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MeiraGTx Focuses on Genetic Medicines

MeiraGTx recently released its 10-K report. MeiraGTx Holdings plc is a clinical-stage genetic medicines company focused on ocular, salivary gland, and neurological diseases, with programs in inherited retinal disorders, radiation-induced xerostomia, Parkinson’s disease, and other conditions. The company also develops manufacturing capabilities and a riboswitch-based gene regulation platform, while maintaining collaborations with Johnson & Johnson Innovative Medicine and Hologen Limited.

In Item 7, MeiraGTx said it remains a vertically integrated clinical-stage company with a pipeline that includes AAV-hAQP1 for radiation-induced xerostomia and Sjogren’s syndrome, AAV-GAD for Parkinson’s disease, AAV-AIPL1 for LCA4, and AAV-RPE65, AAV-CNGB3, and AAV-CNGA3 in Phase 2 studies. It also said it has preclinical programs including AAV-RDH12, AAV-BBS10, AAV-RetGC, and AAV-UPF1, plus programs in Alzheimer’s disease and wet and dry neovascular age-related macular degeneration, and it is pursuing cell therapies for oncology, autoimmune disease, and long-term intractable pain.

The company said its internal manufacturing base includes two GMP viral vector production facilities, internal GMP plasmid production, and an in-house quality control hub for stability and release. It also described a proprietary manufacturing platform and a synthetic riboswitch technology designed to control transgene expression with oral small molecules.

MeiraGTx said it had cash, cash equivalents, and restricted cash of $68.2 million at December 31, 2025, plus $3.0 million expected from Johnson & Johnson Innovative Medicine in the first quarter of 2026. It also said it had received $55.0 million in the first quarter of 2026 from Hologen, with another $95.0 million expected from the strategic collaboration, and estimated those funds would support operations into the second half of 2027.

The company reported net losses of $114.2 million in 2025 and $147.8 million in 2024, with an accumulated deficit of $816.2 million at year-end 2025. Total operating expenses were $187.4 million in 2025, down from $197.5 million in 2024.

MeiraGTx said it raised $15.1 million through at-the-market sales in 2025 and $8.4 million in 2024, and it also completed a $50.0 million public offering in August 2024 and a $1.0 million private placement later that month. The company said it may need additional capital through equity, debt, collaborations, or licensing arrangements, and that it had no product revenue to date. As a result of these announcements, the company's shares have moved -1.18% on the market, and are now trading at a price of $7.53. For more information, read the company's full 10-K submission here.

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