Dystroglycanopathies are a subset of rare congenital muscular dystrophies (CMDs) caused by dysregulation in the assemblage of glycans on the α-dystroglycan (α-DG) transmembrane glycoprotein.
Based on an analysis of critical transcription factors, researchers have stratified rheumatoid arthritis (RA) into two subtypes where gene expression was driven by RAR-α or TGF-β, respectively. The findings, published in Nature Communications on Oct. 20, 2022, by researchers from the University of California, San Diego, give new insights into factors that affect the treatment response in RA, and could point to novel treatment strategies.
Vicore Pharma AB has divulged new angiotensin AT2 receptor (AGTR2) agonists reported to be useful for the treatment of rheumatoid arthritis, diabetic nephropathy, pulmonary arterial hypertension, myocardial infarction, pneumonia viral, sarcoidosis, idiopathic pulmonary fibrosis and systemic scleroderma (systemic sclerosis), among other disorders.
Vicore Pharma AB has divulged new angiotensin AT2 receptor (AGTR2) agonists reported to be useful for the treatment of rheumatoid arthritis, diabetic nephropathy, pulmonary arterial hypertension, myocardial infarction, pneumonia viral, sarcoidosis, idiopathic pulmonary fibrosis and systemic scleroderma (systemic sclerosis), among other disorders.
Ascidian Therapeutics Inc. secured $50 million in series A funding from its founder Apple Tree Partners to take a therapy based on its RNA exon editing technology into a first-in-human trial in ABCA4 retinopathy and to advance additional programs in neuromuscular, neurological, and rare disease indications.
Cabaletta Bio Inc. has announced CABA-201, a newly designed, fully human CD19 chimeric antigen receptor (CAR) containing a 4-1BB co-stimulatory domain.
Endpoint Health Inc. has announced a new precision immunology program targeting inflammation mediated by tissue factor (TF)-dependent intracellular signaling.
The wholesale acquisition cost for Amylyx Pharmaceuticals Inc.'s new U.S. FDA-approved amyotrophic lateral sclerosis (ALS) treatment, Relyvrio, has been set at about $158,000 for the first year’s treatment. It jumps to about $163,000 in the second year, the company said, a change that would move it closer to the cost of competitor Mitsubishi Tanabe Pharma Corp.'s Radicava (edaravone), which costs about $165,000 annually. A 28-day prescription will cost $12,504.
Solid Biosciences Inc. found a way to continue its Duchenne muscular dystrophy push, inking a deal to acquire privately held Aavantibio Inc., at the same time nailing down $75 million by way of a private placement with institutional investors.
Eli Lilly & Co. has identified new fibroblast growth factor receptor 3 (FGFR3) inhibitors reported to be useful for the treatment of achondroplasia, cancer, pulmonary fibrosis, systemic scleroderma and thanatophoric dysplasia, among other disorders.
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