MeiraGTx Announces the Presentation of Four Posters at the American Society of Gene and Cell Therapy (ASGCT) 2025 Annual Meeting

Core Insights - MeiraGTx Holdings plc is showcasing four poster presentations at the ASGCT 2025 Annual Meeting, highlighting advancements in gene and cell therapy technologies [1][19] Group 1: Poster Presentations - Poster 507 discusses an ultra-low dose localized CNS gene therapy targeting severe pediatric obesity, utilizing BDNF delivered via AAV to improve metabolic health and prevent severe obesity-related conditions [3][4][5] - Poster 893 presents novel CAG-based promoters that outperform traditional promoters in both mouse and human models, aiming to enhance gene therapy safety and efficacy by reducing required dosages [6][7][8][9][10] - Poster 897 explores the differential usage of transcription factor binding sites to enhance synthetic promoter activity, demonstrating significant improvements in gene expression through optimized designs [11][12][13][14] - Poster 1040 details the preclinical efficacy of an optimized AAV-hUPF1 gene therapy for ALS and Frontotemporal Dementia, showing promise in improving motor neuron survival and therapeutic efficacy [15][16][18] Group 2: Company Overview - MeiraGTx is a vertically integrated, clinical-stage genetic medicines company with a diverse pipeline, including four late-stage clinical programs targeting inherited and common diseases [19][20] - The company has developed a proprietary manufacturing platform and possesses comprehensive manufacturing capabilities, including GMP facilities for viral vector production [20] - MeiraGTx is focusing on innovative technologies for gene therapy, including riboswitch gene regulation technology for precise control of gene expression [20]