MeiraGTx Netherlands B.V.
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
small (1-50)
MeiraGTx Netherlands B.V.
Amsterdam, Noord-Holland, The Netherlands, Europe
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
End-to-end GMP viral vector manufacturing and QC
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.
Vector engineering and promoter optimization collaborations
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.
Riboswitch platform licensing and development partnerships
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.
Expanded access request evaluation and management
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.
Global clinical study enrollment and site management
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.
End-to-end GMP viral vector manufacturing and QC
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.
Vector engineering and promoter optimization collaborations
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.
Riboswitch platform licensing and development partnerships
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.
Expanded access request evaluation and management
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.
Global clinical study enrollment and site management
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