Passage Bio to explore therapeutic potential of PBFT02 in multiple diseases

“Driven by promising initial data for PBFT02 in FTD-GRN and evidence supporting progranulin’s role in neurodegeneration, we are refining our strategic priorities to explore the therapeutic potential of PBFT02 in multiple diseases, including FTD-C9orf72, amyotrophic lateral sclerosis, and Alzheimer’s disease,” said William Chou, M.D., president and chief executive officer at Passage Bio. “As we pursue this strategy, we are actively exploring potential partnerships to advance our GM1 gangliosidosis program as well as our other clinical-stage pediatric programs. This shift in strategy aims to optimize focus and resources and provides each of our gene therapy candidates the best chance to get to patients in need.” Strategic Priorities: Continuing clinical development of PBFT02 to treat FTD-GRN. Pursuing PBFT02 in additional adult neurodegenerative diseases, including FTD-C9orf72, ALS and Alzheimer’s disease. Prioritizing Huntington’s disease preclinical program through existing Penn Gene Therapy Program partnership. Pursuing potential partnership opportunities for clinical-stage pediatric programs in GM1 gangliosidosis, Krabbe disease and metachromatic leukodystrophy.