Onco3r Therapeutics BV has obtained clinical trial application (CTA) approval by the Belgian regulatory authorities for its SIK3 inhibitor O3R-5671. A first-in-human trial will be conducted in Belgium and is expected to open enrollment in the coming weeks. Final data are expected in the first half of next year and will inform subsequent patient trials across a range of autoimmune diseases, which are planned to commence next year.
Researchers have identified a potential therapeutic target for muscular dystrophies, a group of genetic disorders characterized by progressive muscle weakness and degeneration. The study reveals that inhibiting the microRNA (miRNA) known as miRNA-33 can significantly improve muscle regeneration and ameliorate the dystrophic phenotype in animal models.
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.
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.
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