The remedy is constructed by introducing TCF4 isoform B into an AAV9 expression cassette. Credit score: Inventory-Asso/Shutterstock.com. Mahzi Therapeutics has dosed the primary affected person in its Part I/II UNITE trial evaluating MZ-1866, an investigational gene remedy concentrating on Pitt Hopkins syndrome. The multicenter, open-label, international examine will examine the tolerability, early efficacy and security of a single administration of […]