Press release
MeiraGTx Reports Fourth Quarter and Full Year 2021 Financial and Operational Results
- Phase 3 Lumeos Clinical Trial of botaretigene sparoparvovec (AAV-RPGR) for the Treatment of X-linked Retinitis Pigmentosa (XLRP) Enrolling and Dosing

About this update from Meiragtx Holdings Plc
[{"type":"text","content":"- Phase 3 Lumeos Clinical Trial of botaretigene sparoparvovec (AAV-RPGR) for the Treatment of X-linked Retinitis Pigmentosa (XLRP) Enrolling and Dosing Patients- Received $30 Million Cash Milestone Payment from Janssen Pharmaceuticals, Inc.- Positive Preliminary Clinical Data from AQUAx AAV-hAQP1 Phase 1 Trial for Treatment of Grade 2/3 Radiation-Induced Xerostomia Announced- First In Vivo Data from Riboswitch Gene Regulation Platform at R&D Day Presented LONDON and NEW YORK, March 10, 2022 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced financial and operational results for the fourth quarter and full-year ended December 31, 2021, and provided a corporate update. “MeiraGTx had an exceptional year in 2021, marked by advancements in all aspects of our pipeline and technology platforms,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “We initiated our first pivotal study with our partner Janssen for the treatment of XLRP using drug product manufactured in-house at our cGMP facility using our proprietary manufacturing process, we announced positive preliminary clinical data from our wholly-owned grade 2/3 xerostomia program and we presented the first in vivo data from our transformative gene regulation platform.” “This progress is enabled by the broad end-to-end capabilities we have focused on building at the Company since inception,” continued Dr. Forbes. “We have established leading capabilities in vector engineering and optimization, viral vector production process development, manufacturing and quality, all while developing our synthetic biology platform which for the first time allows precise and specific control of genetic medicines using novel orally dosed small molecules.” Recent Development Highlights and Anticipated 2022 Milestones Botaretigene Sparoparvovec (AAV-RPGR) for the Treatment of XLRP: MeiraGTx, in collaboration with Janssen Pharmaceuticals, Inc. (Janssen), one of the Janssen Pharmaceutical Companies of Johnson & Johnson, is dosing and enrolling patients in the Phase 3 Lumeos clinical trial of botaretigene sparoparvovec.MeiraGTx received a $30 million payment from Janssen for a clinical milestone in the Phase 3 Lumeos trial of botaretigene sparoparvovec. AAV-hAQP1 for the Treatment of Grade ...