What Epicrispr Biotechnologies does

Epicrispr Biotechnologies is a clinical-stage biotechnology company developing in vivo gene therapies that modulate gene expression through epigenetic editing rather than cutting DNA. Its proprietary Gene Expression Modulation System (GEMS) is a modular platform that combines compact DNA-binding proteins (including the exclusively licensed CasMINI, the smallest dCas protein functional in human cells, and the proprietary CasONYX), customized guide RNAs, and a library of transcriptional and epigenomic modulators (histone modifiers, DNA methylators/demethylators, chromatin remodelers) to either dial up or down expression of disease-causing genes. The company's lead candidate, EPI-321, is an AAV-delivered therapy designed to re-methylate the D4Z4 region and suppress toxic DUX4 expression in facioscapulohumeral muscular dystrophy (FSHD); it entered a global Phase 1/2 trial in 2025 and has reported interim data showing statistically significant increases in lean muscle volume. Two additional pipeline programs, EPI-331 for Duchenne Muscular Dystrophy (mutation-agnostic utrophin upregulation) and EPI-141 for autosomal dominant retinitis pigmentosa 4 (suppress-and-replace), apply the same GEMS architecture to other rare genetic diseases.

The company is headquartered in South San Francisco, California, was spun out of Stanford University in 2018 by CRISPR pioneer Lei Stanley Qi, Ph.D., and officially launched in July 2022 with a $55 million Series A led by Horizons Ventures; Amber Salzman, Ph.D., serves as CEO. As of the most recent disclosures, Epicrispr operates with approximately 41 team members and has raised a total of approximately $123 million across its Series A and a $68 million Series B in March 2025 led by Ally Bridge Group with participation from SOLVE FSHD. The business model is pre-commercial: Epicrispr is not yet generating product revenue and instead funds operations through venture capital and a research collaboration with Kite Pharma to engineer next-generation CAR T-cell therapies using GEMS. The company partners with Forge Biologics for AAV cGMP manufacturing and with Springbok Analytics for AI-powered MRI biomarker analysis, and has secured FDA Fast Track, Orphan Drug, and Rare Pediatric Disease Designations for EPI-321 as the first and only open epigenetic editing IND authorized in the US.

Name

Epicrispr Biotechnologies

Legal name

Epicrispr Biotechnologies, Inc.

Website

https://epicrispr.com

Company type

Private

Founded year

2022

Operating status

Operating

Headcount range

11–50 employees

Short description

Epicrispr Biotechnologies is a clinical-stage biotech developing in vivo epigenetic editing therapies using its GEMS platform, with lead candidate EPI-321 in Phase 1/2 trials for facioscapulohumeral muscular dystrophy and pipeline programs in Duchenne and retinitis pigmentosa.

Ownership category

akta.pro rank

Product category

Epigenetic Gene Therapy

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Gene Therapy for Rare/Monogenic Diseases (HLAAACAE)

akta.pro secondary industries

Gene Therapy CMC/Process Development Platforms (capsid engineering, producer cell lines, plasmids, upstream/downstream) (HLAAACAN), Epigenomics & Chromatin Profiling Services (HLAGANAF)

Keywords

• Epigenetic gene therapy
• Gene expression modulation
• CRISPR-based therapeutics
• Rare neuromuscular disease
• AAV gene therapy

Headquarters

HQ city

South San Francisco

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Supply Chain

Revenue model

1. Gene Therapy Development and Commercialization: Pre-revenue clinical-stage biotech company. Developing proprietary epigenetic therapies targeting genetic diseases. Revenue expected from future therapeutic product sales upon regulatory approval.
2. Research Collaborations: Strategic research collaboration with Kite Pharma to develop next-generation CAR T-cell therapies using Epicrispr's epigenetic editing technology.

Go-to-market motion2 records

Distribution channels1 record

Marketing channels5 records

Core offering

Epicrispr Biotechnologies is a clinical-stage biotechnology company that develops and commercializes epigenetic gene therapies using its proprietary Gene Expression Modulation System (GEMS) platform, which combines compact DNA-binding proteins (CasMINI, CasONYX), customized guide RNAs, and epigenomic modulators to precisely dial gene expression up or down without altering DNA. Its lead clinical asset, EPI-321, is an AAV-delivered epigenetic therapy that re-methylates the D4Z4 region to suppress toxic DUX4 expression in facioscapulohumeral muscular dystrophy (FSHD), currently in Phase 1/2 trials with positive interim muscle volume data. The pipeline additionally includes EPI-331 for Duchenne Muscular Dystrophy and EPI-141 for autosomal dominant Retinitis Pigmentosa 4.

Product overview

Epicrispr Biotechnologies is a clinical-stage company pioneering epigenetic editing therapies using its proprietary Gene Expression Modulation System (GEMS) platform. The GEMS platform combines compact DNA-binding proteins (including CasMINI with exclusive license), guide RNAs, and epigenetic modulators to create customizable gene expression modulation therapies. Lead clinical program EPI-321 (FSHD, Phase 1/2) is in active clinical trials with positive interim data. Pipeline includes EPI-331 (DMD) and EPI-141 (RP4). The platform enables mutation-agnostic treatments that can dial up or down gene expression without permanently altering DNA.

Differentiator

Problem solved

Functional benefit

Products and services

• EPI-321 EPI-321 is an AAV-delivered (AAVrh74) one-time IV infusion epigenetic gene therapy for adults with genetically confirmed FSHD1 that re-methylates the D4Z4 region to suppress toxic DUX4 expression, addressing the underlying genetic cause of FSHD. Delivered as a single intravenous infusion and the first and only open epigenetic editing IND authorized in the US, with FDA Fast Track, Orphan Drug, and Rare Pediatric Disease Designations.
• EPI-331 EPI-331 is a preclinical-stage epigenome editing therapy for Duchenne Muscular Dystrophy (DMD) that uses the GEMS platform with CRISPR-based components to upregulate utrophin (UTRN) gene expression as a mutation-agnostic treatment strategy, working regardless of the specific dystrophin mutation a patient carries. Designed for pediatric and young male DMD patients; targeting rare X-linked muscle disorder affecting approximately 1 in 3,500-5,000 live male births.
• EPI-141 EPI-141 is a preclinical AAV-delivered gene therapy for autosomal dominant Retinitis Pigmentosa 4 (adRP4) that uses a "suppress and replace" strategy to block mutated rhodopsin gene expression while providing a healthy rhodopsin DNA sequence via a single AAV vector. Indication affects 5,000-8,000 people in the US with no available therapies.
• GEMS Platform (Gene Expression Modulation System) The GEMS Platform is Epicrispr's proprietary modular epigenetic editing platform that combines DNA-binding proteins (including exclusively licensed CasMINI and proprietary CasONYX), customized guide RNAs (designed using the APPRAISR machine learning model), and a library of epigenomic modulators (transcriptional activators and repressors, histone modifiers, DNA (de)methylators, chromatin remodelers) to precisely modulate gene expression without altering DNA. Enables mutation-agnostic, customizable therapies via mix-and-match architecture and is the underlying technology licensed to Kite Pharma for CAR T-cell engineering.

Quantifiable outcome

• Statistically significant increases in whole-body lean muscle volume - average ~370mL (~0.8 pounds) with individual gains from 0.5 to 1.3 pounds, some muscles gaining 15% lean muscle volume at 6 months
• +4 more outcomes

Named customers2 records

Segments4 records

Ideal customer profiles4 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

AI capability3 records

Feature6 records

Partnerships

Four partnerships are on record, tiered core.

• Forge BiologicscoreTechnology or Integration · 5 May 2025AAV development and manufacturing partnership. Forge Biologics' Columbus, Ohio facility provides adeno-associated virus process development, analytical development, and cGMP manufacturing for EPI-321.
• Springbok AnalyticscoreTechnology or Integration · 23 April 2025Partnership to integrate AI-powered muscle MRI imaging and analysis into EPI-321 first-in-human clinical trial. Springbok provides automated segmentation and quantification of muscle volume, fat content, and inflammation across dozens of muscle groups.
• Kite PharmacoreStrategic or Co-development Partner · 31 October 2023Collaboration to develop next-generation CAR T-cell therapies for cancer. Kite tapped Epicrispr's epigenetic editing technology to engineer T cells by modulating gene expression.
• Pacific Clinical Research NetworkcoreImplementation/ SI/ Consulting PartnerClinical research center in New Zealand conducting EPI-321 first-in-human trial in partnership with Epicrispr. Principal investigator Dr. Richard Roxburgh at University of Auckland.

Scale indicators5 records

Recent moves7 records

Expansion highlights6 records

Direct peers

• Tune Therapeutics: Tune Therapeutics is an epigenetic editing company developing tunable gene-expression therapies using CRISPR-based platforms. Direct competitor in the same therapeutic modality with overlapping target disease areas including rare genetic disorders.
• Omega Therapeutics: Omega Therapeutics is an epigenomic programming company developing DNA-encoded regulatory medicines that target genomic "insulators" to tune gene expression. Closely comparable platform approach to GEMS for precise gene-expression modulation without DNA alteration.
• Chroma Medicine: Chroma Medicine is a direct competitor developing an epigenetic editing platform using dCas9 fused to chromatin regulators, targeting genetic diseases and oncology. Highly comparable technology stack to Epicrispr's GEMS, including mix-and-match epigenetic modulator approach.
• Solid Biosciences: Solid Biosciences is a clinical-stage gene therapy company focused on Duchenne muscular dystrophy and other neuromuscular diseases. Directly competes with EPI-331 in DMD using AAV-delivered approaches, with overlapping clinical and regulatory pathways.

Emerging players

• Shape Therapeutics: Shape Therapeutics develops RNA editing and AAV-delivered genetic medicines, including platforms that combine RNA editing with epigenetic regulation. Adjacent technology category with shared delivery (AAV) and rare-disease focus, but differentiated at the editing modality.

Broad incumbents

• Ultragenyx Pharmaceutical: Ultragenyx is a rare-disease-focused biotech with a broad gene therapy and biologics pipeline, including neuromuscular and metabolic programs. Board member Eric Crombez serves as CMO, creating a strategic relationship; competes for the same rare-disease patient populations.
• Sarepta Therapeutics: Sarepta is the established gene therapy leader for Duchenne muscular dystrophy (DMD), with multiple approved therapies including AAV-delivered Elevidys. Represents the dominant competitor in Epicrispr's EPI-331 DMD indication with deep commercial infrastructure.
• Krystal Biotech:

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/epicrispr-biotechnologies

Revenue estimate

Valuation estimate

Number of profiles

Profiles17 records

Funding overview

Funding rounds

Investors

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Epicrispr Biotechnologies