Pepgen Inc. fell slightly short of its phase II dystrophin goal with PGN-EDO51 for patients with Duchenne muscular dystrophy (DMD) whose mutations are amenable to an exon 51-skipping approach, but Wall Street reacted in a big way, sending the Boston-based firm’s stock (NASDAQ:PEPG) down 33%, or $5.55, to close July 31 at $11.43.
Researchers from the Second Affiliated Hospital of Fujian Medical University and Shaoxing People's Hospital presented preclinical data for the signal transducer and activator of transcription 3 (STAT3) inhibitor STX-0119, which has been previously validated in models of cancer.
The EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended approval of 14 drugs and the extension of the label of 11 others at its July meeting, but, inevitably, it was the decision to turn down the Alzheimer’s disease therapy Leqembi (lecanemab) that stirred the greatest reaction.
Three Chinese biopharmaceuticals have filed for IPOs on the Hong Kong exchange in July alone, highlighting a potential “newfound positivity” to strengthen the HKEX IPO market in the second half of 2024.
Researchers from Shandong University and affiliated organizations have presented data from a study that aimed to assess the role of endosome-associated trafficking regulator 1 (ENTR1) in adipogenesis.
The U.S. FDA approval won in June by Argenx SE of subcutaneously given Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase) for adults with chronic inflammatory demyelinating polyneuropathy (CIDP) sparked interest in other prospects taking aim at the rare disease. Some – including Argenx – are going after generalized myasthenia gravis and multifocal motor neuropathy with their compounds.
Astellas Pharma Inc.’s subsidiaries Astellas Institute for Regenerative Medicine (AIRM) and Universal Cells Inc. have entered into a research collaboration with Osaka University to develop a pluripotent stem cell-derived cartilage organoid cell therapy for the treatment of intervertebral disc degenerative disease.
Rheumatoid arthritis (RA) is an autoimmune disease characterized by bone and cartilage destruction, where immune cells such as macrophages, among others, play a crucial role in RA pathogenesis.
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