Passage Bio is a genetic medicines company focused on developing AAV-delivered therapeutics to treat rare monogenic CNS diseases. The company works with the University of Pennsylvania’s Gene Therapy Program and Orphan Disease Center under research, collaboration and license agreements. Passage Bio has a development portfolio of five product candidates and has licensed two additional indications from Penn, with options to license up to seven more. The company planned to initiate clinical trials for lead programs in GM1 gangliosidosis and frontotemporal dementia in the first half of 2020, and a Krabbe disease program in the second half of 2020. Passage Bio is based in Philadelphia and conducts clinical development, regulatory strategy and commercialization while Penn’s GTP conducts IND‑enabling preclinical work. Financially, the company announced a $110.0 million Series B and, since launch, has raised $225.5 million in total. Passage Bio is developing a portfolio of AAV-delivered therapeutics targeting rare monogenic CNS diseases, with initial lead programs in GM1 gangliosidosis and frontotemporal dementia. The company has a research, collaboration and license agreement with the University of Pennsylvania’s Gene Therapy Program and Orphan Disease Center; Penn will perform IND-enabling preclinical work while Passage Bio is responsible for clinical development, regulatory, manufacturing and commercialization. Passage Bio plans a portfolio of five therapeutic candidates and holds an option to fund preclinical development of up to seven additional Penn programs and license resulting intellectual property. The company completed a $115.5 million Series A financing to advance its pipeline and build its team. Leadership and scientific expertise include co-founders James M. Wilson (scientific advisor), Stephen Squinto (interim CEO) and Tachi Yamada (chairman), and a Board with partners from key investors. The ODC will support natural history studies, KOL engagement and patient advocacy outreach as programs move toward the clinic.

Total raised

$226M

Funding rounds

2

Latest round

Series B

Latest activity

Sep 2019