MeiraGTx Holdings plc

MeiraGTx Holdings plc is a vertically integrated, clinical-stage genetic medicines company dedicated to developing potentially curative treatments for patients suffering from serious diseases. Headquartered in New York, NY, with significant research, development, and manufacturing operations in the United Kingdom and Ireland, the company focuses on harnessing the full potential of genetic therapies for both rare and prevalent conditions. Its mission is to improve patients' lives through cutting-edge science, utilizing its expertise in viral vector design, optimization, and gene therapy manufacturing.

The company's broad pipeline includes multiple programs in clinical development across several therapeutic areas. Key product candidates target inherited retinal diseases such as X-linked retinitis pigmentosa (XLRP) and AIPL1-associated congenital blindness (LCA4), neurodegenerative diseases like Parkinson's disease, and radiation-induced xerostomia (RIX). MeiraGTx leverages advanced technologies, including adeno-associated virus (AAV)-based gene therapies and a proprietary riboswitch gene regulation platform, which allows for precise, dose-responsive control of gene expression by oral small molecules. The company also boasts end-to-end in-house manufacturing capabilities, including GMP viral vector production facilities.

Led by President and CEO Dr. Alexandria Forbes, MeiraGTx has recently secured up to $400 million in strategic investment from Oberland Capital in July 2026 to advance its late-stage gene therapies toward commercialization. This financing supports key programs including AAV2-hAQP1 for RIX, botaretigene sparoparvovec (bota-vec) for XLRP, and AAV-AIPL1 for LCA4. The company reacquired bota-vec from Johnson & Johnson earlier in 2026 and received FDA Breakthrough Therapy Designation for AAV2-hAQP1 in March 2026. MeiraGTx also reported positive Phase 2 data for its Parkinson's disease gene therapy, AAV-GAD, in late 2024.