Passage Bio and UPenn Orphan Disease Center AAV Gene Therapy and Natural History Study

Passage Bio was recently launched with the vision to be a first in class fully-integrated biotech company developing life transforming AAV-delivered in vivo therapeutics for the treatment of rare CNS diseases. In collaboration with the University of Pennsylvania Gene Therapy Program, Philadelphia-based Passage Bio plans to advance a portfolio of 5 rare disease indications in the neuro/CNS genetic space through IND-enabling studies using best-in-class AAV technology and know-how. Subsequently, Passage Bio will be responsible for all clinical development and will collaborate with Penn’s Orphan Disease Center to support these efforts.

The lead program for Passage Bio is for GM1 Gangliosidosis.   In addition, Penn’s Orphan Disease Center will also be initiating a natural history study for GM1.

Read the Press Release Here.

Watch this video for more information on the program and the launch.

We look forward to a future with multiple possible clinical trials for GM1 and the continued advancement of GM1 through a new natural history study to expand the understanding of all forms of GM1.

Please follow the web page and or our Facebook page for more news about this and other research being carried forward.  Updates will be provided regarding enrollment in both the natural history study and the Passage Bio clinical trial.