Company intelligence / Profile preview
Epicrispr Biotechnologies
South San Francisco, California, United Statesepicrispr.com ↗
- Ownership
- Private
- Development stage
- Other
01
Overview
Epicrispr’s lead program EPI-321 is entering Phase 1 clinical trials in New Zealand for FSHD after receiving FDA IND clearance. Preclinical data show robust suppression of DUX4 expression and muscle protection. The therapy has received Fast Track, Rare Pediatric Disease, and Orphan Drug designations from the FDA. Other pipeline programs remain at the preclinical stage.
- Therapeutic areas
- Metabolic DisordersOncologyRare DiseasesHematologyOphthalmology
- Modalities
- Epigenetic modulationGene expression modulationCRISPR-based gene regulation (non-cutting, dCas proteins)AAV-delivered gene therapies
- Industry
- Biotech
02
Drug pipeline
7 assets6 more assets in the full pipeline.
03
Partnerships
Kite Pharma/Gilead Sciences – research collaboration to develop next-generation CAR T-cell therapies using GEMS platform technology.SOLVE FSHD – advocacy/investor partnership focused on FSHD clinical development funding.Ally Bridge Group – lead investor with strategic involvement.
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