Press release
MeiraGTx Announces an Oral Presentation and Eight Posters at the European Society of Gene and Cell Therapy (ESGCT) 2023 Annual Congress
Multiple Posters and Oral Presentations Highlight the Depth of Innovation from MeiraGTx’s Technology Platforms for Gene and Cell Therapy LONDON and NEW YORK,
About this update from Meiragtx Holdings Plc
[{"type":"text","content":"Multiple Posters and Oral Presentations Highlight the Depth of Innovation from MeiraGTx’s Technology Platforms for Gene and Cell Therapy\nLONDON 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 presentation at this year’s ESGCT congress underscores our commitment to remain at the forefront of genetic medicine by developing a new generation of therapies for multiple indications, including xerostomia and ALS,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “The collective data presented provide further clinical and preclinical validation of our platform and reflect the progress we continue to make, particularly in riboswitch and neurodegenerative programs. We look forward to sharing data at ESGCT highlighting our innovative pipeline, manufacturing, and R&D capabilities.” The posters will be available on the Posters and Publications page of the Company’s website. The details of the oral and poster presentations are below: Oral Presentation OR82: Preclinical Efficacy of AAV-hUPF1 with an optimized vector genome and novel CNS capsid: Gene Therapy for ALS and FTDFriday, October 27th at 11:00-13:00 CEST in Shed 2B (3rd talk)Session 11b: CNS and Sensory Disease III ALS is a progressive neurodegenerative disease that affects neurons in the brain and spinal cord. Here, we present preclinical efficacy of AAV-hUPF1 in multiple models of ALS. hUPF1 is an RNA helicase that regulates NMD that we have optimized for AAV gene therapy of ALS. At 4.9 kb, the optimized hUPF1 construct is 1.5 kb smaller than the original, thus offering AAV packaging and manufacturing benefits while demonstrating greater efficacy. When tested in vitro, the optimized AAV-hUPF1 rescued toxicity in both the TDP-43 and C9orf72 iNeurons models, at lower MOIs than the original. Optimized AAV-hUPF1 also rescued the pathophysiology of neurons derived from C9orf72 patient cell lines. Transduction in C9 patient-derived neurons indicated target engagement with down-regulation of C9 int...