HuidaGene Therapeutics

What HuidaGene Therapeutics does

HuidaGene Therapeutics Co., Ltd. is a Shanghai-headquartered, clinical-stage biotechnology company founded in 2018 that develops CRISPR-based genome medicines for ophthalmology, neurology, and neuromuscular diseases. The company's pipeline is powered by HG-PRECISE, an AI- and deep machine learning-driven platform that mines metagenomic DNA databases to discover and engineer proprietary compact, high-fidelity Cas nucleases. From this platform have emerged hfCas12Max (a single-AAV-packaged DNA editor protected by USPTO patent US11,649,444B1), hfCas13Y (a compact RNA editor), and Cas13X-based base editors. Four programs are in active clinical trials: HG004 (gene replacement therapy for RPE65-IRDs), HG202 (the first-ever FDA-cleared CRISPR/Cas13 RNA-editing therapy, for nAMD), HG204 (the first CRISPR RNA-editing therapy for a neurodevelopmental disease, for MECP2 duplication syndrome), and HG302 (the first CRISPR/DNA-editing trial beyond Cas9, for DMD). Preclinical assets extend the pipeline into ALS, Alzheimer's, Huntington's disease, and OTOF-related hearing loss, while HG301 targets RHO-associated retinitis pigmentosa and HG205 is the first Cas13X RNA base-editing therapy. Eight FDA Orphan Drug and Rare Pediatric Disease designations have been secured across four programs.

The company monetizes through a hybrid biotech model. It runs trials directly at tier-1 Chinese hospital sites (Xinhua Hospital, Peking University First Hospital, Shanghai Children's Medical Center, Tianjin Medical University Eye Institute, Shanghai Eye and ENT Hospital of Fudan University) and pursues multinational clinical execution, with HG004's STAR trial enrolling in both China and the U.S. Revenues today come primarily from upfront fees, milestones, and royalties on out-licensing of platform IP: hfCas12Max has been licensed to Synthego (manufacturing and commercialization), sublicensed through Synthego to Vita Therapeutics (cell therapy programs), and licensed to NovoCrops (agricultural crop applications) and Kactus Biosystems. Scientific credibility is reinforced through heavy publication output (Nature Methods 2021, Nature Biotechnology 2022, Nature Neuroscience, Protein & Cell) and a conference-first go-to-market approach spanning ASGCT, ESGCT, EURETINA, World Muscle Society, ARVO, BIO, and CRISPR MEDiCiNE. Customers in the buyer sense are pharmaceutical and ag-bio partners for licensing and co-development; ultimate end-users are patients with inherited retinal dystrophies, nAMD, MECP2 duplication syndrome, and Duchenne muscular dystrophy.

Name

HuidaGene Therapeutics

Legal name

HuidaGene Therapeutics Co., Ltd.

Website

https://huidagene.com

Company type

Private

Founded year

2018

Operating status

Operating

Headcount range

51–100 employees

Short description

HuidaGene Therapeutics is a Shanghai-based, clinical-stage biotechnology company founded in 2018 that develops CRISPR-based gene and RNA-editing therapies — using its AI-driven HG-PRECISE platform and proprietary hfCas12Max and hfCas13Y nucleases — for patients with inherited retinal dystrophies, nAMD, MECP2 duplication syndrome, Duchenne muscular dystrophy, and other neurological and neuromuscular diseases.

Ownership category

akta.pro rank

Product category

Gene Therapy / Biotechnology Pharmaceuticals

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414)

SIC

Biological Products, (No Disgnostic Substances) (2836), Services-Commercial Physical & Biological Research (8731), Patent Owners & Lessors (6794)

akta.pro industry

PRIMARYHLAAACAD

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

Healthcare & Life Sciences/Biotechnology & Biologics/Gene Therapy (AAV/lentiviral/other vectors)

SECONDARYHLAAACAH

Neurology/CNS Gene Therapies

Healthcare & Life Sciences/Biotechnology & Biologics/Gene Therapy (AAV/lentiviral/other vectors)

SECONDARYHLAAACAE

Gene Therapy for Rare/Monogenic Diseases

Healthcare & Life Sciences/Biotechnology & Biologics/Gene Therapy (AAV/lentiviral/other vectors)

SECONDARYHLAAACAL

Gene Therapy Delivery Devices & Administration Systems (catheters, intrathecal, ocular delivery)

Healthcare & Life Sciences/Biotechnology & Biologics/Gene Therapy (AAV/lentiviral/other vectors)

SECONDARYHLAAAIAG

CRISPR & Functional Genomics Screening Platforms (pooled/arrayed screens, perturb-seq)

Healthcare & Life Sciences/Biotechnology & Biologics/Biologic Drug Discovery Platforms (screening, antibody engineering, synthetic biology)

SECONDARYHLAAACAN

Gene Therapy CMC/Process Development Platforms (capsid engineering, producer cell lines, plasmids, upstream/downstream)

Healthcare & Life Sciences/Biotechnology & Biologics/Gene Therapy (AAV/lentiviral/other vectors)

Keywords

• CRISPR gene therapy
• Gene editing platform
• AAV delivery vectors
• RNA editing therapeutics
• Rare disease biologics

Headquarters

HQ city

Shanghai

HQ country

China

HQ region

Asia

Offices2 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Gene therapy development and commercialization: As a clinical-stage biotech, HuidaGene is developing a pipeline of CRISPR-based gene therapies across ophthalmology, neurology, and neuromuscular diseases. Revenue will be generated through future product sales upon regulatory approval. The company also generates upfront, milestone, and royalty payments through licensing agreements.
2. Licensing and sublicensing of CRISPR technology platforms: HuidaGene generates revenue through technology licensing agreements, sublicensing arrangements, and royalty payments. Examples include licensing hfCas12Max to Synthego (manufacturing and commercialization rights) and sublicensing through Synthego to Vita Therapeutics for clinical cell therapy programs. NovoCrops licensing agreement for agricultural applications of hfCas12Max also generates upfront, milestone, and royalty payments.

Go-to-market motion2 records

Distribution channels2 records

Marketing channels5 records

Core offering

HuidaGene develops CRISPR-based gene therapy clinical candidates (HG004, HG202, HG204, HG302, HG205, HG303) for ophthalmology, neurology, and neuromuscular diseases, using its proprietary AI-driven HG-PRECISE platform to engineer high-fidelity DNA and RNA editors (hfCas12Max, hfCas13Y) and a single AAV vector delivery platform. The company monetizes through future product sales upon regulatory approval and through licensing/sublicensing of its CRISPR technology platforms to pharmaceutical, cell therapy, and agricultural partners.

Product overview

HuidaGene Therapeutics is a global clinical-stage biotechnology company that has developed a comprehensive portfolio of CRISPR-based gene therapies and enabling technology platforms. The Company's clinical programs include HG004 (gene replacement therapy for RPE65-associated inherited retinal dystrophies), HG202 (CRISPR/Cas13 RNA-editing therapy for neovascular AMD - the first-ever FDA-cleared CRISPR/Cas13 therapy), HG204 (RNA-editing therapy for MECP2 Duplication Syndrome - world's first CRISPR/RNA-editing therapy for neurodevelopmental disease), and HG302 (DNA-editing therapy for Duchenne muscular dystrophy - world's first CRISPR/DNA-editing trial beyond Cas9). These programs are powered by the Company's proprietary AI-driven HG-PRECISE platform, which has enabled the discovery of high-fidelity nucleases including hfCas12Max (DNA editing) and hfCas13Y (RNA editing). The Company also has preclinical programs including HG303 for ALS and CRISPR RNA-editing therapies for Alzheimer's and Huntington's Disease.

Differentiator

Problem solved

Functional benefit

Products and services

• HG004 Gene replacement therapy using AAV9 vector to deliver functional human RPE65 gene for treatment of inherited retinal dystrophies caused by RPE65 mutations (RPE65-IRDs), including Leber congenital amaurosis. Conducted at Xinhua Hospital (LIGHT first-in-human trial NCT06088992) and multinational STAR Phase 1/2 trial (NCT05906953).
• HG202 CRISPR/Cas13 RNA-editing therapy using hfCas13Y to knockdown VEGF-A mRNA for treatment of neovascular age-related macular degeneration (nAMD); the first-ever FDA-cleared CRISPR/Cas13 RNA-editing therapy (BRIGHT Phase 1 NCT06623279) with SIGHT-I first-in-human trial (NCT06031727) already underway.
• HG204 CRISPR RNA-editing therapy using hfCas13Y to reduce MECP2 mRNA and protein levels for treatment of MECP2 Duplication Syndrome (MDS); world's first CRISPR/RNA-editing therapy for neurodevelopmental disease; HERO first-in-human trial (NCT06615206) with FDA ODD/RPDD and EMA ODD.
• HG302 CRISPR DNA-editing therapy using hfCas12Max to precisely edit DMD exon 51 splice-donor site, enabling exon skipping to restore dystrophin production for treatment of Duchenne muscular dystrophy (DMD); world's first CRISPR/DNA-editing trial beyond Cas9; MUSCLE first-in-human trial (NCT06594094) with FDA ODD/RPDD.
• HG205 World's first Cas13X RNA base-editing therapy (using mini-dCas13X mxABE) for treatment of congenital hearing loss caused by OTOF gene Q829X mutation; received FDA ODD and RPDD.

Quantifiable outcome

• HG302 demonstrated strong safety with no SAEs, DLT, or major toxicities including thrombocytopenia, myocarditis, and thrombotic microangiopathy, with early functional gains (+1 North Star Ambulatory Assessment point, +60 meters in 6-minute walk test, 30 seconds improvement in 4-stair climb at 3 months post-treatment)
• +4 more outcomes

Named customers4 records

Segments3 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

AI capability2 records

Feature8 records

Partnerships

Ten partnerships are on record, tiered core and strategic.

• Vita Therapeutics, Inc.coreTechnology or Integration · 9 April 2025Through Synthego sublicensing agreement, Vita Therapeutics gained access to HuidaGene's engineered hfCas12Max nuclease and optimized guide RNAs for preclinical and clinical applications, including development of hypoimmunogenic iPSC-based cell therapies targeting neuromuscular disorders. This sublicensing milestone underscores the broad applicability of HuidaGene's proprietary hfCas12Max platform.
• NovoCrops BiotechnologystrategicTechnology or Integration · 11 June 2024Licensing partnership for application of hfCas12Max in developing gene-editing crop products. NovoCrops obtains a global license of hfCas12Max in multiple main crops development and production. HuidaGene receives license fees including upfront, milestone, and royalty payments. NovoCrops is a leading multi-crop gene-editing biological breeding company based in China with a state-of-the-art genetic transformation platform.
• Synthego CorporationcoreTechnology or Integration · 29 May 2024HuidaGene granted Synthego manufacturing and commercialization rights for hfCas12Max nuclease and optimized gRNA for research use, along with the right to sublicense for therapeutic applications. Synthego is a leading provider of innovative CRISPR solutions for developing cell and gene therapies with advanced GMP manufacturing capabilities. This partnership provides increased freedom-to-operate and facilitates development of CRISPR-based cell and gene therapies globally.
• Shanghai Institute of Materia Medica, Chinese Academy of SciencescoreStrategic or Co-development PartnerJoint research collaboration on HG204 preclinical research leading to landmark publication in Nature Neuroscience. The collaboration involved HuidaGene scientists working with researchers from Shanghai Institute of Materia Medica, Chinese Academy of Sciences, and Lingang Laboratory.
• Kactus BiosystemsstrategicTechnology or IntegrationStrategic collaboration and license agreement to promote commercialization of next-generation gene editing enzyme hfCas12Max, announced as part of HuidaGene's efforts to expand adoption of its proprietary CRISPR tools.
• Xinhua Hospital affiliated with Shanghai Jiao Tong University School of MedicinecoreStrategic or Co-development PartnerClinical trial partnership for HG004 LIGHT first-in-human trial for Leber Congenital Amaurosis and RPE65-IRDs. Led by Prof. Peiquan Zhao, Director of Ophthalmology. Trial site has demonstrated substantial vision restoration with no serious adverse events in both adult and pediatric populations.
• Tianjin Medical University Eye InstitutecoreStrategic or Co-development PartnerClinical trial site for HG202 SIGHT-I clinical trial for neovascular age-related macular degeneration, led by Prof. XiaoRong Li. Site was first to dose the world's first CRISPR/Cas13 RNA-editing therapy in humans.
• Shanghai Eye and ENT Hospital of Fudan UniversitycoreStrategic or Co-development PartnerClinical trial site for HG202 SIGHT-I clinical trial for neovascular age-related macular degeneration, contributing to China's first CRISPR/Cas13 RNA-editing therapy clinical development.
• Shanghai Children's Medical Center affiliated with Shanghai Jiao Tong University School of MedicinecoreStrategic or Co-development PartnerClinical trial site for HG302 MUSCLE clinical trial for Duchenne muscular dystrophy. Open-label, multidose dose-escalation, first-in-human study enrolling ambulatory boys between 4 and 8 years old with DMD.
• Peking University First HospitalcoreStrategic or Co-development PartnerClinical trial site for HERO clinical trial evaluating HG204 for MECP2 duplication syndrome, the world's first clinical evaluation of an RNA-editing therapy for MDS.

Scale indicators6 records

Recent moves7 records

Expansion highlights6 records

Direct peers

• CRISPR Therapeutics: Clinical-stage CRISPR gene editing company that developed Casgevy, the first FDA-approved CRISPR therapy. Direct competitor in CRISPR-based gene editing, with overlapping Cas9-based platforms and similar pharma partnership strategy. HuidaGene's CTO previously contributed to Casgevy at CRISPR Therapeutics.
• Intellia Therapeutics: Leading in vivo CRISPR gene editing company with both Cas9 and base editing programs in clinical trials. Directly comparable to HuidaGene on in vivo editing modality and pipeline breadth across rare diseases.
• Editas Medicine: CRISPR-based gene editing company developing Cas9 and Cas12a therapies for ophthalmology, hematology, and oncology. Directly comparable on ophthalmology pipeline overlap (Editas has reni-cel) and Cas12a technology that overlaps with HuidaGene's hfCas12Max.
• Beam Therapeutics: Pioneer in base editing technology, with clinical programs in sickle cell disease, alpha-1 antitrypsin deficiency, and other indications. Direct competitor given HuidaGene's own base editor development and overlapping rare disease focus.
• Verve Therapeutics: In vivo gene editing company developing base editing therapies for cardiovascular disease. Directly comparable on in vivo editing modality, single-AAV/LNP delivery, and clinical-stage profile.

Emerging players

• Prime Medicine: Clinical-stage prime editing company with broad pipeline across ophthalmology, liver, and CNS indications. Comparable on next-generation editing technology and ophthalmology overlap, though differentiated by prime editing architecture.
• Korro Bio: Clinical-stage RNA editing company developing ADAR-based therapies for alpha-1 antitrypsin deficiency and other indications. Closest direct competitor to HuidaGene's RNA-editing programs (HG202, HG204), with similar therapeutic modality.
• ProQR Therapeutics: RNA editing and RNA-targeting therapy company developing Axiomer platform for rare diseases including CNS and ophthalmology indications. Comparable on RNA-targeting therapeutic approach and rare disease focus.
• Generation Bio: Non-viral gene therapy company developing LNP- and AAV-delivered therapies for rare diseases. Comparable on alternative delivery technology focus and rare disease pipeline, though differentiated by non-viral approach.

Broad incumbents

• Sangamo Therapeutics: Pioneer in genome editing (ZFN, TALEN) with broader portfolio spanning gene therapy, cell therapy, and ex vivo editing. HuidaGene's CTO previously held positions at Sangamo, and both companies target similar indications with in vivo editing.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• Xtwitter.com/HuidaGene
• LinkedInlinkedin.com/company/huidagene

Compliance2 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles7 records

Funding overview

Funding rounds2 records

Investors9 records

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M&A

Investments

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Frequently asked questions about HuidaGene Therapeutics