What Epigenic Therapeutics does

Epigenic Therapeutics is a clinical-stage biotechnology company founded in 2021 and headquartered in Shanghai's Pilot Free Trade Zone, focused on developing next-generation gene modulation therapies through epigenetic regulation rather than DNA cleavage. The company's core technology is the EPIREG™ platform, which employs nuclease-inactivated dead Cas protein combined with epi-enzymes to precisely control gene expression via DNA methylation and histone modification without cutting, nicking, or altering DNA sequences. This is supported by two complementary platforms: EpiTax™, an in-vivo lipid nanoparticle (LNP) delivery system with tissue tropism for delivery to liver and extrahepatic organs, and AIAID™, a self-developed AI platform featuring an epigenetic foundational model and multi-agent virtual lab for molecular design, target selection, and optimization. The company's pipeline centers on two clinical-stage assets: EPI-003, a first-in-class epigenetic inactivator targeting HBV cccDNA and integrated DNA for functional cure of chronic hepatitis B (currently in Phase I trials across New Zealand, Australia, Hong Kong, with US FDA and China NMPA INDs cleared), and EPI-001, an epigenetic inactivator targeting PCSK9 for hypercholesterolemia.

The business model is pre-revenue pharmaceutical development with future revenue contingent on regulatory approval and commercialization of pipeline drugs. The company does not currently sell products; revenue is expected to derive from pharmaceutical product sales and licensing upon commercialization, with pricing yet to be determined. Go-to-market involves multi-jurisdisdiction clinical trial operations, regulatory submissions across Western and Asia-Pacific markets, and likely future pharmaceutical partnerships for commercialization. The customer base in the near term consists of chronic hepatitis B patients and hypercholesterolemia/ASCVD patients, with future expansion into oncology (CAR-T enhancement), ophthalmology, and CNS disease populations through platform applications. The company has raised approximately $115 million across four funding rounds (Angel: $3M in 2021, Pre-A: $20M in 2022, Series A: $32M in 2023, Series B: $60M in September 2025) from top-tier healthcare investors including Morningside Venture Capital, Qiming Venture Partners, OrbiMed, and Lapam Capital. Headcount is in the 101-250 range, with recent senior leadership additions including a CMO with prior pharma executive experience and a CFO who previously served as a senior biotech equity research analyst at Jefferies.

Name

Epigenic Therapeutics

Legal name

Epigenic (Shanghai) Biotechnology Co., Ltd.

Website

https://epigenictx.com

Company type

Private

Founded year

2021

Operating status

Operating

Headcount range

101–250 employees

Short description

Epigenic Therapeutics is a clinical-stage Shanghai-based biotechnology company developing gene modulation therapies via its proprietary EPIREG™ epigenetic platform that controls gene expression without DNA alteration, with two lead programs — EPI-003 for chronic hepatitis B and EPI-001 for hypercholesterolemia — currently in Phase I trials.

Ownership category

akta.pro rank

Product category

Epigenetic Biopharmaceuticals

NAICS

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

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Genome Editing Gene Therapies (CRISPR/TALEN/ZFN; in vivo/ex vivo) (HLAAACAD)

akta.pro secondary industries

Cardiometabolic & Other Systemic Gene Therapies (HLAAACAI), Protein Engineering & Directed Evolution Platforms (enzyme/therapeutic protein optimization) (HLAAAIAC)

Keywords

• Epigenetic gene therapy
• Epigenetic modulation platform
• Lipid nanoparticle delivery
• Clinical-stage biotechnology
• Gene regulation therapeutics

Headquarters

HQ city

Shanghai

HQ country

China

HQ region

Asia

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Personnel, Technology or R&D, Operations, Marketing or Sales, Infrastructure

Revenue model

1. Pharmaceutical Product Sales: Pre-revenue clinical-stage biotech company developing epigenetic medicines. Revenue expected from sales of proprietary drugs upon regulatory approval and commercialization. Pipeline includes EPI-003 for chronic hepatitis B and EPI-001 for hypercholesterolemia currently in clinical development.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels4 records

Core offering

Epigenic Therapeutics is a clinical-stage biotechnology company developing gene modulation therapies through epigenetic regulation. The company's proprietary EPIREG™ platform uses nuclease-inactivated dead Cas protein and epi-enzymes to precisely control gene expression without cutting, nicking, or altering DNA sequences, enabling safe and durable gene silencing. Its clinical pipeline includes EPI-003, a first-in-class epigenetic inactivator for chronic hepatitis B (Phase I trials in multiple regions) and EPI-001, an epigenetic PCSK9 inactivator for hypercholesterolemia.

Product overview

Epigenic Therapeutics is a clinical-stage biotechnology company offering a suite of proprietary technology platforms for epigenetic gene modulation therapy. The core EPIREG™ platform enables precise control of gene expression through DNA methylation and histone modification without altering DNA sequences. This platform is supported by the EpiTax™ in-vivo LNP delivery system for targeted organ delivery and the AIAID™ AI platform for drug discovery optimization. The company's pipeline includes EPI-001 for hypercholesterolemia (targeting PCSK9) and EPI-003 for chronic hepatitis B, both currently in clinical development. Additional technology platforms cover epigenetic modulation, AI-driven drug discovery, nano-delivery systems, and immune/stem cell applications for CAR-T enhancement.

Differentiator

Problem solved

Functional benefit

Brands

• EPIREG™: Proprietary technology platform that employs AI algorithms to obtain innovative CRISPR-Cas components to regulate target gene expression without changing DNA sequence, combined with LNP delivery system.
• EpiTax™
• AIAID™

Products and services

• EPI-003 First-in-class epigenetic inactivator for the treatment of chronic hepatitis B virus (HBV) infection; designed to block transcription of HBV cccDNA and integrated HBV DNA through epigenetic modification (DNA methylation and histone modification) to eliminate HBsAg and induce serological conversion, targeting functional or complete cure. Currently in Phase I clinical trials in New Zealand, Australia, and Hong Kong, with planned expansion to mainland China.
• EPI-001 Epigenetic inactivator targeting the PCSK9 gene for the treatment of hypercholesterolemia and atherosclerotic cardiovascular disease (ASCVD); designed to provide durable LDL cholesterol (LDL-C) reduction through single-course gene silencing via EPIREG™ technology and EpiTax™ LNP delivery. Preclinical data in non-human primates demonstrated approximately 90% PCSK9 suppression and approximately 60% LDL-C reduction lasting over 343 days from a single dose.
• EPIREG™ Epigenetic Modulator Platform Proprietary epigenetic modulation technology platform that utilizes nuclease-inactivated dead Cas protein and epi-enzymes to precisely control gene expression without DNA cutting, nicking, or altering, achieving stable and heritable gene regulation through DNA methylation and histone modification. Serves as the foundational technology for Epigenic's drug pipeline.
• EpiTax™ LNP Delivery Platform In-vivo lipid nanoparticle (LNP) delivery platform that precisely delivers EPIREG™ epigenetic tools to the liver as well as multiple extrahepatic organs; based on non-viral LNP carriers with tissue tropism and cellular targeting properties, conferring low toxicity and organ specificity.
• AIAID™ AI Platform AI-powered platform for molecular design, target selection, and precise modulation of epigenetic medicine; equipped with a self-developed foundational model for epigenetics and a multi-agent collaborative virtual lab performing target discovery, target investigation, sgRNA design and evaluation, virtual epigenetic modulation, and multi-omics analysis; uses AI-driven de novo protein design and AI-directed evolution.

Quantifiable outcome

• Approximately 90% PCSK9 gene suppression and approximately 60% LDL-C reduction in non-human primates with single 2.25mg/kg dose of EpiReg-T, lasting over 343 days without reversal
• +2 more outcomes

Named customers1 record

Segments4 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

AI capability4 records

Feature6 records

Partnerships

One partnership is on record.

• Bayer Co.LabcoreStrategic or Co-development Partner · 10 December 2024Epigenic joined Bayer Co.Lab in China as a resident company, gaining access to Bayer's global network, expertise, and resources to accelerate breakthrough innovations in epigenetic modulation technology. Bayer Co.Lab provides state-of-the-art infrastructure, lab space, and access to Bayer's global R&D network.

Scale indicators10 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• Tune Therapeutics: US-based epigenetic editing company developing programmable gene silencing/tuning therapeutics using CRISPR-based epi-effectors; most direct competitor to Epigenic's EPIREG platform approach.
• Beam Therapeutics: Pioneer in base editing with multiple clinical programs targeting liver and rare diseases; comparable as a programmable in vivo gene editing platform using LNP delivery, competing for similar patient populations.
• Verve Therapeutics: Lead clinical asset VERVE-101/102 targets PCSK9 for hypercholesterolemia using in vivo base editing; direct competitor to EPI-001 in the durable PCSK9 silencing space with overlapping one-and-done treatment thesis.
• Intellia Therapeutics: Clinical-stage in vivo CRISPR/Cas9 gene editing company with programs in transthyretin amyloidosis and HAE; comparable as a competing in vivo gene modulation platform targeting liver-expressed genes.
• Chroma Medicine: Clinical-stage epigenetic editing company building a pipeline of single-dose gene tuning medicines; directly comparable to Epigenic in mechanism (epigenetic modulation without DNA cleavage) and stage.

Broad incumbents

• CRISPR Therapeutics: Leading commercial-stage CRISPR gene editing company with approved Casgevy and broad pipeline including in vivo programs; broad-incumbent benchmark for the gene editing competitive landscape.
• Editas Medicine: Pioneer CRISPR-based gene editing company with clinical programs in sickle cell disease and ocular indications; broader-incumbent comparator operating across in vivo and ex vivo gene editing modalities.
• Sangamo Therapeutics: Clinical-stage genomic medicine company developing zinc finger and adeno-associated virus-based in vivo gene regulation therapies; comparable for genomic medicine platform ambition and clinical-stage execution.

Emerging players

• Arbutus Biopharma: Clinical-stage HBV therapeutics developer focused on functional cure via RNAi and oral capsid inhibitors; direct competitive overlap with EPI-003 in the chronic HBV functional cure race.

Regional players

• Brii Biosciences: China-headquartered clinical-stage company developing siRNA-based HBV functional cure therapies; directly comparable China-based peer pursuing the same chronic HBV cure indication as EPI-003.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks5 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/epigenic-therapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles3 records

Funding overview

Funding rounds5 records

Investors9 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 Epigenic Therapeutics