MeiraGTx, LLC
MeiraGTx is a clinical-stage genetic medicine company dedicated to harnessing the full potential of genetic therapies for both rare and prevalent diseases. They focus on innovative technologies, including a riboswitch gene regulation platform that allows precise control of gene expression, and develop therapies for a wide range of disorders such as inherited retinal diseases, Parkinson's disease, ALS, xerostomia, and more. With a broad pipeline supported by end-to-end manufacturing capabilities, MeiraGTx aims to deliver transformative treatments and improve patients' lives.
Industries
Nr. of Employees
medium (51-250)
MeiraGTx, LLC
450 East 29th Street, 14th Floor, New York, NY 10016, United States
Products
AAV-AQP1 (salivary gland gene therapy candidate)
AAV-based gene therapy candidate designed to introduce a water-conducting channel into irradiated salivary glands to increase saliva production for radiation-induced xerostomia and related conditions.
AAV-GAD (CNS gene therapy candidate for Parkinson’s disease)
Locally delivered AAV gene therapy designed to increase GABA production in the subthalamic nucleus to modulate motor circuits and improve symptoms of Parkinson’s disease.
AAV-AIPL1 (gene therapy for AIPL1-associated LCA4)
AAV-based ocular gene therapy developed to deliver the intact AIPL1 gene to cone and rod photoreceptors via subretinal injection to restore function and slow degeneration in Leber congenital amaurosis type 4.
AAV-RPGR (candidate for X‑linked retinitis pigmentosa)
AAV-based gene therapy candidate targeting RPGR for X-linked retinitis pigmentosa, with late-stage clinical data and regulatory designations reported.
AAV-AQP1 (salivary gland gene therapy candidate)
AAV-based gene therapy candidate designed to introduce a water-conducting channel into irradiated salivary glands to increase saliva production for radiation-induced xerostomia and related conditions.
AAV-GAD (CNS gene therapy candidate for Parkinson’s disease)
Locally delivered AAV gene therapy designed to increase GABA production in the subthalamic nucleus to modulate motor circuits and improve symptoms of Parkinson’s disease.
AAV-AIPL1 (gene therapy for AIPL1-associated LCA4)
AAV-based ocular gene therapy developed to deliver the intact AIPL1 gene to cone and rod photoreceptors via subretinal injection to restore function and slow degeneration in Leber congenital amaurosis type 4.
AAV-RPGR (candidate for X‑linked retinitis pigmentosa)
AAV-based gene therapy candidate targeting RPGR for X-linked retinitis pigmentosa, with late-stage clinical data and regulatory designations reported.
Services
On-site GMP production, quality control, and release testing for AAV-based gene therapies using licensed manufacturing capacity and platform processes designed for clinical and commercial supply.
Collaborative R&D and licensing to design and optimize capsids, promoters, and regulatory elements to improve potency, tissue targeting, and safety of gene therapy vectors.
Licensing and joint development of an oral small-molecule-inducible gene regulation platform for controlled in vivo transgene expression applicable to gene and cell therapies.
Policy-based review and processing of physician-initiated requests for investigational product use outside clinical trials, consistent with international regulatory criteria and available manufacturing capacity.
Operational support for global patient recruitment and multi-site conduct of clinical research studies in inherited retinal disease, X-linked retinitis pigmentosa, xerostomia, and Parkinson’s disease.
On-site GMP production, quality control, and release testing for AAV-based gene therapies using licensed manufacturing capacity and platform processes designed for clinical and commercial supply.
Collaborative R&D and licensing to design and optimize capsids, promoters, and regulatory elements to improve potency, tissue targeting, and safety of gene therapy vectors.
Licensing and joint development of an oral small-molecule-inducible gene regulation platform for controlled in vivo transgene expression applicable to gene and cell therapies.
Policy-based review and processing of physician-initiated requests for investigational product use outside clinical trials, consistent with international regulatory criteria and available manufacturing capacity.
Operational support for global patient recruitment and multi-site conduct of clinical research studies in inherited retinal disease, X-linked retinitis pigmentosa, xerostomia, and Parkinson’s disease.
Expertise Areas
- Gene therapy development
- AAV vector engineering and promoter design
- Inducible gene expression systems (riboswitches and aptamers)
- Clinical trial management for advanced therapies (randomized, sham‑controlled designs)
Key Technologies
- Adeno-associated virus (AAV) vector engineering
- Riboswitch-based inducible gene regulation
- Synthetic promoter design and discovery
- GMP viral vector production platforms
News & Updates
MeiraGTx CEO Zandy Forbes Fireside Chat at the Bank of America Securities 2025 Healthcare Conference
MeiraGTx CEO Zandy Forbes will speak at 8:55am PT / 11:55am ET today, May 14, at the Bank of America Securities 2025 Healthcare Conference in Las Vegas. The webcast is available for free registration.
The company announced it will exhibit four poster presentations at the American Society of Gene and Cell Therapy (ASGCT) 2025 Annual Meeting, highlighting its technology platforms for gene and cell therapy.
MeiraGTx reported its financial and operational results for Q1 2025, including progress on its pipeline and collaborations.
The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to AAV-GAD for Parkinson’s disease, based on clinical data demonstrating potential benefit.
Forbes reports that MeiraGTx’s investigational gene therapy has given eyesight to 11 children who were born blind.
Newsweek highlights the success of MeiraGTx’s gene therapy in restoring vision in children with AIPL1-related retinal dystrophy.
MeiraGTx CEO Zandy Forbes Fireside Chat at the Bank of America Securities 2025 Healthcare Conference
MeiraGTx CEO Zandy Forbes will speak at 8:55am PT / 11:55am ET today, May 14, at the Bank of America Securities 2025 Healthcare Conference in Las Vegas. The webcast is available for free registration.
The company announced it will exhibit four poster presentations at the American Society of Gene and Cell Therapy (ASGCT) 2025 Annual Meeting, highlighting its technology platforms for gene and cell therapy.
MeiraGTx reported its financial and operational results for Q1 2025, including progress on its pipeline and collaborations.
The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to AAV-GAD for Parkinson’s disease, based on clinical data demonstrating potential benefit.
Forbes reports that MeiraGTx’s investigational gene therapy has given eyesight to 11 children who were born blind.
Newsweek highlights the success of MeiraGTx’s gene therapy in restoring vision in children with AIPL1-related retinal dystrophy.