Mahzi Therapeutics

Mahzi Therapeutics is a clinical-stage biotechnology company dedicated to developing precision therapies for rare, genetically defined neurodevelopmental disorders, such as Pitt Hopkins syndrome, WOREE syndrome, and CHD2 deficiency. The company distinguishes itself by working closely with patient advocacy groups, academic researchers, and industry partners from the earliest stages of drug discovery to accelerate the development of gene and RNA-based therapies. Founded in 2021 and headquartered in South San Francisco, Mahzi utilizes a collaborative model to bridge the gap between academic research and clinical application. With over $60 million in funding and support from prominent venture capital firms, the company is advancing its lead AAV9 gene therapy program into clinical trials while maintaining a pipeline of additional preclinical assets aimed at addressing significant unmet medical needs in the rare disease space.