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.
Inflammatory bowel disease (IBD) is sometimes associated with spondyloarthritis (SpA) and it highly impacts patients’ quality of life. It is crucial to understand the pro-inflammatory processes that take place during the pathogenesis of IBD-associated SpA.
Santa Ana Bio Inc. has emerged from stealth with $168 million in combined series A and B funding and a focus on developing targeted therapies for patients with autoimmune and inflammatory diseases.
The good news for Sarepta Therapeutics Inc. is bad news for Pfizer Inc. as the phase III study of its mini-dystrophin gene therapy in Duchenne muscular dystrophy (DMD) has missed its primary endpoint. Now Sarepta’s Elevidys (delandistrogene moxeparvovec), a single-dose, adeno-associated virus-based gene transfer therapy for DMD, is barreling toward a June 21 PDUFA date with the U.S. FDA as the near competition shrinks in the rearview mirror.
Shares of Avidity Biosciences Inc. hit a 52-week high on reports of promising data from the first efficacy cohort of its phase I/II Fortitude study testing delpacibart braxlosiran (del-brax) in facioscapulohumeral muscular dystrophy (FSHD), including impressive biomarker results that could indicate a path for potential accelerated approval.
Hangzhou Zhongmei Huadong Pharmaceutical Co. Ltd. has described cyclic GMP-AMP synthase (MB21D1; cGAS) inhibitors reported to be useful for the treatment of psoriatic arthritis, diabetes, inflammatory bowel disease, multiple sclerosis, rheumatoid arthritis, sepsis, systemic lupus erythematosus and transplant rejection, among others.
Fibrodysplasia ossificans progressiva (FOP) is a rare and life-threatening genetic disease caused by gain-of-function mutations in the ALK2 gene, which encodes activin receptor-like kinase 2. Blueprint Medicines Corp. has elucidated the discovery of their ALK2 inhibitor BLU-782, which is now in phase II studies at Ipsen SA for the treatment of FOP.
Fibrodysplasia ossificans progressiva is a rare and life-threatening genetic disease caused by gain-of-function mutations in the ALK2 gene, which encodes activin receptor-like kinase 2. Blueprint Medicines Corp. has elucidated the discovery of their ALK2 inhibitor BLU-782, which is now in phase II studies at Ipsen for the treatment of FOP.
Bridgebio Pharma Inc.’s data from the phase II study with infigratinib in achondroplasia “swings the pendulum of debate sharply in favor” of the firm, competing with Biomarin Pharmaceutical Inc. in the space, Leerink analyst Mani Foroohar said in a report.
The EMA has been sent back to the drawing board to re-evaluate PTC Therapeutics Inc.’s Duchenne muscular dystrophy therapy Translarna (ataluren), after failing to get the usual rubber stamp following its recommendation in January that the drug’s conditional approval be withdrawn.
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