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MeiraGTx Holdings plc develops clinical-stage genetic medicines for diseases with high unmet need, with recurring updates centered on AAV gene therapy programs, ocular disease, radiation-induced xerostomia, and regulated gene-expression technology. Company news includes clinical disclosures for AAV-hAQP1 and AAV2-hAQP1 in grade 2/3 radiation-induced xerostomia, ophthalmology programs such as AAV-AIPL1 for Leber congenital amaurosis 4, and the acquired bota-vec program for X-linked retinitis pigmentosa.
Updates also cover strategic collaborations and license agreements, in-house manufacturing capabilities for genetic medicines, riboswitch platform development, operating results, financing arrangements, shareholder voting matters, and capital-structure disclosures tied to MGTX ordinary shares.
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MeiraGTx Holdings plc (Nasdaq: MGTX) announced two abstract presentations at the ARVO 2023 Annual Meeting. The first presentation covers the humoral immune response data from the Phase 1/2 MGT009 trial of botaretigene sparoparvovec (bota-vec), aimed at treating X-linked retinitis pigmentosa (XLRP) caused by RPGR gene mutations. This therapy is being developed in partnership with Janssen Pharmaceuticals.
The second focus is on AI-assisted engineered promoters to enhance gene expression in rod photoreceptors. Alexandria Forbes, Ph.D., highlighted the importance of these findings for advancing treatment options for XLRP, which currently has no approved therapies. The presentations will take place on April 23 and 27, 2023, at specific sessions.
MeiraGTx Holdings plc (NASDAQ:MGTX) announced that its CEO, Alexandria Forbes, Ph.D., will participate in several upcoming investor conferences. Key events include:
- Chardan's 7th Annual Genetic Medicines and Cell Therapy Manufacturing Summit - April 24, 2023, at 2:30 p.m. ET
- Panel Discussion: Gene Therapy: Positioning for CMC Success - April 25, 2023, at 12:00 p.m. ET
- BofA Securities 2023 Healthcare Conference - May 10, 2023, at 2:35 p.m. PT
- RBC Capital Markets 2023 Global Healthcare Conference - May 17, 2023, at 10:00 a.m. ET
Live webcasts will be accessible on MeiraGTx's Investors page, with replays available for 30 days post-event. MeiraGTx is focused on gene therapy for ocular and neurodegenerative diseases.
MeiraGTx Holdings plc (Nasdaq: MGTX) announced its fourth quarter and full-year financial results for 2022. They reported cash and equivalents of approximately $115 million and a decrease in license revenue from $37.7 million in 2021 to $15.9 million in 2022.
Significant clinical advancements were noted, including positive Phase 1 trial data for AAV-hAQP1 targeting radiation-induced xerostomia and encouraging top-line results from the MGT009 study in X-linked retinitis pigmentosa (XLRP). The company aims for a BLA submission for XLRP in 2024 and plans new trials for both xerostomia and Parkinson's disease.
MeiraGTx Holdings (NASDAQ: MGTX) announced positive clinical results from its Phase 1 AQUAx study of AAV2-hAQP1 for treating radiation-induced xerostomia. Key findings show significant improvements in xerostomia symptoms and saliva flow rates in both unilateral and bilateral cohorts. The therapy demonstrated safety and durability of effects, with improvements lasting up to 3 years. Plans are underway to advance to a Phase 2 study in 2023. Current treatment options for this condition are limited, highlighting a significant unmet need.