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Cure ADSSL1
Woodbridge, Connecticut, United States
- Ownership
- Private
- Development stage
- Other
01
Overview
Non-profit biotech developing genetic medicines for rare neuromuscular diseases; achieved FDA IND approval for first-in-human CRISPR transcriptional activator for DMD in 2022; no reported clinical results yet.
- Therapeutic areas
- NeurologyRare Diseases
- Modalities
- Gene therapyCRISPR-based therapyAntisense oligonucleotide
- Industry
- Biotech
02
Partnerships
Charles River Laboratories
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