MeiraGTx Holdings plc - Ordinary Shares (MGTX)

-  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

-  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 to receive $200 million in upfront cash consideration

LONDON and NEW YORK, Feb. 21, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced the publication of results from the first-in-human interventional study to treat children with AIPL1-associated severe retinal dystrophy. The data were published in The Lancet in a paper titled, "Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study". This paper presents data from the first 4 children treated unilaterally with rAAV8.hRKp.AIPL1. A further 7 children with LCA4 were treated bilaterally with rAAV8.hRKp.AIPL1 demonstrating similarly remarkable effects on vision, with all 11 of the 11 children treated who were blind at birth now having visual acuity in the treated eyes.

MeiraGTx has recently received Rare Pediatric Disease Designation (RPDD) for four inherited retinal diseases (IRDs) reflecting the transformative therapeutic potential of the Company’s proprietary technology platforms

-  RMAT designation recognizes the preliminary clinical evidence of the potential benefit of AAV2-hAQP1 as a one-time treatment for this debilitating condition

LONDON and NEW YORK, Nov. 13, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced financial and operational results for the third quarter ended September 30, 2024, and provided a corporate update. The Company also announced that following meetings with the UK Medicines and Healthcare products Regulatory Agency (MHRA), the Company intends to submit a Marketing Authorization Application under exceptional circumstances for AAV-AIPL1 in the United Kingdom without the need for further clinical studies. The Company is also initiating discussions with the FDA around the potential for a similar pathway to approval in the U.S. In addition, MeiraGTx announced that the Offices of Orphan Products Development and Pediatric Therapeutics of the U.S. Food and Drug Administration (FDA) have granted the Company three Rare Pediatric Disease Designations to its AAV8-RK-AIPL1 program, AAV8-RK-BBS10 program, and AAV5-RDH12 program, each for the treatment of inherited retinal diseases.

Multiple Posters Highlight the Breadth of Company’s Novel Genetic Medicine and Cell Therapy Platforms

The primary study objective of safety and tolerability was met

Results indicate a potent and effective locally delivered AAV-BDNF gene therapy for the treatment of obesity caused by MC4R deficiency

- 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

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 and offering expenses. All of the ordinary shares in the offering are being sold by MeiraGTx.

- 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

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:

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.

- 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

MeiraGTx to receive $50 million milestone after 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)

- 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

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:

- 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

Best Growth Stocks, a leading independent research and corporate access firm focused on finding and reporting on the best growth stocks utilizing exclusive ai-assisted research recently issued a comprehensive analysis on MeiraGTx Holdings plc a vertically integrated, clinical stage gene therapy company.

LONDON and NEW YORK, Oct. 30, 2023 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced strategic updates including that Sanofi has made a $30 million strategic investment in the Company through the purchase of 4.0 million ordinary shares at a price of $7.50 per share.

Multiple Posters and Oral Presentations Highlight the Depth of Innovation from MeiraGTx’s Technology Platforms for Gene and Cell Therapy

LONDON and NEW YORK, Aug. 10, 2023 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced financial and operational results for the second quarter ended June 30, 2023, and provided a corporate update.

RiboCAR-T cell activity can be precisely tuned and “remotely” controlled to improve the efficacy, durability and safety of CAR-T cell therapy