VC Gene Therapy
VC Gene Therapy (VCGT Inc.) is a Japanese pre-clinical biotech developing ZFN-based genome editing and HITI gene insertion therapies for autosomal dominant retinitis pigmentosa caused by RHO gene mutations, targeting ~100,000 patients worldwide.
- Company typePrivate
- Founded2020
- HeadquartersKobe, Japan
- Headcount1–10
- GTM typeB2B
- OfferingHardware or Manufacturing
What VC Gene Therapy does
VC Gene Therapy (VCGT Inc.) is a Japanese pre-clinical biotechnology company founded in August 2020 as a wholly-owned subsidiary of Vision Care Inc. (a RIKEN Venture). Headquartered at Kobe Eye Center within the Kobe Biomedical Innovation Cluster, the company develops genome-editing-based gene therapies for inherited retinal dystrophies, with its lead pipeline targeting autosomal dominant retinitis pigmentosa caused by mutations in the RHO (rhodopsin) gene — the most prevalent dominant form of RP, representing roughly 30% of dominant cases and an estimated 100,000 patients worldwide.
VCGT's therapeutic platform combines high-specificity Zinc Finger Nuclease (ZFN) genome editing — co-developed with Hiroshima University using the FirmCut nuclease ND1 and reported to achieve CRISPR-Cas9-equivalent cleavage efficiency in adult retinal cells — with Homology-Independent Targeted Integration (HITI), a gene-insertion method that exploits the NHEJ repair pathway to function in non-dividing retinal neurons. A single therapeutic candidate is designed to insert the full-length wild-type rhodopsin gene, addressing all 110+ documented RHO mutations via a mutation-agnostic approach rather than mutation-specific therapies. High-quality AAV vector development is supported by a partnership with Synplogen Co., Ltd.
The company is pre-revenue with a stated goal of entering clinical trials within two years. Commercial revenue, when generated, is expected to follow one-time licensing / royalty models and direct commercialization of therapeutic products for ultra-rare retinal diseases in Japan and ultimately globally. The company's value chain is anchored by a tightly integrated partner ecosystem spanning Kobe Eye Center Hospital (clinical research and genetic counseling), Hiroshima University (ZFN platform), Synplogen (AAV manufacturing), and Ritsumeikan University (talent pipeline), with cumulative funding of approximately 500 million yen (~USD 3.3M) as of December 2024.
VC Gene Therapy firmographics
Firmographics- Name
- VC Gene Therapy
- Legal name
- 株式会社 VC Gene Therapy
- Website
- https://vcgt.jp
- Company type
- Private
- Founded year
- 2020
- Operating status
- Operating
- Headcount range
- 1–10 employees
- Short description
- VC Gene Therapy (VCGT Inc.) is a Japanese pre-clinical biotech developing ZFN-based genome editing and HITI gene insertion therapies for autosomal dominant retinitis pigmentosa caused by RHO gene mutations, targeting ~100,000 patients worldwide.
- Ownership category
- akta.pro rank
VC Gene Therapy industry classification
Industry- Product category
- Ophthalmic Gene Therapy
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714), Research and Development in the Physical, Engineering, and Life Sciences (54171)
- SIC
- Biological Products, (No Disgnostic Substances) (2836), Services-Commercial Physical & Biological Research (8731)
- akta.pro primary industry
- Genome Editing Gene Therapies (CRISPR/TALEN/ZFN; in vivo/ex vivo) (HLAAACAD)
- akta.pro secondary industries
- Ophthalmology Gene Therapies (HLAAACAG), AAV Vector Gene Therapy Developers (HLAAACAA)
Keywords
Where VC Gene Therapy is headquartered
LocationHeadquarters
- HQ city
- Kobe
- HQ country
- Japan
- HQ region
- Asia
Offices1 record
Markets served
VC Gene Therapy business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Personnel, Operations, Others
Revenue model
- Gene Therapy Development and Commercialization: Pre-revenue biotech company developing gene therapy treatments for ultra-rare inherited retinal diseases. Revenue will be generated through commercialization of therapeutic products following regulatory approval. The company targets the treatment of retinitis pigmentosa, specifically dominant retinitis pigmentosa caused by RHO gene mutations.
- Licensing and Partnership Revenue: Potential licensing of proprietary genome editing technologies (ZFN, HITI) and partnership arrangements for co-development of gene therapy products.
Go-to-market motion1 record
Distribution channels2 records
Marketing channels4 records
VC Gene Therapy product offering
Product offeringCore offering
VC Gene Therapy is a clinical-stage biopharmaceutical company developing gene therapy formulations for inherited retinal diseases, with its primary pipeline targeting autosomal dominant retinitis pigmentosa caused by RHO (rhodopsin) gene mutations. The therapy uses proprietary ZFN genome editing combined with HITI gene insertion to deliver a single full-length wild-type rhodopsin gene capable of addressing all 110+ known RHO mutations. The company is currently working toward clinical trials within two years.
Product overview
VCGT is a biopharmaceutical company developing gene therapies for inherited retinal diseases, primarily Retinitis Pigmentosa (RP). The company's core product pipeline centers on a genome editing-based gene therapy for autosomal dominant retinal degenerative diseases, led by its ZFN-mediated HITI (Homology-Independent Targeted Integration) gene insertion technology. The primary therapeutic candidate targets RHO (rhodopsin) gene mutations — which account for approximately 30% of dominant RP cases — using a single full-length wild-type rhodopsin gene insertion approach capable of treating all 110+ known mutation sites. Supporting technology pillars include ZFN genome editing tools (co-developed with Hiroshima University using FirmCut nuclease ND1, achieving CRISPR-Cas9-equivalent efficiency in adult retinal cells) and adenoviral vector development (partnered with Synplogen Co., Ltd.). The company was established in August 2020 as a wholly owned subsidiary of Vision Care Inc. and aims for clinical trials within two years.
Differentiator
Problem solved
Functional benefit
Products and services
- Gene Therapy for Autosomal Dominant RHO Mutation Retinitis Pigmentosa VCGT's primary therapeutic pipeline: a genome editing-based gene therapy targeting autosomal dominant inherited retinal diseases, specifically Retinitis Pigmentosa caused by RHO (rhodopsin) gene mutations. The therapy uses ZFN-mediated HITI (Homology-Independent Targeted Integration) to insert wild-type rhodopsin gene sequences, simultaneously correcting dominant-negative mutations and restoring normal RHO expression. It addresses all 110+ known RHO mutation sites with a single therapeutic intervention, intended for patients with autosomal dominant retinitis pigmentosa and designed for administration by specialty ophthalmology and retinal disease treatment centers.
Quantifiable outcome
- Achieved cleavage efficiency comparable to CRISPR-Cas9 in adult retinal cells using proprietary ZFN technology
- +1 more outcomes
Companies that use VC Gene Therapy
Customer profileSegments1 record
Ideal customer profiles2 records
VC Gene Therapy technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature4 records
VC Gene Therapy partnerships and signals
Strategic signalPartnerships
Five partnerships are on record, tiered major and core.
- Ritsumeikan University Faculty of Pharmaceutical SciencesmajorEducational and research collaboration agreement for talent exchange and human resource development in regenerative medicine and gene therapy fields. Partnership aims to cultivate excellent talent for next-generation medical technologies and advance industry-academia collaborative research.
- Synplogen Co., Ltd.coreCollaboration for development and manufacturing of gene therapy products for retinal degenerative diseases. Synplogen provides high-quality adenoviral vector (AAV) development services using proprietary ultra-long DNA synthesis technology. Located in Kobe Biomedical Innovation Cluster enabling close operational coordination.
- Hiroshima UniversitycoreJoint technology development for high-specificity ZFN genome editing. Collaboration utilizing Hiroshima University's FirmCut nuclease ND1 to achieve high-throughput ZFN development with cleavage efficiency comparable to CRISPR-Cas9 in adult retinal cells.
- Kobe City Eye Center HospitalcoreClinical research partnership for gene therapy development. Hospital conducts genetic diagnosis and genetic counseling for patients, maintains patient clinical information database. Joint research on precise gene insertion techniques and delivery methodologies.
- Kobe City Medical Hospital Organization (Kobe Citizens' Hospital Organization)majorThree-party partnership agreement with Ritsumeikan University and Vision Care Group for educational and research collaboration in regenerative medicine and gene therapy. Aims to advance research and cultivate next-generation medical talent.
Scale indicators5 records
Recent moves7 records
Expansion highlights5 records
VC Gene Therapy competitors and assessment
Company assessmentDirect peers
- MeiraGTx: Clinical-stage gene therapy company with a deep AAV-based inherited retinal disease pipeline, directly overlapping VCGT's target indication and delivery approach.
- Ocugen: Developing modifier gene therapy (OCU400) specifically for retinitis pigmentosa, the same disease VCGT targets, via AAV delivery.
- Beacon Therapeutics: AAV gene therapy company (formerly AGTC assets) dedicated to inherited retinal diseases including RP-related indications, closely comparable to VCGT.
- Editas Medicine: Genome-editing company that ran EDIT-101, an in-vivo CRISPR therapy for inherited retinal disease (LCA10), making it a direct peer in editing-based retinal gene therapy.
- ProQR Therapeutics: Focused on genetic medicines for inherited retinal diseases, targeting the same ultra-rare retinal degeneration patient population as VCGT.
- Sangamo Therapeutics: Pioneer of Zinc Finger Nuclease (ZFN) genome editing therapeutics, the same core editing modality VCGT uses; directly comparable on technology platform.
Broad incumbents
- Spark Therapeutics: Developer of Luxturna, the first approved AAV gene therapy for inherited retinal disease; an established incumbent VCGT explicitly references as a pricing comparator.
Emerging players
- Nanoscope Therapeutics: Clinical-stage company developing gene therapy for retinitis pigmentosa, a direct disease-area overlap with VCGT though using an optogenetic approach.
- 4D Molecular Therapeutics: Develops engineered AAV vector gene therapies including ophthalmology programs, overlapping VCGT's vector-delivery and retinal focus.
Regional players
- Coave Therapeutics: European AAV gene therapy developer with ophthalmology/CNS programs targeting inherited retinal disorders, comparable in modality but primarily serving a different geography.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat4 records
Key risks6 records
Key highlights6 records
Customer concentration
VC Gene Therapy financial estimates
Financial estimateRevenue estimate
Valuation estimate
VC Gene Therapy leadership team
Management profileNumber of profiles
Profiles5 records
VC Gene Therapy funding detail
Funding detailFunding overview
Funding rounds1 record
Investors1 record
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
VC Gene Therapy M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about VC Gene Therapy
What does VC Gene Therapy do?
VC Gene Therapy is a clinical-stage biopharmaceutical company developing gene therapy formulations for inherited retinal diseases, with its primary pipeline targeting autosomal dominant retinitis pigmentosa caused by RHO (rhodopsin) gene mutations. The therapy uses proprietary ZFN genome editing combined with HITI gene insertion to deliver a single full-length wild-type rhodopsin gene capable of addressing all 110+ known RHO mutations. The company is currently working toward clinical trials within two years.
Is VC Gene Therapy a public or private company?
VC Gene Therapy is a private company. It is classified as corporate owned and is currently operating.
When was VC Gene Therapy founded?
VC Gene Therapy was founded in 2020. It employs 1 to 10 people.
Where is VC Gene Therapy based?
VC Gene Therapy is headquartered in Kobe, Japan, in the Asia region.
How does VC Gene Therapy make money?
Two revenue lines are on record. Gene Therapy Development and Commercialization is the primary driver. The others are licensing and Partnership Revenue.
Who are VC Gene Therapy's main competitors?
Direct peers on record are MeiraGTx, Ocugen, Beacon Therapeutics, Editas Medicine, ProQR Therapeutics and Sangamo Therapeutics. Spark Therapeutics is listed as a broad incumbent. Emerging players are Nanoscope Therapeutics and 4D Molecular Therapeutics. Coave Therapeutics is listed as a regional player.
Does VC Gene Therapy have an API?
No public API is recorded for VC Gene Therapy.
What industry is VC Gene Therapy in?
VC Gene Therapy's product category is Ophthalmic Gene Therapy. Its primary akta.pro industry code is HLAAACAD, Genome Editing Gene Therapies (CRISPR/TALEN/ZFN; in vivo/ex vivo), with a secondary code of HLAAACAG, Ophthalmology Gene Therapies. Its NAICS code is 541714 and its SIC code is 2836.