It’s a go for the two phase III Ensure studies of Immunic Inc.’s lead asset in treating relapsing multiple sclerosis. An unblinded data monitoring committee’s interim futility analysis concluded that the placebo-controlled, pivotal studies using vidofludimus calcium may continue as planned, with the program expected by the company to be completed in 2026.
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.
Researchers from Kexing Biopharm Co. Ltd. have published details on the development and preclinical characterization of GB18-06, a novel nanobody, also known as variable domain of heavy-chain antibody (VHH), targeting growth differentiation factor 15 (GDF15) and being developed for the treatment of cachexia.
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.
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