# Epicrispr Biotechnologies

> Epicrispr Biotechnologies is a South San Francisco clinical-stage biotech developing epigenetic medicines that change gene expression without cutting DNA. Its lead candidate, EPI-321, is a one-time AAV-delivered therapy intended to silence DUX4 in facioscapulohumeral muscular dystrophy. The company reported early muscle-volume and biomarker signals from an open-label Phase 1/2 study, while its broader GEMS platform supports additional research programs and a cancer cell-therapy partnership.

- **Founded:** 2018
- **Headquarters:** South San Francisco, United States
- **Founders:** Lei Stanley Qi (Scientific founder)
- **Products:** EPI-321, GEMS platform, EPI-331, EPI-141
- **Notable:** FDA Fast Track, Rare Pediatric Disease and Orphan Drug designations for EPI-321, First EPI-321 patient dosed in August 2025, Twelve participants dosed in the Phase 1/2 dose-escalation study by July 2026

## Products & services

- **EPI-321** — Investigational single-dose AAV-delivered epigenetic therapy designed to re-methylate D4Z4 and suppress DUX4 in FSHD; in Phase 1/2 clinical study.
- **GEMS platform** — Modular Gene Expression Modulation System combining a DNA-binding protein, guide RNA, and epigenetic or transcriptional modulator; an internal drug-development platform and basis for a Kite collaboration.
- **EPI-331** — In vivo research program for Duchenne muscular dystrophy.
- **EPI-141** — In vivo research program for retinitis pigmentosa 4 using a suppress-and-replace strategy.

## Achievements

- FDA Fast Track, Rare Pediatric Disease and Orphan Drug designations for EPI-321
- First EPI-321 patient dosed in August 2025
- Twelve participants dosed in the Phase 1/2 dose-escalation study by July 2026
- First three evaluable patients at six months showed an average approximately 370 mL gain in lean muscle volume in a company-reported interim analysis
- Named to Fast Company's 2026 Most Innovative Companies list

## Latest updates

- **2026-08** — Closed $90 million Series C to support EPI-321 pivotal development and platform expansion.
- **2026-07** — Completed enrollment and dosing of 12 participants across two EPI-321 dose cohorts.
- **2026-06** — Reported approximately 370 mL average gain in lean muscle volume in the first three evaluable participants at six months.
- **2026-01** — Reported early safety and functional observations from first four dosed participants.

## Links

- Website: https://epicrispr.com/
- LinkedIn: https://www.linkedin.com/company/epicrispr-biotechnologies
- Twitter/X: https://twitter.com/epicbiotech

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Last updated: 2026-09-29