Press release
MeiraGTx Announces Poster Presentation on a Potential Treatment for MC4R Genetic Deficiency at the 2024 Society for Neuroscience Conference
Results indicate a potent and effective locally delivered AAV-BDNF gene therapy for the treatment of obesity caused by MC4R deficiency LONDON and NEW YORK,
About this update from Meiragtx Holdings Plc
[{"type":"text","content":"Results indicate a potent and effective locally delivered AAV-BDNF gene therapy for the treatment of obesity caused by MC4R deficiency\nLONDON and NEW YORK, Oct. 09, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicine company, today announced a poster presentation at the 2024 Society for Neuroscience Conference (SfN), which is being held from October 5-9, 2024, in Chicago, IL. “We are pleased to share data at this year’s Society for Neuroscience Conference on the remarkable efficacy of our AAV-BDNF treatment in diet-induced obesity animal models,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “We leveraged our proprietary vector design platform to create a highly potent construct with the potential to treat children with severe early-onset obesity caused by MC4R genetic deficiency via the local delivery of a small dose of AAV-BDNF to a specific site in the ventromedial hypothalamus. This represents a powerful mechanism to treat these severely affected patients with no current treatment options available.” The poster is available on the Posters and Publications page of the Company’s website on October 9, 2024. The details of the poster presentation are as follows: Poster Number: 5090Abstract Title: A CNS-targeted gene therapy for the treatment of severe pediatric obesityDate: October 9, 2024Time: 1pm CT In the ventromedial hypothalamus, the leptin-proopiomelanocortin pathway interprets energy signals from the periphery to initiate feeding or fasting through two opposing neuronal populations. In a fed state, elevated leptin signals a decrease in food intake via the release of brain derived neurotrophic factor (BDNF) from Melanocortin 4 receptor (MC4R) expressing neurons. Loss-of-function mutations along this pathway, including in MC4R or BDNF, cause severe obesity in humans. MC4R deficiency leads to a severe form of early-onset pediatric obesity that is characterized by an increased drive to eat and impaired satiety. Hyperphagia and food-related distress have been reported in patients as young as 8 months old. Current therapeutic approaches such as bariatric surgery and glucagon-like peptide 1 (GLP1) agonists do not result in significant, durable treatment for persons with MC4R deficiency. MC4R agonists are being developed, but ...