Company intelligence / Profile preview
Cure Rare Disease
Woodbridge, Connecticut, United Statescureraredisease.org ↗
- Ownership
- Private
- Development stage
- Other
01
Overview
Successfully developed a First-in-Human CRISPR transcriptional activator that received FDA IND approval in July 2022. The first patient (founder's brother) unfortunately passed away during early clinical trial stages due to an immune reaction to the viral vector (AAV).
- Therapeutic areas
- Rare DiseasesNeurology
- Modalities
- Gene therapyCRISPR gene editingantisense oligonucleotides (ASOs)
- Industry
- Biotech
02
Drug pipeline
14 assets13 more assets in the full pipeline.
03
Partnerships
Collaborations with academic and industry partners across the United States, Canada, and Europe
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