Hypochondroplasia (HCH) is a skeletal dysplasia similar to achondroplasia (ACH) but with milder features, that affects particularly the ossification of proximal long bones of arms and legs. Around 70% to 80% of cases of HCH are caused by N540K alterations in the FGFR3 gene. At the recent 6th Annual Achondroplasia & Skeletal Dysplasia Research Conference, researchers from Tyra Biosciences Inc. presented preclinical proof-of-concept data of TYRA-300, an oral FGFR3-selective inhibitor in a model of HCH.
Two drugs were pushed back by the EMA last week, with a recommendation that Ocaliva, currently the only second line standard of care for treating primary biliary cholangitis, be withdrawn from the market, and a refusal to grant conditional approval for masitinib in the treatment of amyotrophic lateral sclerosis.
Work at Shanghai Meiyue Biotech Development Co. Ltd. has led to the discovery of spiro-heterocycle substituted pyrimidine compounds acting as C-C chemokine receptor type 4 (CCR4) antagonists.
Monte Rosa Therapeutics Inc. has submitted an IND application to the FDA for MRT-6160, a highly selective and orally bioavailable molecular glue degrader directed against VAV1 in development for systemic and neurological autoimmune diseases.
The generation of anti-citrullinated protein autoantibodies (ACPA) are known key drivers in the pathogenesis of rheumatoid arthritis (RA) and are generated by peptidyl arginine deiminases (PADs).
Transmembrane serine protease 6 (TMPRSS6) is a negative regulator of hepcidin, which is the main iron homeostasis-regulating hormone. Regeneron Pharmaceuticals Inc. has recently presented preclinical data for the monoclonal antibody targeting TMPRSS6, REGN-7999, which is being developed for the treatment of iron overload.
Sarepta Therapeutics Inc. CEO Douglas Ingram said he expects “ferocious” demand for gene therapy Elevidys (delandistrogene moxeparvovec), granted full approval by the U.S. FDA for Duchenne muscular dystrophy (DMD). Shares of the Cambridge, Mass.-based firm closed June 21 at $16.72, up $37.22, or about 30% on the news.
Cellular communication network factor 1 (CCN1) is overexpressed in endothelial cells and synovial tissues of patients with rheumatoid arthritis. The effects of inhibiting CCN1 in two experimental models of RA were tested.
The immune cell restricted guanine nucleotide exchange factor (GEF) and scaffolding protein VAV1 plays a key role in mediating T-cell receptor (TCR) and B-cell receptor (BCR) activity and signaling.
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