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.
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.
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