NEW YORK , Nov. 20, 2025 /PRNewswire/ -- Graviton Bioscience Corporation , a clinical-stage biotechnology company developing selective ROCK2 inhibitors for metabolic, inflammatory, fibrotic, and central nervous system disorders, among other therapeutic indications, today announced that Samuel Waksal , Ph.D., Chief Executive Officer, will participate in a fireside chat at the 8th Annual Evercore HealthCONx Conference. Details... Read More

Entered into broad strategic collaboration with Eli Lilly and Company (“Lilly”) in the area of ophthalmology, granting Lilly worldwide exclusive rights to the Company’s AAV-AIPL1 program for treatment of one of the most severe inherited retinopathies, Leber congenital amaurosis 4 (LCA4) Released material under the Company’s Specials license for a second rare pediatric ophthalmology condition caused by mutations in BBS10,... Read More

LONDON and NEW YORK, Nov. 10, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced a broad strategic collaboration in the area of ophthalmology with Eli Lilly and Company (“Lilly”). MeiraGTx will grant Lilly worldwide exclusive rights to its AAV-AIPL1 program for treatment of one of the most severe inherited retinopathies, Leber... Read More

Multiple Posters Highlight the Breadth of Company’s Novel Genetic Medicine and Cell Therapy Platforms LONDON and NEW YORK, Oct. 07, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical-stage genetic medicines company, today announced the Company will exhibit two posters at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress, which is being held from October... Read More

Gained alignment with U.S. Food and Drug Administration (FDA) on the ongoing Phase 2 AQUAx2 randomized double-blind, placebo-controlled pivotal study in Grade 2/3 radiation-induced xerostomia (RIX) to support a potential Biologics License Application (BLA) filing; on track for potential data readout late 2026 FDA Granted Regenerative Medicine Advanced Therapy (RMAT) designation for AAV-GAD for the treatment of Parkinson’s... Read More

Multiple Poster Presentations Highlight the Depth and Novelty of MeiraGTx’s Technology Platforms for Gene and Cell Therapy LONDON and NEW YORK, May 13, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced the Company will exhibit four poster presentations at the American Society of Gene and Cell Therapy (ASGCT) 2025 Annual Meeting,... Read More

Announced strategic collaboration with Hologen AI, including a $200 million cash upfront payment to MeiraGTx and the formation of a joint venture, Hologen Neuro AI Ltd, with a further $230 million in capital committed to initially focus on expediting Phase 3 clinical development of AAV-GAD for Parkinson’s disease U.S. Food and Drug Administration (FDA) Granted Regenerative Medicine Advanced Therapy (RMAT) designation for... Read More

This RMAT designation is based on data from 3 clinical studies demonstrating the potential benefit of AAV-GAD as a one-time treatment for Parkinson’s disease RMAT designation includes the benefits of the Fast Track and Breakthrough Therapy designations, allows frequent regulatory interactions with the FDA, and potential routes to accelerated approval and Priority Review LONDON and NEW YORK, May 09, 2025 (GLOBE NEWSWIRE) --... Read More

Today announced strategic collaboration with Hologen AI, including a $200 million upfront payment to MeiraGTx and the formation of a joint venture, Hologen Neuro AI Ltd, with a further $230 million in capital committed to initially focus on expediting Phase 3 clinical development of AAV-GAD for Parkinson’s disease MeiraGTx and Hologen have created the first neuro-AI clinical drug development company to transform the... Read More

MeiraGTx to receive $200 million in upfront cash consideration MeiraGTx and Hologen will form a joint venture, Hologen Neuro AI Ltd, with an additional $230 million committed capital from Hologen to fund 100% of the development of AAV-GAD for Parkinson’s disease through to commercialization, as well as other potential pipeline products MeiraGTx will enter into clinical and commercial supply agreements with Hologen Neuro AI... Read More

As disclosed in the paper, 4 out of 4 young children with the AIPL1-related retinal dystrophy, LCA4, benefited substantially from unilateral subretinal administration of rAAV8.hRKp.AIPL1 with improved visual acuity, functional vision, and protection against progressive retinal degeneration Following the strong safety and substantial efficacy demonstrated in this first cohort of 4 children treated unilaterally, a further 7... Read More

LONDON and NEW YORK, Jan. 22, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings Plc (Nasdaq: MGTX), a vertically integrated, clinical-stage genetic medicines company, today announced the U.S. Food and Drug Administration (FDA) has granted the Company Rare Pediatric Disease Designation to its AAV8-RK-RetGC program for the treatment of patients with Leber congenital amaurosis due to GUCY2D mutations (LCA1). This is the fourth Rare... Read More

RMAT designation recognizes the preliminary clinical evidence of the potential benefit of AAV2-hAQP1 as a one-time treatment for this debilitating condition RMAT designation includes the benefits of the Fast Track and Breakthrough Therapy designations, allows frequent regulatory interactions with the FDA, and potential routes to accelerated approval and Priority Review LONDON and NEW YORK, Dec. 09, 2024 (GLOBE NEWSWIRE) --... Read More

ROCKVILLE, Md. and SUZHOU, China , Nov. 24, 2024 /PRNewswire/ -- Ascentage Pharma (6855.HK), a global biopharmaceutical company engaged in discovering, developing and commercializing therapies to address global unmet medical needs primarily for malignancies, today announced that it has appointed Ms. Marina S. Bozilenko and Dr. Debra Yu as additional independent non-executive directors of the company with effect from November... Read More

Received 3 Rare Pediatric Disease Designations (RPDD) from FDA for each of 3 potential therapies for 3 different rare inherited retinopathies including AAV-AIPL1 Agreed on pathway with MHRA for Marketing Authorization Application (MAA) under exceptional circumstances for AAV-AIPL1 for the treatment of Leber congenital amaurosis (LCA4) retinal dystrophy without further clinical studies Announced positive data from randomized,... Read More

LONDON and NEW YORK, Oct. 22, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical-stage genetic medicines company, today announced the Company will exhibit five posters at the European Society of Gene and Cell Therapy (ESGCT) 2024 Annual Congress, which is being held from October 22-25, 2024, in Rome, Italy. The posters are available on the Posters and Publications page of the... Read More

The primary study objective of safety and tolerability was met Significant and clinically meaningful improvements from baseline demonstrated for key efficacy endpoints at 26 weeks Significant improvement of 18 points in Unified Parkinson’s Disease Rating Scale (UPDRS) Part 3 in the high dose group at 26 weeks Significant improvement in the Parkinson’s Disease Questionnaire (PDQ-39) score, a key quality of life measure, for... Read More

LONDON and NEW YORK, Oct. 09, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicine company, today announced a poster presentation at the 2024 Society for Neuroscience Conference (SfN), which is being held from October 5-9, 2024, in Chicago, IL. “We are pleased to share data at this year’s Society for Neuroscience Conference on the remarkable efficacy of our... Read More

Positive data from the Phase 1 AQUAx study in radiation-induced xerostomia (RIX) presented at the American Academy of Oral Medicine 2024 annual meeting (AAOM) showed meaningful improvements in patient-reported outcomes and saliva production with AAV2-hAQP1 treatment Company awarded Innovation Passport Designation by the U.K. Innovative Licensing and Access Pathway Steering Group for AAV8-RK-AIPL1 for the treatment of... Read More

LONDON and NEW YORK, Aug. 12, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicine company, today announced the pricing of an underwritten offering of 12,500,000 of its ordinary shares at an offering price of $4.00 per share. Gross proceeds to MeiraGTx from the offering are expected to be $50 million, before deducting underwriting discounts and commissions... Read More

Positive data from the Phase 1 AQUAx study in radiation-induced xerostomia (RIX) presented in an oral session at the American Academy of Oral Medicine 2024 annual meeting (AAOM) April 17-20, 2024 Received $50 million milestone following initiation of the extension study for the Phase 3 LUMEOS clinical trial for botaretigene sparoparvovec (bota-vec, formerly AAV-RPGR) for the treatment of X-linked retinitis pigmentosa (XLRP)... Read More

LONDON and NEW YORK, April 24, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (NASDAQ: MGTX), a vertically integrated, clinical stage gene therapy company, today announced that Alexandria Forbes, Ph.D., President and Chief Executive Officer, will participate in the following investor conferences: Chardan 8th Annual Genetic Medicines and Cell Therapy Manufacturing Summit, Virtual Corporate presentation: Monday, April 29,... Read More

LONDON and NEW YORK, April 18, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced the Company gave an oral presentation at the American Academy of Oral Medicine Annual Conference, being held from April 16-20, 2024, at the Hyatt Regency Grand Cypress in Orlando, FL. The details of the oral presentation are below: Session: Oral Abstract... Read More

Company aligned with FDA on requirements for ongoing Phase 2 AQUAx2 clinical trial for Grade 2/3 radiation-induced xerostomia to be considered pivotal supporting potential BLA filing Company to present data from the Phase 1 AQUAx study in an oral presentation at the American Academy of Oral Medicine 2024 annual meeting (AAOM) April 17-20, 2024 Completed dosing the three-arm randomized, sham-controlled Phase 1 clinical... Read More

LONDON and NEW YORK, Feb. 13, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced the achievement of the first milestone under the asset purchase agreement the Company entered into with Janssen Pharmaceuticals, Inc. (J&J), a Johnson & Johnson company, in December 2023 relating to bota-vec for the treatment of XLRP. “We are very happy to... Read More

MeiraGTx enters into an Asset Purchase Agreement related to botaretigene sparoparvovec (bota-vec, formerly AAV-RPGR) for the treatment of X-linked retinitis pigmentosa (XLRP) for a total of up to $415 million MeiraGTx to receive $130 million in upfront and near-term milestone payments as part of the agreement MeiraGTx to receive up to an additional $285 million upon first commercial sales of bota-vec in U.S. and EU and... Read More

LONDON and NEW YORK, Nov. 21, 2023 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (NASDAQ:MGTX), a vertically integrated, clinical stage gene therapy company, today announced that Alexandria Forbes, Ph.D., President and Chief Executive Officer, will participate in the following investor conferences: 6th Annual Evercore ISI HealthCONx Conference Corporate presentation: Tuesday, November 28, 2023, at 10:25 a.m. ET Piper Sandler... Read More

Received strategic investment from Sanofi purchasing $30 million of ordinary shares of the Company at a price of $7.50 per share in October 2023 Announced a right of first negotiation (ROFN) with Sanofi for the use of MeiraGTx’s Riboswitch gene regulation technology for certain Central Nervous System (CNS) and Immunology and Inflammation (I&I) targets, including IL-4 and IL-13, as well as for GLP-1 and other gut peptides for... Read More

Sanofi to purchase $30 million of ordinary shares of the Company at a price of $7.50 per share Sanofi to receive a right of first negotiation (ROFN) for the use of MeiraGTx’s Riboswitch gene regulation technology for certain Immunology and Inflammation (I&I) and Central Nervous System (CNS) targets, as well as for GLP-1 and other gut peptides for metabolic disease, and for MeiraGTx’s Phase 2 Xerostomia program In addition,... Read More

LONDON and NEW YORK, Oct. 24, 2023 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced the Company will exhibit eight posters and deliver one oral presentation at the European Society of Gene and Cell Therapy (ESGCT) 2023 Annual Congress, which is being held from October 24-27, 2023, in Brussels, Belgium. “The number of acceptances for... Read More

Phase 3 LUMEOS clinical trial of botaretigene sparoparvovec (bota-vec, formerly AAV-RPGR) for the treatment of X-linked retinitis pigmentosa (XLRP) enrollment target has now been surpassed Announced positive clinical data from the completed AQUAx Phase 1 study of AAV2-hAQP1 for the treatment of grade 2/3 radiation-induced xerostomia (RIX), showing bilaterally treated participants reaching normal levels of whole saliva flow... Read More

LONDON and NEW YORK, Aug. 01, 2023 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced a poster presentation at the 2023 American Society of Gene and Cell Therapy (ASGCT) Spotlight on Immuno-oncology, which is being held from August 1-2, 2023, in Seattle, WA. “We are pleased to share data at this year's ASGCT Spotlight on Immuno-oncology on... Read More

LONDON and NEW YORK, July 19, 2023 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced that after a successful inspection by the Irish Health Products Regulatory Authority (HPRA), it has received a Commercial Manufacturer’s/Importer’s Authorization (MIA) for its Quality Control (QC) testing facility at its GMP manufacturing site in Shannon,... Read More

Clinically meaningful improvements in xerostomia symptoms measured by two different xerostomia PROs demonstrated across both unilaterally and bilaterally treated cohorts at 12 months Increases in whole saliva flow rates observed post-treatment, providing objective evidence of biological activity, reaching the normal range in bilaterally treated participants by 2 months and persisting through the Month 12 assessment Across... Read More

LONDON and NEW YORK, June 22, 2023 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (NASDAQ: MGTX), a vertically integrated, clinical stage gene therapy company, today announced it will host a conference call and webcast to present an update on the Company’s AAV-hAQP1 clinical program for the treatment of grade 2/3 radiation-induced xerostomia (RIX) on Tuesday, June 27, 2023, at 8:00 a.m. ET. The presentation will include: 1) Data... Read More

LONDON and NEW YORK, May 16, 2023 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced the Company will exhibit nine poster presentations at the American Society of Gene and Cell Therapy (ASGCT) 2023 Annual Meeting, which is being held from May 16-20, 2023, in Los Angeles, CA. “Our presentations at this year’s ASGCT Annual Meeting reflect the... Read More

Raised approximately $60 million in a private placement in May 2023 with investors consisting of several of the Company’s top shareholders On track for BLA submission of botaretigene sparoparvovec (bota-vec, formerly AAV-RPGR) for the treatment of X-linked retinitis pigmentosa (XLRP) in 2024 Company will present 12-month data from bilateral treated cohorts from the AQUAx AAV-hAQP1 Phase 1 study for treatment of Grade 2/3... Read More

LONDON and NEW YORK, May 03, 2023 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced that it has agreed to sell approximately 10.7 million ordinary shares at a price of $5.75 per share. MeiraGTx anticipates aggregate gross proceeds from the private placement will be approximately $60 million. The Company intends to use the proceeds from this... Read More

LONDON and NEW YORK, April 21, 2023 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced two abstract presentations at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting . The first abstract will present humoral immune response data from the Company’s Phase 1/2 MGT009 trial for the investigational gene therapy... Read More

LONDON and NEW YORK, April 20, 2023 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (NASDAQ:MGTX), a vertically integrated, clinical stage gene therapy company, today announced that Alexandria Forbes, Ph.D., President and Chief Executive Officer, will participate in the following investor conferences: Chardan's Virtual 7th Annual Genetic Medicines and Cell Therapy Manufacturing Summit Corporate presentation: Monday, April 24,... Read More

Announced positive clinical data from AQUAx AAV-hAQP1 Phase 1 trial for treatment of Grade 2/3 Radiation-Induced Xerostomia in December 2022 Announced positive top-line data from the MGT009 Phase 1/2 clinical study demonstrating safety and improvement in multiple domains of vision in X-linked retinitis pigmentosa (XLRP) patients treated with botaretigene sparoparvovec (AAV-RPGR) compared to untreated randomized control at... Read More