How grave they might be remains unknown, but regulatory questions have surfaced in briefing documents related to the soon-to-happen panel meeting on Sarepta Therapeutics Inc.’s gene transfer therapy delandistrogene moxeparvovec in Duchenne muscular dystrophy (DMD). The U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee will meet May 12 to discuss the compound, also known as SRP-9001.

Suzhou Genassist Therapeutic Co. Ltd. has announced its pre-IND application of its first base-editing product, GEN-6050, and acceptance by the FDA. GEN-6050 is an in vivo base-editing drug that targets exon 50 skipping in the Duchenne muscular dystrophy (DMD) gene.

Sarepta Therapeutics Inc. CEO Doug Ingram said the U.S. FDA has promised to schedule “expeditiously” an advisory committee meeting on the BLA related to SRP-9001 (delandistrogene moxeparvovec) for Duchenne muscular dystrophy (DMD).

Researchers have linked Duchenne muscular dystrophy (DMD) to a loss of regenerative capacity of muscle stem cells. The findings, which were published in the March 1, 2023, issue of Science Translational Medicine, suggest that boosting the regenerative capacity of muscle stem cells could delay or perhaps even prevent the progression of DMD. DMD is “an early and horrible disease,” senior author Frederic Relaix, who is the director of a research team studying the biology of the neuromuscular system at the Mondor Institute for Biomedical Research told BioWorld.

Tevard Biosciences Inc. has entered into a 4-year global research collaboration with Vertex Pharmaceuticals Inc. aimed at creating new tRNA-based therapies for patients with Duchenne muscular dystrophy (DMD) caused by nonsense mutations, with options to expand into additional muscular dystrophies and a second indication.

Satellos Bioscience Inc. has reported results from preclinical studies in a disease model of Duchenne muscular dystrophy (DMD). The company's proprietary Myoregenx assay platform identified a protein kinase (K9) as a potential drug target to modulate polarity in muscle stem cells.

The lack of dystrophin causes Duchenne muscular dystrophy (DMD), a muscle-wasting disease that is often accompanied by heart failure due to cardiomyocyte death and fibrosis that can lead to death of the patient. It has been proven that telomere shortening is a hallmark of DMD cardiomyocytes. Researchers from the Stanford School of Medicine have recently investigated whether preventing telomere shortening and attrition may be a therapeutic approach in DMD.

Inmune Bio Inc., together with a collaborator at University of California, Irvine School of Medicine, has shown targeting soluble TNF (sTNF) using a dominant-negative TNF (DN-TNF) biologic significantly decreased muscle damage in a murine mdx model of Duchenne muscular dystrophy (DMD) and showed a statistically significant increase in muscle regeneration.

Sysnav Healthcare SA and Roche Holding AG entered a new collaboration to develop digital endpoints for use in clinical trials of therapies for a range of neuromuscular disorders. The alliance combines Sysnav’s expertise in wearable technologies and movement evaluation with Roche’s clinical experience. It builds on an existing collaboration that led to the qualification of the world’s first digital endpoint, for evaluating therapies in development for Duchenne muscular dystrophy (DMD). They now aim to put that experience to work in widening its application to other disorders in which movement is a key parameter.