The Japanese government, industry and academia are deliberating health care policies and initiatives to boost Japan’s role in the future of regenerative medicine, experts at Bio Japan 2024 said, as the fruits of cell and gene therapy research come to fruition with new approvals.
Cellus Inc. has disclosed compounds acting as disintegrin and metalloproteinase with thrombospondin motifs 4 (ADAMTS4; aggrecanase-1) and 5 (ADAMTS5; aggrecanase-2) inhibitors reported to be useful for the treatment of septic arthritis, osteoarthritis, osteonecrosis, rheumatoid arthritis and tuberculosis.
Satellos Bioscience Inc. has developed and presented data for a compound that targeted the process of muscle regeneration based on modulation of satellite stem cell polarity.
Facioscapulohumeral muscular dystrophy (FSHD) is a severe muscle disorder caused by aberrant DUX4 mRNA expression in skeletal muscle. DUX4 activates downstream target transcriptome, known as D4T, leading to myofiber loss and muscle weakness.
Duchenne muscular dystrophy is a severe and progressive disorder caused by mutations in the dystrophin (DMD) gene that lead to malfunction or absence of dystrophin. This protein stabilizes the sarcolemma and protects muscle cells during contraction.
Shares of Scholar Rock Holding Corp. (NASDAQ:SRRK) soared $26.86, or 362%, to close Oct. 7 at $34.28, after the Cambridge, Mass.-based firm disclosed positive top-line data from the phase III Sapphire study testing apitegromab in patients with spinal muscular atrophy (SMA). Apitegromab, which Wainwright analyst Andres Maldonado said will “transform SMA” therapy, met the primary endpoint with statistically significant and clinically meaningful improvement in motor function as measured by the Hammersmith Functional Motor Scale Expanded.
Petragen Inc. has described ectonucleotide pyrophosphatase/phosphodiesterase family member 1 (ENPP1) inhibitors reported to be useful for the treatment of cancer, gingivitis, musculoskeletal and connective disorders, hematological diseases, bone, cardiovascular, immunological and neurological disorders, among others.
Capricor Therapeutics Inc. just wrapped up a visit with the U.S. FDA and is prepping to file a BLA in October for its Duchenne muscular dystrophy treatment. Linda Marbán, the company’s CEO, is the guest on the newest BioWorld Insider podcast and she talks about deramiocel (CAP-1002), the company’s allogeneic cardiac-derived cell therapy, for treating the rare disease and how the FDA has made strong efforts in helping lay the groundwork for deramiocel.
Interim phase II data of Wave Life Sciences Ltd.’s oligonucleotide, WVE-N531, revealed “impressive” dystrophin expression, solid safety and the potential for once-monthly dosing for boys with Duchenne muscular dystrophy who are amenable to exon 53 skipping. The findings drove Wave Life’s stock (NASDAQ:WVE) up by 53.4%, or $2.85, to close Sept. 24 at $8.19, after peaking earlier in the day at $8.35, its 52-week high.
Argenx SE’s ARGX-119 is a monoclonal antibody (MAb) targeting the muscle-specific kinase (MuSK) Frizzled (Fz)-like domain, and has entered early clinical development for the treatment of neuromuscular diseases.
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