CureDuchenne Ventures Invests in MyoGene Bio and its AAV-Delivered CRISPR/Cas9 Gene Editing Approach

Newport Beach, Calif. (May 11, 2023) – CureDuchenne Ventures announced funding for MyoGene Bio, LLC, a biotech company developing a novel gene editing therapy for Duchenne muscular dystrophy (DMD). MyoGene will use this funding to advance development of MyoDys^45-55, an AAV-delivered CRISPR/Cas9 gene editing approach that would be applicable to up to 50 percent of all DMD patients.   

MyoGene’s therapy aims to permanently remove a hotspot region where a large proportion of DMD patient mutations occur, with the goal of restoring a smaller version of the dystrophin protein. The rationale for this approach comes from clinical cases of individuals naturally lacking this hotspot region between exons 45 and 55. This deletion is associated with a very mild Becker muscular dystrophy disease progression, including individuals who were asymptomatic into their 60s, and produces a dystrophin protein that retains 87 percent of the normal protein sequence found in healthy muscle.   

“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,” said Debra Miller, founder and chief executive officer of CureDuchenne. “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. We are excited about this approach and grateful to those in the Duchenne community who have supported us, allowing us to make this investment.” 

“We are honored to receive this investment and to align with CureDuchenne in driving transformative treatments for Duchenne muscular dystrophy,” said Courtney Young, PhD, chief executive officer of MyoGene Bio. “CureDuchenne’s commitment to this work was critical for securing further funding from California’s Institute for Regenerative Medicine (CIRM). Since MyoGene’s approach is to permanently remove a mutational hotspot using gene editing, it would correct the underlying cause of disease for half of all patients while producing a much larger protein than micro-dystrophin gene replacement. We believe this would offer a truly meaningful therapy for a large proportion of Duchenne patients.” 

About Duchenne Muscular Dystrophy (DMD) 
Duchenne muscular dystrophy (DMD) is a rare, lethal, inherited neuromuscular disease that occurs in approximately one in every 3,500-5,000 males. It is caused by a mutation in the gene that encodes instructions for dystrophin, a key structural protein in muscle. Symptoms of DMD usually appear in infants and toddlers as difficulty in walking, climbing stairs, or standing from a sitting position. Most individuals with DMD will require full-time use of a wheelchair in their teens, and as muscle weakness and degeneration progresses to their upper limbs, lose the ability to perform other activities of daily living.  Cardiopulmonary complications are the primary cause of death. 

About MyoGene Bio LLC 
MyoGene Bio was founded in 2018 by University of California, Los Angeles researchers Drs Courtney Young, Melissa Spencer, and April Pyle, with the mission to develop cutting edge genetic therapies for muscle diseases. MyoGene Bio’s lead program is a gene editing therapy, MyoDys^45-55, for Duchenne muscular dystrophy, a devastating muscle wasting disease. MyoGene Bio utilizes new technologies to have a strong positive effect on disease progression by targeting the underlying cause of disease. The company philosophy is to make meaningful impacts on muscle diseases through dedication to honesty, integrity, and innovation. For more information, please visit www.myogenebio.com. 

About CureDuchenne Ventures  
CureDuchenne Ventures, LLC is the investment arm of CureDuchenne, a non-profit patient advocacy organization and global leader in funding research, patient care, and innovations for improving and extending the lives of those with Duchenne muscular dystrophy (DMD). CureDuchenne Ventures has supported transformative treatments for DMD, including 17 projects that advanced to human clinical trials, and multiple equity investments in companies striving to overcome the limitations of first-generation exon-skipping and gene therapy. For more information, please visit cureduchenne.org.