Passage Bio is a genetic medicines company based in Philadelphia, PA that is developing a deep pipeline of adeno-associated virus (AAV) delivered therapeutics for the treatment of rare monogenic central nervous system disorders (CNS).

We seek to deliver transformative therapies to patients by leveraging our team’s decades of experience, including our founders, as well as the transformative potential genetic medicine technology to develop treatments that improve outcomes for patients with serious, life-threatening, rare CNS disorders.


Patients are at the center of every decision we make. Key elements for our strategy include:

  • Focus on rare, underserved CNS indications for which we can have a transformative impact on patients’ lives
  • Rapidly advance our lead product candidates into clinical development through commercialization
  • Advance and expand our pipeline by identifying, advancing into the clinic and developing additional product candidates
  • Extend existing and establish new relationships with patients and patient advocacy groups
  • Continue to develop proprietary manufacturing capabilities
  • Expand our existing collaboration with University of Pennsylvania's (Penn) Gene Therapy Program (GTP) and selectively enter into new discovery relationships where appropriate

Critical to this strategy is what we believe to be a differentiated approach to genetic medicine development via our collaboration with Penn’s GTP and Orphan Disease Center, led by our co-founder and Chief Scientific Advisor Dr. James Wilson. The differentiation is focused on (1) a rigorous process for selecting product candidates, (2) mitigation of early development risks prior to IND submission, and (3) mitigation of clinical development risks.

In addition to our strategy, we have recruited, beginning with our founders, Dr. Squinto, Dr. Wilson and Dr. Yamada, a world class team of leaders who have decades of experience in working in, creating and successfully growing rare disease, central nervous disorders and gene therapy focused companies.

We have assembled a deep pipeline of genetic medicine product candidates, including our three lead product candidates: PBGM01 for the treatment of GM1 gangliosidosis (GM1), PBFT02 for the treatment of frontotemporal dementia (FTD) and PBKR03 for the treatment of Krabbe disease.

Our Mission

Our mission is to discover and develop life-transforming therapies for patients with rare monogenic CNS disorders, while also building strong relationships with the communities we serve.