Passage Bio, a genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system disorders, announced that U.S. Food and Drug Administration has cleared an investigational new drug (IND) application for the company’s lead product candidate, PBGM01, an adeno-associated virus (AAV)-delivery gene therapy that is being studied for the treatment of infantile GM1 gangliosidosis (GM1).
GM1 is a rare and often life-threatening CNS disorder with no approved disease-modifying therapies available. The company expects to dose the first patient for the global PBGM01 clinical trial program in the first quarter of 2021.