Prostaglandins induce the regeneration of muscle in rodents and humans through the prostaglandin E2 receptor EP4 subtype receptor, but this therapeutic pathway's potential is limited due to systemic tolerability. Researchers from Mesentech Inc. recently presented new results on their prostaglandin E2 receptor EP4 subtype receptor agonist irodanoprost trying to address this limitation issue.
Eight months after announcing the $18.5 million first tranche of its series A, Augustine Therapeutics has closed the oversubscribed round at $85 million and is now ready to begin clinical development of its novel histone deacetylase-6 (HDAC6) inhibitors.
Researchers from Suzhou Genassist Therapeutics Co. Ltd. have developed a novel canine model of Duchenne muscular dystrophy (DMD), expected to be more predictive of disease pathogenesis and treatment efficacy.
Charcot-Marie-Tooth disease type 2E (CMT2E) is a slow and progressive neuropathy characterized by axonal dysfunction. Its clinical phenotype includes muscle weakness and atrophy, sensory loss and reduced nerve conduction velocity, among others.
Investigators from Insmed Inc. have presented new preclinical data on the efficacy of their adenoviral vector (AAV9)-based gene therapy INS-1201 for the treatment of Duchenne muscular dystrophy (DMD).
At this week’s Muscular Dystrophy Association Clinical and Scientific Conference in Dallas, researchers from Suzhou Genassist Therapeutics Co. Ltd. presented preclinical data for GEN-6050X (ss.AAV9.oTAM and ss.AAV9.hE50-sgRNA).
Sarcopenia is an age-related condition in which muscle mass and strength decrease, leading to reduced overall physical performance. As fast-twitch muscle fibers are more impacted by age-related decline, strategies aimed at enhancing the regeneration and functionality of these fibers are essential to prevent the progression of sarcopenia.
Celltrion Inc. is on a biosimilar roll with the U.S. FDA this month, having gained clearance of Stoboclo and Osenvelt as products referencing Amgen Inc.’s biologic, denosumab (Prolia, Xgeva), along with Omlyclo becoming the first and only interchangeable biosimilar to omalizumab (Xolair, Genentech Inc. and Novartis AG).
Wall Street was weighing the gravity of the death from acute liver failure of a patient who was treated for Duchenne muscular dystrophy (DMD) with Sarepta Therapeutics Inc.’s gene therapy, Elevidys (delandistrogene moxeparvovec). Liver injury is a known possible side effect of the product, first approved by the U.S. FDA in June 2023 for DMD, as well as other AAV-mediated gene therapies, and the potential problem is highlighted in Elevidys’ prescribing information.
While data on functional endpoints are still to come, Avidity Biosciences Inc. executives said the firm is moving ahead with plans for a BLA filing by the end of 2025 for del-zota, an antibody-oligonucleotide conjugate, in Duchenne muscular dystrophy with mutations amenable to exon 44 skipping (DMD44), based on positive top-line data that analysts say bode well for Avidity’s other late-stage programs targeting rare neuromuscular diseases.
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