MeiraGTx Holdings plc Stock News

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 ...

MeiraGTx Holdings plc is advancing AAV-AIPL1 for AIPL1-associated Leber congenital amaurosis 4, with a near-term BLA filing in the UK under Exceptional Circumstance possible. Bota-vec, developed with Janssen, could see a BLA filing for X-linked Retinitis Pigmentosa in 2025. Initiation of phase 3 study, using AAV-GAD for the treatment of patients with Parkinson's Disease, expected in 2025.

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 MeiraGTx has recently received Rare Pediatric Disease Designation (RPDD) for four inherited retinal diseases (IRDs) reflecting the transformative therapeutic potential of the Comp...

MeiraGTx Holdings plc has rallied over 40% following promising Parkinson's disease data, with significant potential in its Riboswitch platform for gene expression control. The company's diverse pipeline includes gene therapies for salivary gland conditions, neurological disorders, and inherited retinal diseases, with multiple BLAs expected from 2024-2027. Recent strategic deals with J&J and San...

MeiraGTx Holdings plc's GAD gene therapy for Parkinson's Disease shows promising phase 1/2 results, with significant improvements in UPDRS Part 3 and PDQ-39 scores. The company plans to discuss phase 3 study designs with regulatory agencies in Q4 2024, potentially initiating late-stage studies in 2025. MeiraGTx secured $50 million in funding, including a $30 million equity investment from Sanof...

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