MeiraGTx Holdings plc will present four posters at the 2025 ASGCT Annual Meeting, showcasing advancements in gene therapy for various conditions including severe pediatric obesity, ALS, and frontotemporal dementia. The company is highlighting its research on optimized promoters for gene therapy and a novel approach to treating obesity through targeted BDNF delivery. This research also underscores MeiraGTx’s commitment to developing innovative gene therapies.
These advancements are important because they address significant unmet medical needs. The research on pediatric obesity offers a potential solution for a serious condition often resistant to traditional treatments, while the work on ALS and FTD explores promising therapies for debilitating neurodegenerative diseases. The promoter engineering research is crucial for advancing the entire field of gene therapy, potentially leading to safer and more effective treatments across a wider range of diseases by allowing for lower doses of viral vectors.
MeiraGTx has developed a more potent AAV-BDNF vector for treating obesity, achieving significant weight loss in preclinical models. Their engineered CAG promoter variants are shorter and more potent than traditional promoters, which is vital for gene therapy applications involving large transgenes. The company is also investigating transcription factor binding sites to further enhance synthetic promoter activity. For their ALS and FTD program, they have optimized the AAV-hUPF1 construct, improving packaging and efficacy, and are working on developing a robust potency assay for clinical manufacturing.
These developments position MeiraGTx at the forefront of gene therapy innovation. The combination of optimized vectors, potent promoters, and targeted therapies has the potential to transform treatment paradigms for several diseases. The continued development and eventual clinical translation of these technologies could significantly impact patient outcomes and shape the future of genetic medicine.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
