In the meantime, the work has spawned a new biotechnology company, Exonics Therapeutics Inc., which is working to further optimize and extend the approach to additional DMD mutations, as well as other neuromuscular diseases. (sciencedaily.com)
• Exonics Therapeutics, Inc, a Boston - based biotechnology company focused on developing SingleCut CRISPR technology to repair mutations causing Duchenne muscular dystrophy and other neuromuscular diseases, raised $ 40 million in Series A funding. (fortune.com)
Our goal is, through the greater understanding of pathological mechanisms implicated in this disease, to identify new therapeutic strategies for this neuromuscular disease and to extend this experimental approach to other neuromuscular diseases. (istem.eu)