Amid the increasingly competitive myasthenia gravis drug development space, siRNA candidate cemdisiran met phase III endpoints, with the monotherapy showing numerically higher results than a combination product. Regeneron Pharmaceuticals Inc., which has a worldwide license to cemdisiran from Alnylam Pharmaceuticals Inc., plans to file for U.S. approval in generalized myasthenia gravis, a rare and chronic autoimmune disease leading to life-threatening muscle weakness, in the first quarter of 2026.
Ribomic’s umedaptanib pegol (RBM-007) looks to have some advantages compared to competitors in the achondroplasia space, and the company plans to progress the oligonucleotide-based aptamer that targets anti fibroblast growth factor 2 to phase III trials, Ribomic’s business development head, Kihei Yamashita, told BioWorld.
Osteoarthritis arises from breakdown of cartilage covering bone joints, which leads to chronic inflammation, and current therapies provide only short-term relief with risk of side effects. In an effort to identify next-generation treatments, researchers at Chang Gung University and Chang Gung Memorial Hospital have identified a potential novel therapeutic target, platelet-derived growth factor receptor-like protein (PDGFRL).
Rheumatoid arthritis (RA) is a chronic autoimmune disease characterized by the formation of an invasive and proliferative tissue called synovial pannus, consisting of fibroblast-like synoviocytes (FLSs), immune cells and newly formed vessels.
Homeobox genes, a conserved family of transcription factors, are key regulators of embryogenesis, cell growth and differentiation, and have been linked to bone mass regulation and osteogenesis. However, their specific roles in postmenopausal osteoporosis and osteoclast development are still not well understood, with limited and fragmented knowledge on which genes are central to these processes.
Alterity Therapeutics Ltd. helped develop a new neuroimaging biomarker called the multiple system atrophy index (MSA-AI), which looks to be a more reliable biomarker for tracking disease progression of MSA.
Muna Therapeutics ApS has patented triggering receptor expressed on myeloid cells 2 (TREM2) agonists reported to be useful for the treatment of osteoporosis, rheumatoid arthritis, systemic lupus erythematosus, type 2 diabetes and more.
Alterity Therapeutics Ltd. helped develop a new neuroimaging biomarker called the multiple system atrophy index (MSA-AI), which looks to be a more reliable biomarker for tracking disease progression of MSA. Developed using deep learning methods, the MSA-AI offers a superior, objective and quantifiable measure of brain atrophy in MSA patients.
Sarepta Therapeutics Inc.’s adventure with the Duchenne muscular dystrophy (DMD) AAV-based gene therapy Elevidys (delandistrogene moxeparvovec) continued as the firm said it would restart shipments of the compound for ambulatory patients “imminently,” with the U.S. FDA’s blessing.
Osteoporosis involves degradation of bone throughout the body, and it already affects nearly a quarter of a billion people in the aging global population.
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