After raising AU$16.75 million (US$10.4 million) in a series A round, Celosia Therapeutics Pty Ltd. is heading toward the clinic with its novel gene therapy that targets TDP-43, a protein directly linked to amyotrophic lateral sclerosis (ALS) pathology.
Epirium Bio Inc. has obtained IND clearance from the FDA for MF-300, a first-in-class orally administered, 15-hydroxyprostaglandin dehydrogenase (15-PGDH) enzyme inhibitor.
Mitsubishi Tanabe Pharma Corp. has identified serine/threonine-protein salt-inducible kinases (SIK) inhibitors reported to be useful for the treatment of psoriatic arthritis, lupus nephritis, osteoarthritis, pancreatitis, rheumatoid arthritis, Sjögren’s syndrome, systemic lupus erythematosus and ulcerative colitis, among others.
Sudo Biosciences Ltd. has synthesized non-receptor tyrosine-protein kinase TYK2 (JH2 domain) inhibitors reported to be useful for the treatment of rheumatoid arthritis, multiple sclerosis, lupus and more.
Genescience Pharmaceuticals Co. Ltd. has identified fibroblast growth factor receptor 2 (FGFR2) and/or FGFR3 inhibitors reported to be useful for the treatment of cancer, osteochondrodysplasia and achondroplasia.
Dewpoint Therapeutics Inc. and Mitsubishi Tanabe Pharma Corp. have entered a research collaboration worth up to $480 million to advance Dewpoint’s novel TDP-43 small-molecule condensate modulator for amyotrophic lateral sclerosis. Under terms of the deal, Boston-based Dewpoint will receive an undisclosed up-front payment and is eligible to receive R&D-based milestone payments up to $480 million. Upon reaching those milestones, Osaka, Japan-based MTPC will have an exclusive option to license the program and assume responsibility for global clinical development and commercialization. Dewpoint will also receive tiered royalties on net sales.
At the Breakthroughs in Muscular Dystrophy special meeting held in Chicago Nov. 19-20, 2024, and organized by the American Society of Gene & Cell Therapy (ASGCT), multiple interventions at the RNA level were among the approaches that were presented to fight muscular dystrophies.
Since the isolation of the gene that causes Duchenne muscular dystrophy (DMD), scientists have progressed in understanding the mechanisms that lead to muscular diseases that can be evident from the early stages of childhood. This has led to the development of diagnostics and therapeutics, some approved by the FDA.
Autoimmunity Biosolutions has closed a seed financing to support its work advancing a next-generation, immuno-corrective therapy for autoimmune diseases. The company’s approach targets a branch of the interleukin-7 (IL-7)/interleukin-7 receptor (IL-7R) pathway controlled by the soluble IL-7R (sIL-7R), a critical amplifier of autoimmune reactions.
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