The University of Pennsylvania's Gene Therapy Program (GTP) and The Center for Breakthrough Medicines (CBM) have signed a landmark collaboration agreement to combine Penn's knowledge and expertise in the development of gene therapies with CBM's manufacturing capacity and singular focus on advanced therapies.
GTP announced the continued expansion of its R&D partnership with Passage Bio, a Penn spinout focused on developing transformative therapies for central nervous system disorders. Passage Bio exercised two additional options with GTP, which focused on CNS diseases with high unmet need.
Swipe to view
Discovery
GTP is innovating high-performance vectors to enhance the safety and efficiency of genetic therapeutics.
Preclinical
GTP provides preclinical pharmacologic and safety testing in appropriate in vitro and in vivo models, as well as IND-enabling studies designed to support the advancement of a clinical candidate to first-in-human clinical trials.
IND
GTP provides strategic regulatory guidance and support for the Investigational New Drug (IND) application in order to advance a product candidate to clinical trials.
Phase I
Phase I trials assess the safety and dose-dependent effects of a treatment in healthy volunteers.
Phase II
Phase II trials assess the effectiveness of a treatment in a small group of patients, typically up to several hundred. For rare diseases, however, studies may only be able to enroll one to three patients per year for a given site.
Phase III
Phase III studies involve randomized, blind testing on a large patient population. FDA approval for marketing of a therapy can be requested when Phase III studies are complete.
Commercialization
The cell and gene therapy industry represents a global market that is expected to exceed $20 billion by 2025. With recently approved products in both the US and EU, this industry is poised for dramatic growth.
Wilson Lab
Core Laboratories
(Vector, Immunology, Animal Models, Histology, and Biostatistics)
Penn Hospital
CHOP
Partner
