MyoGene Bio, a San Diego, CA-based biotech company developing a novel gene editing therapy for Duchenne muscular dystrophy (DMD), received an investment from CureDuchenne Ventures.
The amount of the deal was not disclosed.
The company intends to use the funds to advance development of MyoDys45-55, an AAV-delivered CRISPR/Cas9 gene editing approach that would be applicable to up to 50 % of all DMD patients.
Founded in 2018 by University of California, Los Angeles researchers Drs Courtney Young, Melissa Spencer, and April Pyle, MyoGene Bio aims to develop genetic therapies for muscle diseases. Its lead program is a gene editing therapy, MyoDys45-55, for Duchenne muscular dystrophy, a devastating muscle wasting disease.
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.
FinSMEs
11/05/2023