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CureDuchenne Ventures
Newport Beach, California, United Statescureduchenne.org ↗
- Ownership
- Private
- Development stage
- Other
01
Overview
CureDuchenne Ventures has invested over $26 million into research and biotech ventures targeting Duchenne muscular dystrophy. Their investments have catalyzed more than $3 billion in follow-on funding from VCs and pharma/biotech partners. At least nineteen projects funded have advanced into human clinical trials—including early support for Sarepta Therapeutics’ Exondys51®, the first FDA-approved drug for DMD—and several successful exits/acquisitions such as Prosensa’s acquisition by BioMarin.
- Therapeutic areas
- Rare Diseases
- Modalities
- Gene therapy (AAV-based and non-AAV)Gene editing (CRISPR/Cas9)tRNA-based therapeuticsNucleic acid delivery platforms (mRNA, DNA, siRNA, ASOs)Small molecules
- Industry
- Investors
02
Partnerships
Vertex Pharmaceuticals (via Exonics acquisition)BioMarin Pharmaceutical Inc. (via Prosensa acquisition)Sarepta TherapeuticsCapricor TherapeuticsEntos PharmaceuticalsCode BiotherapeuticsGennao Bio
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