MeiraGTx Holdings PLC (NASDAQ: MGTX) is a clinical-stage gene therapy company that develops viral vector-based treatments for inherited retinal diseases, neurological disorders, and metabolic diseases, along with a riboswitch gene regulation platform. Revenue comes primarily from collaboration agreements, including upfront license fees, funded research and development services, and milestone payments tied to development and regulatory events. The company received a $100.0 million upfront fee from Johnson & Johnson Innovative Medicine in March 2019 and a $30.0 million milestone in December 2021 under a collaboration for botaretigene sparoparvovec (bota-vec) targeting X-linked retinitis pigmentosa. A separate strategic collaboration with Hologen includes a $200.0 million upfront payment and up to $230.0 million in additional committed funding for the AAV-GAD Parkinson's disease program. As of the 10-K filed March 30, 2026, the company has not yet generated product sales revenue and operates at a loss, with manufacturing facilities in London, UK and Shannon, Ireland.

Revenue model

Collaboration and licensing revenue: non-refundable upfront fees, funded research and development and manufacturing services, and development or regulatory milestone payments. To date, no product sales revenue or sales-based royalties have been recognized. Key agreements include the Johnson & Johnson Innovative Medicine Asset Purchase Agreement and the Hologen strategic collaboration (10-K, filed 2026-03-30).

Products and services

Botaretigene sparoparvovec (bota-vec / AAV-RPGR): gene therapy for X-linked retinitis pigmentosa partnered with Johnson & Johnson Innovative Medicine. AAV-GAD: gene therapy for Parkinson's disease, awarded RMAT designation, Phase 3 initiation expected, developed through joint venture Hologen Neuro AI Ltd. Riboswitch platform: gene regulation technology enabling precise dosing of proteins via oral small molecule inducers; lead program Ribo-leptin targets metabolic disease with leptin deficiency. Trigeminal neuralgia program: locally delivered gene therapy, clinic entry intended in 2026. Achromatopsia programs: two genetic forms under the Johnson & Johnson Innovative Medicine collaboration (10-K, filed 2026-03-30).

Customers and end markets

Pharmaceutical and biotech collaborators as primary revenue sources: Johnson & Johnson Innovative Medicine and Hologen. End patient markets include inherited retinal diseases (X-linked retinitis pigmentosa, achromatopsia), Parkinson's disease, trigeminal neuralgia, and metabolic diseases (leptin deficiency). No product sales to end patients as of the filing date (10-K, filed 2026-03-30).

Value-chain role

Integrated gene therapy developer and manufacturer. The company conducts internal gene therapy research, clinical development, and GMP viral vector manufacturing at its own facilities in London, UK and Shannon, Ireland. It licenses intellectual property and provides research, development, and manufacturing services to collaborators. Under the Hologen joint venture, MeiraGTx leads all clinical development and manufacturing for AAV-GAD and will supply product under exclusive manufacturing agreements (10-K, filed 2026-03-30).

Geographic exposure

Operations primarily in the United Kingdom (29,000 sq ft GMP viral vector facility at 92 Britannia Walk, London; office at 34-38 Provost Street, London; laboratory at 15 Ebenezer Street, London) and Ireland (150,000 sq ft campus including large-scale GMP viral vector manufacturing and plasmid/DNA production facilities in Shannon Free Zone, Shannon). Incorporated as a public limited company in the UK. Ordinary shares trade on the NASDAQ Global Select Market (10-K, filed 2026-03-30).

Source: SEC 10-K, filed 2026-03-30

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