaceutical Co. Ltd. has launched Terrosa, a teriparatide biosimilar for the treatment of osteoporosis, acquired from Hungarian firm Richter-Helm Biotec GmbH & Co KG. It’s the first biosimilar launch for the company.
Though Quralis Corp., of Cambridge, Mass., has been developing its pipeline for more than three years, the company now has a $42 million series A in hand to continue researching and developing therapies for amyotrophic lateral sclerosis and frontotemporal dementia.
DUBLIN – Dyno Therapeutics Inc., an early stage gene therapy firm applying artificial intelligence to advanced capsid engineering, has entered partnerships with Novartis AG and Sarepta Therapeutics Inc., in ophthalmic indications and muscle diseases, respectively, which have over $2 billion in biobucks attached.
Weeks after raising an oversubscribed $60 million series A, Affinia Therapeutics Inc., of Waltham, Mass., is collaborating with Vertex Pharmaceuticals Inc. in a far larger deal, one potentially worth more than $1.6 billion.
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