NEW YORK – CureDuchenne Ventures on Thursday said it is funding MyoGene Bio's efforts to advance a gene editing therapy, dubbed MyoDys 45-55, for Duchenne muscular dystrophy (DMD) that up to 50 percent of patients could be eligible to receive.
DMD is a rare, inherited disease caused by DMD gene mutations, which lead to a lack of the dystrophin protein that is necessary for normal muscle fiber function. The muscle weakness characteristic of the disease, which affects around 1 in every 3,500 to 5,000 males, typically shows up in toddlers, and the mobility issues worsen with age, until patients are confined to a wheelchair.
Los Angeles-based biotech MyoGene will use CureDuchenne Ventures' investment for an undisclosed sum to further develop an adeno-associated virus-delivered CRISPR-Cas9 gene editing approach that removes the hotspot region between exons 45 and 55 in the DMD gene where most of the mutations perturbing the dystrophin protein occur. Deletions in this region of the gene are known to cause a mild form of muscular dystrophy where patients tend to be asymptomatic into their 60s and retain 87 percent of the normal dystrophic protein sequence observed in healthy muscles.
"CureDuchenne Ventures has a long history of investing in gene therapy and gene editing research to treat DMD, and MyoGene Bio's approach is an excellent application of CRISPR-Cas9 technology to potentially address a large proportion — up to half — of DMD cases," CureDuchenne CEO and Founder Debra Miller said in a statement. "This approach is aligned with our mission of being a catalyst, helping companies accelerate their research, provide proof of concept, and propel the technology forward to attract further investments."
Earlier this year, MyoGene received a $3.4 million grant from the California Institute for Regenerative Medicine to advance this CRISPR-Cas9 gene editing approach, which the company is collaborating on with researchers at the University of California, Los Angeles. When MyoGene received the CIRM funding in January, the company said it planned to use it to carry out studies necessary to file a pre-investigational new drug application with the US Food and Drug Administration for MyoDys 45-55.