EdiGene

What EdiGene does

EdiGene (博雅辑因) is a clinical-stage biotechnology company founded in 2015 in Beijing by Professor Wei Wensheng of Peking University, focused on translating genome editing technologies into therapies for genetic diseases and cancer. The company operates four complementary technology platforms: LEAPER (in vivo RNA base editing via engineered arRNA recruiting endogenous ADAR proteins; LEAPER 2.0 published in Nature Biotechnology 2022 uses circular RNA to improve efficiency), MAGIC (an allogeneic cell therapy platform based on a single SPPL3 gene knockout that enables immune-compatible universal CAR-T by altering glycosylation while preserving TCR function), ex vivo CRISPR/Cas9 hematopoietic stem cell editing (lead product ET-01 reactivates fetal hemoglobin via BCL11A enhancer editing for transfusion-dependent beta-thalassemia), and a high-throughput genome editing screening platform capable of systematically knocking out ~20,000+ genes in 1 billion cells within 3–4 weeks.

The company's lead clinical asset ET-01 received China's first NMPA-approved gene editing therapy IND and first hematopoietic stem cell therapy IND in January 2021; the multi-center Phase I trial completed last patient infusion in November 2023. Pipeline programs also include SPPL3KO allogeneic CAR-T for relapsed/refractory B-cell lymphoma and autoimmune diseases (systemic lupus erythematosus), and LEAPER-based in vivo RNA editing therapies for Duchenne muscular dystrophy and Usher syndrome type II. In August 2025, EdiGene and collaborators published in Cell the world's first clinical proof-of-concept for a single-gene-edited TCR-sufficient allogeneic CAR-T, demonstrating >6 months persistence with durable tumor control and no GvHD.

EdiGene is pre-revenue with no commercially approved products. The historical business model included a cell line services business that was divested to a UK buyer in 2019; current operations focus exclusively on therapeutic development. Future revenue is intended to come from commercialization of approved gene editing therapies in China and partner markets, supplemented by pharma collaboration income from the screening platform. The company employs 200+ staff across four cities (Beijing HQ, Guangzhou cGMP manufacturing and clinical center, Cambridge MA US R&D, Shanghai business development office) and has raised over 1.1 billion CNY in cumulative disclosed venture financing from IDG Capital, Lilly Asia Venture, Sequoia China, 3H Health Investment, Loyal Valley Capital, and others. Distribution, upon approval, will be hospital-based specialty infusion typical for cell and gene therapies.

Name

EdiGene

Legal name

博雅辑因(北京)生物科技有限公司

Website

https://edigene.com

Company type

Private

Founded year

2015

Operating status

Operating

Headcount range

101–250 employees

Short description

EdiGene is a clinical-stage Chinese biotechnology company developing gene editing therapies across four platforms — LEAPER RNA editing, MAGIC allogeneic CAR-T, ex vivo hematopoietic stem cell editing, and high-throughput screening — with ET-01 being China's first NMPA-approved gene editing therapy for transfusion-dependent beta-thalassemia.

Ownership category

akta.pro rank

Product category

Gene Therapy

NAICS

Biological Product (except Diagnostic) Manufacturing (325414), 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

Cell Therapy Enabling Technologies (gene editing, cell engineering, delivery methods) (HLAAABAL), CRISPR & Functional Genomics Screening Platforms (pooled/arrayed screens, perturb-seq) (HLAAAIAG), RNA Therapeutics Clinical Development (trial design, endpoints, patient stratification) (HLAAADAH), Gene Therapy Delivery Devices & Administration Systems (catheters, intrathecal, ocular delivery) (HLAAACAL)

Keywords

• Gene editing therapies
• CRISPR therapeutics
• Cell therapy
• RNA base editing
• Biopharmaceutical research

Headquarters

HQ city

Beijing

HQ country

China

HQ region

Asia

Offices4 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Therapeutic product development and commercialization (future): EdiGene is a clinical-stage biotech company that generates no commercial revenue currently. It is developing gene editing therapies for genetic diseases and cancer, with its lead product ET-01 in Phase I clinical trials. The company plans to commercialize its pipeline products upon regulatory approval.
2. Technology services and platform collaborations (historical): Early in its history, EdiGene operated a service-based business model providing genome editing services and high-throughput screening to pharma partners. The company sold its cell line business (2,000+ gene knockout cell lines) to a UK company in 2019 and pivoted to focus exclusively on therapeutic development.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels4 records

Core offering

EdiGene is a clinical-stage biotechnology company developing gene editing-based therapeutics across four proprietary platforms: LEAPER RNA base editing, MAGIC allogeneic cell therapy via SPPL3 knockout, ex vivo CRISPR/Cas9 hematopoietic stem cell editing, and high-throughput genome-wide screening. Its lead asset ET-01 is an autologous CRISPR/Cas9-edited CD34+ hematopoietic stem cell therapy that received China's first NMPA gene editing IND (January 2021) for transfusion-dependent beta-thalassemia. The company also advances allogeneic CAR-T therapies for hematologic malignancies and autoimmune diseases, plus in vivo LEAPER-based RNA editing programs for Duchenne muscular dystrophy, Usher syndrome type II, and alpha-1 antitrypsin deficiency.

Product overview

EdiGene is a clinical-stage biopharmaceutical company developing gene editing-based therapeutics through four technology platforms: LEAPER (in vivo RNA base editing), MAGIC (allogeneic cell therapy with single-gene SPPL3 knockout), hematopoietic stem cell ex vivo editing, and high-throughput genome editing screening. The lead product ET-01 is China's first approved CRISPR gene-editing therapy for β-thalassemia. The company is also advancing allogeneic CAR-T therapies for hematologic malignancies and autoimmune diseases, as well as in vivo RNA editing programs for Duchenne muscular dystrophy and Usher syndrome.

Differentiator

Problem solved

Functional benefit

Products and services

• ET-01 Autologous, ex vivo CRISPR/Cas9 gene-modified CD34+ hematopoietic stem progenitor cell injection for the treatment of transfusion-dependent beta-thalassemia. Patient hematopoietic stem cells are collected, CD34+ cells enriched, the BCL11A erythroid enhancer edited via CRISPR/Cas9, and the edited cells re-infused to reactivate fetal hemoglobin expression. Designed as a potential one-time curative therapy applicable across transfusion-dependent beta-thalassemia patients regardless of specific mutation.
• MAGIC Allogeneic Anti-CD19 CAR-T (SPPL3KO) for Hematologic Malignancies Allogeneic CAR-T cell therapy targeting CD19 for relapsed/refractory non-Hodgkin B-cell lymphoma using the proprietary MAGIC platform's single SPPL3 gene knockout technology. The SPPL3 knockout enables glycan shielding to evade host immune rejection while retaining TCR function, solving both GvHD and HvG bottlenecks of allogeneic CAR-T.
• MAGIC Allogeneic CAR-T for Autoimmune Diseases (SLE) Allogeneic CAR-T cell therapy using SPPL3 knockout via the MAGIC platform for autoimmune diseases including systemic lupus erythematosus (SLE). Designed as an off-the-shelf cell therapy leveraging the same immune-compatibility mechanism as the oncology programs.
• LEAPER-based In Vivo RNA Editing Therapy for Duchenne Muscular Dystrophy (DMD) In vivo RNA base editing therapy for Duchenne muscular dystrophy using the LEAPER platform to correct point mutations underlying the disease. Leverages arRNA-mediated recruitment of endogenous ADAR for precise A-to-I editing without introducing exogenous enzymes.
• LEAPER-based In Vivo RNA Editing Therapy for Usher Syndrome Type II In vivo RNA base editing therapy for Usher syndrome type II (inherited retinal degeneration) using the LEAPER platform to target specific disease-causing mutations. Co-developed with Peking Union Medical College Hospital based on genetic variation characteristics of Chinese IRD patients.
• LEAPER RNA Base Editing Platform Proprietary in vivo RNA base editing platform using engineered arRNA to recruit endogenous ADAR proteins for precise A-to-I editing. LEAPER 2.0 uses circular RNA design to enhance editing efficiency and reduce off-target effects. The platform is offered for partnerships and licensing deals with biotech and pharma collaborators.
• MAGIC Allogeneic Cell Therapy Platform Proprietary allogeneic cell therapy platform based on single SPPL3 gene knockout, discovered through genome-wide CRISPR screening. Enables immune-compatible off-the-shelf cell products via glycan shielding that protects from host immune rejection while retaining TCR function. The platform supports internal pipeline products and external partnerships.

Quantifiable outcome

• First patient infused in ET-01 Phase I clinical trial in September 2021 at China Academy of Medical Sciences Blood Disease Hospital
• +3 more outcomes

Named customers1 record

Segments3 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Seven partnerships are on record, tiered medium and high.

• Peking University Cancer Hospital (北京大学肿瘤医院)mediumStrategic or Co-development Partner · 13 April 2022EdiGene entered a research collaboration with Professor Shen Lin's team at Peking University Cancer Hospital to analyze gene mutation frequencies and clinical-pathological characteristics in Chinese advanced colorectal cancer patients, to advance research on gene mutations related to targeted therapy response and provide a foundation for precision treatment of gene mutation-pre-screened patient groups.
• Cell Ecosystem Haihe Laboratory (细胞生态海河实验室)highStrategic or Co-development Partner · 1 January 2022Strategic R&D collaboration leveraging respective technical and resource advantages to jointly advance hematopoietic stem cell R&D translation and gene therapy technologies and products, building China's original innovation research and clinical translation platform for hematopoietic stem cell therapy. Collaboration includes developing innovative gene-modified hematopoietic stem cell therapies for specific Chinese hematological genetic diseases and exploring new hematopoietic stem cell biomarkers to optimize production quality control processes.
• Peking Union Medical College Hospital (北京协和医院)mediumStrategic or Co-development Partner · 1 November 2021Research collaboration with Professor Sui Ruifang's team at Peking Union Medical College Hospital to advance in vivo gene editing therapy, based on the genetic variation characteristics of Chinese inherited retinal degeneration (IRD) patient population.
• University of Wisconsin-Madison (威斯康星大学麦迪逊分校)mediumStrategic or Co-development Partner · 1 November 2021Research collaboration with University of Wisconsin-Madison to advance in vivo gene editing therapy development.
• Boston Children's Hospital (波士顿儿童医院)mediumTechnology or Integration · 1 March 2021EdiGene entered a global license agreement with Boston Children's Hospital to access certain intellectual property for gene therapy development.
• Arbor BiotechnologiesmediumTechnology or Integration · 1 February 2021EdiGene entered a global non-exclusive license agreement with US-based Arbor Biotechnologies, expanding its access to proprietary genome editing tools.
• Yimiao Shenzhou (艺妙神州)highStrategic or Co-development Partner · 1 May 2020EdiGene and Yimiao Shenzhou entered a collaboration to jointly develop allogeneic CAR-T cell therapy for cancer treatment. EdiGene is responsible for using gene editing to remove immune-rejection molecules on healthy donor T cells and then expanding them to maintain tumor-killing activity; Yimiao Shenzhou is responsible for designing and arming T cells with CAR molecules, involving design, production, and other complex processes. This is the first cooperation model of its kind in China for CAR-T development.

Scale indicators6 records

Recent moves9 records

Expansion highlights6 records

Direct peers

• CRISPR Therapeutics: CRISPR Therapeutics is the most direct peer - a clinical-stage gene editing company whose Casgevy (exa-cel) became the first FDA-approved CRISPR therapy for sickle cell disease and beta-thalassemia, directly competing with EdiGene's ET-01 in the same indication.
• Editas Medicine: Editas Medicine develops CRISPR/Cas9 and CRISPR/Cas12a gene editing therapies for genetic diseases including sickle cell and beta-thalassemia, overlapping directly with EdiGene's core CRISPR platform and ET-01 indication.
• Intellia Therapeutics: Intellia is a leading in vivo and ex vivo CRISPR gene editing company developing NTLA-2001 for transthyretin amyloidosis and other programs, with overlapping gene editing modality and ex vivo/in vivo therapeutic strategy as EdiGene.
• Beam Therapeutics: Beam Therapeutics is a base editing pioneer developing precision genetic medicines using adenine and cytosine base editors - directly comparable to EdiGene's LEAPER RNA base editing platform and ex vivo beta-thalassemia programs.
• Caribou Biosciences: Caribou Biosciences develops allogeneic CAR-T cell therapies using CRISPR gene editing - closely comparable to EdiGene's MAGIC platform and SPPL3KO allogeneic CAR-T program for hematologic malignancies.
• Allogene Therapeutics: Allogene is a clinical-stage allogeneic CAR-T company developing off-the-shelf cell therapies for hematologic malignancies, directly competing with EdiGene's MAGIC platform allogeneic CAR-T candidates.
• Precision BioSciences: Precision BioSciences develops in vivo gene editing therapies using its proprietary ARCUS platform and allogeneic CAR-T programs - comparable gene editing modality and cell therapy pipeline overlap with EdiGene.
• Verve Therapeutics: Verve Therapeutics is a clinical-stage company developing in vivo gene editing medicines for cardiovascular disease using base editing - directly comparable to EdiGene's LEAPER in vivo RNA editing approach for monogenic diseases.

Regional players

• HuidaGene Therapeutics: HuidaGene is a Shanghai-based clinical-stage gene editing company developing RNA editing and CRISPR therapies for rare diseases - a direct Chinese peer to EdiGene operating in the same modality space with overlapping monogenic disease programs.
• Correctsequence Therapeutics: Correctsequence Therapeutics (精缮科技) is a China-based gene editing company developing in vivo CRISPR therapies for genetic diseases and ophthalmology indications - a regional Chinese peer with comparable technology platform and target patient populations.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• Xx.com/Edigene_Inc
• LinkedInlinkedin.com/company/edigene

Compliance4 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles4 records

Subsidiaries1 record

Funding overview

Funding rounds7 records

Investors14 records

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M&A

Investments

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Frequently asked questions about EdiGene