Mahzi Therapeutics

From Autism to Action: A New Model in Precision Neuroscience

About this Event

Mahzi Therapeutics is building something rare in biotechnology: a precision neuroscience company designed from the ground up to work closely with patients and advocacy groups to develop treatments for children with epilepsy, autism, and other severe neurodevelopmental disorders.

These disorders are common – each year, up to 1 in 6 children in the U.S. are diagnosed with a neurodevelopmental condition. Advances in genetics have identified hundreds of causal genes, yet most families still receive a diagnosis without a path forward. Mahzi exists to change that.

The company focuses on rare, genetically defined neurological diseases with clear biology, limited competition, and strong patient advocacy engagement. Its lead program, an AAV9 gene therapy targeting Pitt Hopkins syndrome has begun a Phase 1/2 clinical trial and an IND filing is planned for a second AAV9 program for WOREE syndrome in H1 2026. Additional programs are advancing behind them with the support of both patient organizations and academic collaborators

Please watch our online event with Mahzi founder and CEO, Yael Weiss, MD, PhD. With over $60M raised and meaningful non-dilutive support, Mahzi is entering a data-rich clinical phase with the potential to deliver precision therapies for families who currently have none.

Video On Demand

– Recorded

February 17

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