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Epicrispr Biotechnologies

Private
#Rare Disease#Muscular Dystrophy#Gene Therapy

Epigenetic therapies for facioscapulohumeral muscular dystrophy (FSHD). Lead program EPI-321 targets the genetic root cause. Also researching alpha-1 antitrypsin deficiency and familial hypercholesterolemia.

Company Info

Sector:Gene Therapy / Epigenetics
Headquarters:Cambridge, Massachusetts, USA
Founded:2020
Website:epicrispr.com/
Funding Round:Series B ($68M initially, $90M in latest round)
Total Funding:~$158M

Funding & Investor Relations

Notable Investors

  • Regeneron Ventures
  • Cormorant Asset Management
  • MPM Capital

Upcoming Milestones & Key Dates

2026-09
Clinical Data

FSHD Phase 1/2 data readout expected

2026 Q4
Regulatory

Potential IND filing for alpha-1 antitrypsin deficiency

Clinical Development Pipeline

EPI-321

Target: DUX4 gene silencing via epigenetic editing
Facioscapulohumeral muscular dystrophy (FSHD)
Preclinical
Phase 1
Phase 2
Phase 3
Approved

EPI-311

Target: Epigenetic silencing
Alpha-1 antitrypsin deficiency
Preclinical
Phase 1
Phase 2
Phase 3
Approved

EPI-421

Target: PCSK9 epigenetic silencing
Familial hypercholesterolemia
Preclinical
Phase 1
Phase 2
Phase 3
Approved

Recent News

Leadership Team

Amber Salzman

Chief Executive Officer

Matthew Porteus

Co-Founder, Scientific Advisory Board

Charles Gersbach

Co-Founder, Scientific Advisory Board

Careers & Job Openings

See all open roles

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