MeiraGTx

What MeiraGTx does

MeiraGTx Holdings plc is a clinical-stage genetic medicine company developing gene therapies for inherited and acquired disorders, with operations headquartered in New York, NY, incorporated as a public limited company in England and Wales, and listed on NASDAQ under ticker MGTX. Founded in 2015, the company has built a vertically integrated platform spanning proprietary vector design, riboswitch-based gene regulation technology that enables oral small-molecule control of transgene expression, AI/ML-guided promoter and capsid optimization via the proprietary CLARA Convolutional Neural Network, and end-to-end GMP manufacturing through two licensed facilities in London, UK and Shannon, Ireland with more than 180 manufacturing staff.

The pipeline centers on four late-stage clinical programs: botaretigene sparoparvovec (bota-vec) for X-linked retinitis pigmentosa (XLRP), which completed Phase 3 and was reacquired from Johnson & Johnson in April 2026 with planned US, EU, and Japan filings targeting 2027; AAV-hAQP1 for radiation-induced xerostomia, holding FDA Breakthrough Therapy and RMAT designations with 3-year sustained Phase 1 data; AAV-GAD for Parkinson's disease, the only CNS gene therapy with two positive randomized, double-blind, sham-surgery-controlled trials and FDA RMAT designation; and AAV-AIPL1 for Leber congenital amaurosis type 4 (LCA4), which restored vision in 11 of 11 children treated and is being filed under exceptional circumstances with the UK MHRA. Earlier-stage programs extend into ALS/FTD, Stargardt's disease, wet AMD, geographic atrophy (via ZipBio license), metabolic disease including riboswitch-regulated GLP-1 combinations, and Ribo-CAR-T cell therapy.

The business model today is licensing and collaboration-driven: revenue is generated through upfront payments, milestone payments, and royalties from pharmaceutical partners, with Eli Lilly ($75M upfront, $400M+ potential milestones) and Hologen Limited ($200M upfront, up to $230M committed via the Hologen Neuro AI Ltd joint venture) representing the two largest recent transactions. The go-to-market is transitioning toward direct specialty distribution through hospital-administered and specialty pharmacy channels for ultra-rare disease launches targeted for 2027 (bota-vec) and early 2028 (AAV-hAQP1). Capital is sourced from a mix of public equity offerings, strategic investments from Sanofi, and asset-backed financing from Perceptive Advisors collateralized by the UK and Ireland manufacturing facilities.

Name

MeiraGTx

Legal name

MeiraGTx Holdings plc

Website

https://meiragtx.com

Company type

Public

Founded year

2015

Operating status

Operating

Headcount range

251–500 employees

Short description

MeiraGTx is a clinical-stage genetic medicine company developing AAV gene therapies for inherited retinal diseases, xerostomia, Parkinson's, and rare disorders, using proprietary riboswitch regulation and AI-driven vector optimization, with planned first commercial launches in 2027-2028.

Ownership category

akta.pro rank

Product category

Gene Therapy Pharmaceuticals

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414), Electromedical and Electrotherapeutic Apparatus Manufacturing (334510), Medical and Diagnostic Laboratories (6215), Medical Laboratories (621511)

SIC

Electromedical & Electrotherapeutic Apparatus (3845), In Vitro & In Vivo Diagnostic Substances (2835), Wholesale-Drugs, Proprietaries & Druggists' Sundries (5122)

akta.pro primary industry

Ophthalmology Gene Therapies (HLAAACAG)

akta.pro secondary industries

Neurology/CNS Gene Therapies (HLAAACAH), Gene Therapy CMC/Process Development Platforms (capsid engineering, producer cell lines, plasmids, upstream/downstream) (HLAAACAN), Genome Editing Gene Therapies (CRISPR/TALEN/ZFN; in vivo/ex vivo) (HLAAACAD), Cardiometabolic & Other Systemic Gene Therapies (HLAAACAI), Gene Therapy for Rare/Monogenic Diseases (HLAAACAE)

Keywords

• Gene therapy development
• Adeno-associated virus vectors
• Rare disease therapeutics
• Riboswitch gene regulation
• GMP viral manufacturing

Headquarters

HQ city

New York

HQ country

United States

HQ region

North America

Offices3 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Licensing and Collaboration Revenue: MeiraGTx generates license revenue through strategic collaborations with pharmaceutical companies. Recent deals include $75M upfront payment from Eli Lilly for AAV-AIPL1 ophthalmology program with potential milestones exceeding $400M, and $30M investment from Sanofi for rights of first negotiation on certain technologies.
2. Non-dilutive Funding: Company has secured non-dilutive funding through strategic partnerships and financing arrangements, including $200M upfront from Hologen AI joint venture, $130M upfront from Janssen for XLRP program, and various milestone payments tied to clinical and regulatory achievements.
3. Equity Financing: Company has raised equity capital through public offerings and private placements. Recent $100 million share offering completed in 2026, $50 million offering in August 2024 led by Sanofi, and $18.2 million from Perceptive Advisors share purchase agreement in January 2026.
4. Future Product Revenue: Pre-commercial stage; plans to generate product revenue from commercializing gene therapies including bota-vec for XLRP (targeting 2027 launch) and AAV-hAQP1 for xerostomia (targeting early 2028 US launch).

Pricing tiers

Go-to-market motion2 records

Distribution channels2 records

Marketing channels5 records

Core offering

MeiraGTx is a clinical-stage genetic medicine company that develops adeno-associated virus (AAV)-based gene therapies to treat inherited retinal diseases, radiation-induced xerostomia, Parkinson's disease, ALS/FTD, genetic obesity, and other rare and prevalent genetic disorders. Its offerings are built on three proprietary technology platforms: the riboswitch gene regulation system, AI/ML-driven vector and promoter optimization (CLARA), and a vertically integrated end-to-end GMP manufacturing ecosystem. The company's lead pipeline includes AAV-hAQP1 for xerostomia, AAV-GAD for Parkinson's, botaretigene sparoparvovec (bota-vec) for X-linked retinitis pigmentosa, and AAV-AIPL1 for Leber congenital amaurosis 4.

Product overview

MeiraGTx is a vertically integrated, clinical-stage genetic medicine company with a broad pipeline of gene therapy candidates and proprietary technology platforms. The company's core portfolio includes four late-stage clinical programs: AAV-GAD2 for Parkinson's disease (Phase 3 ready with RMAT designation), AAV-hAQP1 for radiation-induced xerostomia (Phase 2 with Breakthrough Therapy and RMAT designations), AAV-AIPL1 for LCA4 congenital blindness, and botaretigene sparoparvovec (bota-vec) for XLRP (reacquired from J&J in 2026). The technology infrastructure comprises three proprietary platforms: the Riboswitch Gene Regulation Platform enabling oral small molecule control of gene expression, the Next-Generation Vector Optimization Platform with AI/ML-driven promoter and capsid engineering, and the End-to-End GMP Manufacturing Ecosystem with facilities in London and Shannon, Ireland. Earlier-stage programs include AAV-UPF1 for ALS, AAV-RDH12 for retinal dystrophy, AAV-ABCA4 for Stargardt's disease, Ribo-CAR-T for oncology, and metabolic programs including RiboLeptin and GLP-1 combinations. The company maintains strategic collaborations with Eli Lilly (ophthalmology), Hologen AI (Parkinson's disease and CNS), and Sanofi (riboswitch technology rights).

Differentiator

Problem solved

Functional benefit

Products and services

• AAV-hAQP1 (Aquaporin-1 Gene Therapy) Gene therapy candidate that introduces a water-conducting channel (aquaporin-1) into damaged salivary glands via AAV2 vector for the treatment of Grade 2/3 late radiation-induced xerostomia in head and neck cancer survivors. Received FDA Breakthrough Therapy Designation (March 2026) and RMAT Designation. Targets the approximately 435,000 patients globally with persistent Grade 2/3 RIX.
• AAV-GAD2 (Glutamic Acid Decarboxylase Gene Therapy) Gene therapy candidate designed to reprogram dysfunctional brain circuits through local production of GABA in the subthalamic nucleus via AAV vector for the treatment of Parkinson's disease not adequately controlled with anti-Parkinsonian medications. Phase 3-ready with FDA RMAT Designation. The only CNS gene therapy program with two randomized, double-blind, sham-surgery-controlled trials meeting prespecified primary endpoints. 58 patients treated across three clinical studies.
• Botaretigene Sparoparvovec (bota-vec) Gene therapy using AAV5 vector to deliver the RPGR gene for the treatment of X-linked retinitis pigmentosa (XLRP) caused by RPGR gene mutations. Phase 3 LUMEOS trial demonstrated statistically significant improvements in retinal sensitivity and visual function. Received PRIME (EMA), Fast Track, and Orphan Drug designations. MeiraGTx reacquired global rights from Janssen (J&J) in April 2026 for $25 million upfront plus milestone payments and royalties beginning in 2029.
• AAV-AIPL1 Gene therapy using AAV8 vector to deliver the AIPL1 gene for the treatment of AIPL1-associated Leber congenital amaurosis 4 (LCA4) congenital blindness in children. Data published in The Lancet in February 2025 demonstrated meaningful responses in all 11 children treated. Received FDA Rare Pediatric Disease Designation, FDA Orphan Drug Designation, and European Commission Orphan Designation. Made available under MHRA Specials License in the UK for physician-led studies. Licensed exclusively to Eli Lilly for $75M upfront plus $400M+ milestones (announced November 2025).
• AAV-RDH12 Gene therapy candidate for the treatment of RDH12-associated retinal dystrophy, an inherited retinal disease causing progressive vision loss. IND-ready with FDA Rare Pediatric Disease Designation and FDA Orphan Drug Designation.
• AAV-ABCA4 Gene therapy candidate for Stargardt's disease, an inherited retinal disease causing progressive vision loss due to ABCA4 gene mutations. Part of MeiraGTx's ophthalmology pipeline.
• AAV-VEGFR2

Quantifiable outcome

• 11 out of 11 LCA4 children treated with AAV-AIPL1 who were blind at birth now have visual acuity in treated eyes
• +4 more outcomes

Named customers4 records

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 capability9 records

Feature4 records

Partnerships

Five partnerships are on record, tiered core and minor.

• Johnson & Johnson (Janssen)coreStrategic or Co-development Partner · 16 April 2026MeiraGTx reacquired global rights to botaretigene sparoparvovec (bota-vec) for X-linked retinitis pigmentosa from Johnson & Johnson. $25 million upfront payment plus milestone payments tied to regulatory approval and sales royalties beginning in 2029. Originally J&J had acquired rights in 2023 for up to $415 million including $130M upfront. MeiraGTx plans to file for US, EU, and Japan approvals targeting 2027 launch.
• ZipBiominorStrategic or Co-development Partner · 3 February 2026Exclusive license agreement for AAV gene therapies for geographic atrophy. MeiraGTx received exclusive global rights to ZipBio's first-in-class therapies targeting the complement pathway for geographic atrophy. Deal combines ZipBio's AI-driven COMPACT protein therapeutics platform with MeiraGTx's vector design, clinical development, and in-house manufacturing capabilities. Financial terms not disclosed.
• Eli Lilly and CompanycoreStrategic or Co-development Partner · 10 November 2025Strategic collaboration to develop and commercialize genetic medicines for ophthalmology. MeiraGTx granted exclusive license to AAV-AIPL1 program for treating Leber congenital amaurosis 4 (LCA4). Lilly made $75 million upfront payment with potential milestones exceeding $400 million plus royalties. Lilly also gained access to MeiraGTx's ophthalmology gene therapy technologies and riboswitch technology.
• Hologen LimitedcoreStrategic or Co-development Partner · 13 March 2025Strategic collaboration to expedite Phase 3 development of AAV-GAD for Parkinson's disease and industrialize MeiraGTx's manufacturing process. MeiraGTx received $200 million upfront payment. Joint venture Hologen Neuro AI Ltd formed with up to $230 million committed from Hologen to fully finance AAV-GAD development through commercialization. Hologen owns minority stake in MeiraGTx's manufacturing subsidiary.
• Moorfields Eye Hospital and UCL Institute of OphthalmologycoreStrategic or Co-development Partner · 20 February 2025Founding collaboration for MeiraGTx's ocular division. Dr. Michel Michaelides serves as both Head of Clinical Ophthalmology at MeiraGTx and Consultant Ophthalmologist at Moorfields Eye Hospital. Clinical studies for inherited retinal diseases conducted in partnership with these institutions.

Scale indicators7 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• REGENXBIO: Clinical-stage gene therapy company developing AAV-based therapeutics with strong ophthalmology franchise (RGX-314 for wet AMD), directly comparable to MeiraGTx's ophthalmology pipeline and AAV manufacturing capabilities.
• Spark Therapeutics: Pioneer in AAV gene therapy for inherited retinal diseases (Luxturna for RPE65), now part of Roche. Direct overlap with MeiraGTx's inherited retinal disease pipeline including LCA and XLRP programs.
• Voyager Therapeutics: Clinical-stage gene therapy company developing AAV-based treatments for CNS diseases including Parkinson's and ALS, directly comparable to MeiraGTx's AAV-GAD Parkinson's program and CNS pipeline.
• Adverum Biotechnologies: Clinical-stage gene therapy company focused on ocular diseases including wet AMD and diabetic retinopathy, directly competing with MeiraGTx in intravitreal AAV delivery for ophthalmology indications.
• uniQure: Clinical-stage gene therapy company developing AAV-based treatments for hemophilia, Huntington's disease, and other CNS/rare diseases, with comparable AAV manufacturing infrastructure and platform technology approach.
• Krystal Biotech: Commercial-stage gene therapy company with approved topical gene therapy Vyjuvek for dystrophic epidermolysis bullosa, sharing MeiraGTx's rare disease gene therapy focus and platform-based approach.

Broad incumbents

• BioMarin Pharmaceutical: Established rare disease biotech with approved AAV gene therapy Roctavian for hemophilia A and broader gene therapy pipeline, representing a larger incumbent with overlapping AAV manufacturing and regulatory capabilities.
• Sarepta Therapeutics: Commercial-stage gene therapy company with multiple approved AAV-based therapies for Duchenne muscular dystrophy, representing a larger incumbent with established specialty distribution and gene therapy commercial capabilities.

Emerging players

• Coave Therapeutics: Clinical-stage gene therapy company developing AAV-based treatments for CNS and ocular diseases, with comparable focus on rare neurological and inherited retinal disorders and next-generation capsid engineering.

Others

• Oxford BioMedica: UK-based cell and gene therapy CDMO with lentiviral vector manufacturing capabilities; the CSO Stuart Naylor was previously CSO at Oxford BioMedica, making it a relevant adjacency in the AAV/lentiviral manufacturing ecosystem where MeiraGTx competes.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/MeiraGTx
• LinkedInlinkedin.com/company/meiragtx
• Facebookfacebook.com/meiragtx

Compliance16 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles11 records

Subsidiaries1 record

Funding overview

Funding rounds9 records

Investors7 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A2 records

Investments1 record

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about MeiraGTx