Company intelligence / Profile preview

MyoGene Bio

San Diego, California, United Statesmyogenebio.com ↗

Ownership: Private
Employees: ~5
Development stage: Preclinical

Overview

MyoGene Bio is developing MyoDys45-55, a gene editing therapy that permanently removes a mutational hotspot in the dystrophin gene to restore protein expression. The therapy is applicable to approximately 50% of Duchenne muscular dystrophy patients and has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA as of November 2024[1][5].

Therapeutic areas: Rare Diseases
Modalities: Gene editing therapyAAV-delivered CRISPR/Cas9
Industry: Biotech

Drug pipeline

2 assets

MyoDys45-55

Preclinical

1 more asset in the full pipeline.

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