What Epicrispr Biotechnologies does

Epicrispr Biotechnologies is a clinical-stage biotechnology company developing gene-modulating therapies for neuromuscular diseases using its proprietary Gene Expression Modulation System (GEMS) platform. The GEMS platform is an epigenomic modulation technology that combines modular "epigenomic modulators" in a mix-and-match architecture to build indication-specific therapies, leveraging the epigenome — the ubiquitous gene-regulatory layer present in all mammalian cells — to dynamically control gene expression rather than permanently editing DNA. Founded in 2018 and headquartered in South San Francisco, California, the company operates with 11–50 employees under leadership of CEO Amber Salzman and founder Stanley Qi.

The company's pipeline centers on EPI-321, an investigational epigenetic therapy designed to silence the DUX4 gene and thereby prevent muscle degeneration in facioscapulohumeral muscular dystrophy (FSHD). EPI-321 received FDA IND clearance in April 2025 and is currently in Phase I clinical trials running across the U.S., New Zealand, and Australia; in June 2026 the company reported first clinical evidence of increased lean muscle volume in FSHD patients following treatment. Two additional neuromuscular pipeline candidates, EPI-331 and EPI-141, extend the GEMS platform into adjacent indications. Manufacturing and process development for EPI-321 are handled through a strategic partnership with Forge Biologics at their Ohio facility, announced May 2026.

Epicrispr is pre-revenue and does not yet have commercialized products or publicly disclosed pricing. Its business model is built around successful clinical development, regulatory approval, and eventual commercialization of its gene therapies, with potential future revenue streams expected to derive from one-time licensing, milestone payments, and product sales once approvals are secured. The company is privately held, having raised approximately $123M cumulatively across a $55M Series A in 2022 and a $68M Series B in April 2025 (led by Ally Bridge Group with Solve FSHD), positioning it among the more heavily funded private gene-therapy developers in its peer set.

Name

Epicrispr Biotechnologies

Legal name

Epicrispr Biotechnologies, Inc.

Website

https://epic-bio.com

Company type

Private

Founded year

2018

Operating status

Operating

Headcount range

11–50 employees

Short description

Epicrispr Biotechnologies is a clinical-stage biotechnology company developing epigenetic gene-modulating therapies for neuromuscular diseases via its proprietary GEMS platform, with lead candidate EPI-321 in Phase I trials for FSHD.

Ownership category

akta.pro rank

Product category

Gene Therapy / Epigenetic Therapeutics

NAICS

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

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Engineered Protein Therapeutics (de novo/AI-designed proteins, novel scaffolds) (HLAAAAAJ)

akta.pro secondary industries

Synthetic Biology & Genetic Circuit Design Platforms (pathway engineering, chassis development) (HLAAAIAD), Target Identification & Validation Platforms (omics-driven, chemoproteomics, genetic validation) (HLAAAIAI)

Keywords

• Epigenome editing therapeutics
• Gene modulation platform
• Clinical-stage biotechnology
• Rare disease therapeutics
• CRISPR-inspired medicines

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

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

Revenue model

1. Therapeutic Development and Commercialization: Pre-revenue clinical-stage biotechnology company developing gene therapies for neuromuscular diseases. Revenue model will depend on successful clinical development, regulatory approval, and commercialization of therapies like EPI-321 for FSHD.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels2 records

Core offering

Epicrispr Biotechnologies is a clinical-stage biotechnology company developing a pipeline of CRISPR-inspired epigenome editing therapeutics built on its proprietary GEMS (Gene Expression Modulation System) platform. Its lead candidate, EPI-321, is being developed for facioscapulohumeral muscular dystrophy (FSHD) and received FDA IND clearance in April 2025, with additional candidates EPI-331 and EPI-141 advancing through preclinical development.

Product overview

Epicrispr Biotechnologies is a clinical-stage biotechnology firm developing gene-modulating therapies for neuromuscular diseases using its proprietary Gene Expression Modulation System (GEMS) platform. The GEMS platform enables customized epigenetic therapies by combining epigenomic modulators. The company's product portfolio includes EPI-321 (lead candidate for FSHD, currently in Phase I trials), EPI-331, and EPI-141. The company is advancing programs focused on regulating gene expression through epigenetic mechanisms to treat complex diseases.

Differentiator

Problem solved

Functional benefit

Products and services

• EPI-321 Epicrispr's lead clinical-stage candidate, an epigenome modulating therapeutic built on the GEMS platform, in development for facioscapulohumeral muscular dystrophy (FSHD); received FDA IND clearance in April 2025.
• EPI-331 Pipeline candidate in Epicrispr's portfolio developed using the GEMS epigenome modulation platform for an undisclosed rare disease indication.
• EPI-141 Pipeline candidate in Epicrispr's portfolio developed using the GEMS epigenome modulation platform for an undisclosed rare disease indication.

Quantifiable outcome

• First clinical evidence of increased lean muscle volume in patients with FSHD following treatment with EPI-321

Segments1 record

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature2 records

Partnerships

One partnership is on record.

• Forge BiologicscoreTechnology or Integration · 6 May 2026Strategic partnership for AAV development and cGMP manufacturing of Epicrispr's investigational gene therapy EPI-321 for facioscapulohumeral muscular dystrophy (FSHD). Forge Biologics provides manufacturing and process development services, with all activities conducted at Forge's facility in Ohio, supporting clinical trials across the U.S., New Zealand, and Australia.

Scale indicators5 records

Recent moves6 records

Expansion highlights4 records

Direct peers

• Design Therapeutics: Clinical-stage biotech developing small-molecule and antisense approaches to treat serious genetic diseases driven by nucleotide-repeat expansions. Closely comparable to Epicrispr as both pursue epigenetic/genetic modulation of repeat-driven monogenic disease, with overlapping disease-target archetypes.
• Sangamo Therapeutics: Pioneer in zinc finger protein (ZFP)-based gene regulation and epigenetic editing for monogenic diseases. Directly comparable technology category to Epicrispr's engineered epigenomic modulators and operates in similar neuromuscular and CNS indications.
• Solid Biosciences: Clinical-stage gene therapy company developing AAV-delivered treatments for Duchenne muscular dystrophy and other neuromuscular indications. Directly comparable as a smaller, clinical-stage AAV gene therapy competitor in the same neuromuscular disease space.
• Fulcrum Therapeutics: Clinical-stage biotech developing small-molecule therapies for genetically defined diseases including facioscapulohumeral muscular dystrophy (FSHD) with losmapimod. A direct competitive peer in FSHD specifically — Epicrispr's primary indication.
• uniQure: Clinical-stage gene therapy company developing AAV-based treatments for rare and monogenic diseases including neuromuscular indications. Comparable modality (AAV gene therapy) and clinical-stage profile to Epicrispr.

Broad incumbents

• Sarepta Therapeutics: Established leader in gene therapy and RNA-based treatments for Duchenne muscular dystrophy (DMD) and other neuromuscular diseases. Comparable target patient population (neuromuscular) and disease-modifying gene-based modality, with deep commercial and regulatory infrastructure.
• Intellia Therapeutics: Clinical-stage CRISPR/Cas9-based gene editing company developing in vivo and ex vivo therapies for genetic diseases. Broader gene-modulation peer — different editing modality but comparable mission of treating monogenic disease through engineered genetic interventions.

Emerging players

• Capricor Therapeutics: Clinical-stage biotech developing cell therapy and exosome-based therapeutics for Duchenne muscular dystrophy and other neuromuscular diseases. Operates in the same neuromuscular patient population with a different but adjacent modality.
• Editas Medicine: Clinical-stage CRISPR-based gene editing company developing therapies for serious diseases including rare genetic disorders. Comparable as an emerging engineered-genetics therapeutic player, though focused on nuclease-based editing rather than epigenetic modulation.
• Prime Medicine: Clinical-stage biotech developing prime editing technology to make precise edits in the genome for genetic diseases. Comparable as an emerging engineered-gene-regulation platform peer with a pipeline spanning monogenic and complex genetic disorders.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• LinkedInlinkedin.com/company/epic-bio
• Xx.com/EpicrisprBio

Revenue estimate

Valuation estimate

Number of profiles

Profiles3 records

Funding overview

Funding rounds3 records

Investors11 records

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