PBAL05

PBAL05 is an adeno-associated virus (AAV)-based gene therapy candidate being developed by Passage Bio for the treatment of amyotrophic lateral sclerosis (ALS) and other adult neurodegenerative diseases driven by pathogenic expansions in the C9orf72 gene. It is designed to modulate the C9orf72–SMCR8 complex, delivering a therapeutic transgene to central nervous system cells via AAV-mediated gene transfer to correct disease-causing molecular abnormalities. Originating from the University of Pennsylvania’s Gene Therapy Program and optioned by Passage Bio, PBAL05 has been evaluated in preclinical models but the program has since been discontinued at the preclinical stage.