MyoDys45-55
Duchenne Muscular Dystrophy (mutations in exons 45-55)
Pre-clinicalActive
Key Facts
Indication
Duchenne Muscular Dystrophy (mutations in exons 45-55)
Phase
Pre-clinical
Status
Active
Company
About MyoGene Bio
MyoGene Bio is a private, pre-clinical stage biotech developing gene editing therapies for muscular disorders, with an initial focus on Duchenne muscular dystrophy (DMD). Its lead candidate, MyoDys45-55, is designed as a single-treatment platform targeting the underlying genetic mutation in approximately 50% of DMD patients. The company has secured initial funding and key regulatory designations, building on foundational research from its UCLA-affiliated scientific founders. MyoGene Bio operates in the high-need, high-value rare disease therapeutics space.
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