Press release
MeiraGTx Announces the Presentation of Four Posters at the American Society of Gene and Cell Therapy (ASGCT) 2025 Annual Meeting
Multiple Poster Presentations Highlight the Depth and Novelty of MeiraGTx’s Technology Platforms for Gene and Cell Therapy LONDON and NEW YORK, May 13, 2025
About this update from Meiragtx Holdings Plc
[{"type":"text","content":"Multiple Poster Presentations Highlight the Depth and Novelty of MeiraGTx’s Technology Platforms for Gene and Cell Therapy\nLONDON and NEW YORK, May 13, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced the Company will exhibit four poster presentations at the American Society of Gene and Cell Therapy (ASGCT) 2025 Annual Meeting, which is being held from May 13-17, 2025, in New Orleans, LA. The posters will be available on the Posters and Publications page of the Company’s website. The details of the poster presentations are below: Poster 507: An Ultra-Low Dose of a Localized CNS Gene Therapy for Severe Pediatric ObesityPoster Session: Tuesday, May 13, 6:00-7:30 pm CDTAbstract:Brain-derived neurotrophic factor (BDNF) is a secreted growth factor that promotes neuronal health throughout the central nervous system and is a key signaling component of metabolic homeostasis. In the ventromedial hypothalamus (VmH), elevated leptin signals melanocortin 4 receptor (MC4R) expressing neurons to release BDNF, which then acts through tropomyosin receptor kinase B (TrkB) to signal fullness and reduce food intake. Patients with loss-of function mutations along this pathway develop severe early-onset obesity syndromes characterized by hyperphagia and food-related distress as young as six months old. Current therapeutic approaches such as bariatric surgery and glucagon-like peptide 1 (GLP1) agonists may be effective for generalized obesity, but do not result in significant, durable treatment for individuals with MC4R or BDNF deficiency. By directly delivering BDNF to the VmH through an adeno-associated virus (AAV)-based gene therapy, we aim to treat these individuals early in childhood in a safe, effective, and lasting way. Treating these children early could significantly improve their physical and emotional well-being and prevent the development of irreversible sequelae such as cancer and diabetes. To achieve this, we developed a more potent vector which could decrease the AAV gene therapy dose required to maintain effective weight loss, potentially mitigating adverse effects associated with high levels of AAV administration. Our optimized BDNF clinical construct, designed by altering various cis-regulatory components, expresses BDNF up to 143-fold ...