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Discovery

Discovery

Genome Editing
AAV 3.0

GTP is innovating high-performance vectors to enhance the safety and efficiency of genetic therapeutics.

Preclinical

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

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

Phase I trials assess the safety and dose-dependent effects of a treatment in healthy volunteers.

Phase II

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

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

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

Leading the field in AAV vector innovation

AAV 3.0™

This program represents our ambitious commitment to developing cutting-edge vector technologies. Our diversified approach focuses on delivering advanced vectors for traditional gene therapy applications as well as vectors tailored specifically for genome editing. The design of these transformative next-generation AAV vectors is being guided by experimental results from studies on the basic biology of AAV that leverage our world-class resources in molecular, structural, cell, and computational biology.

capsid mckenna small
Structure of AAV Capsid - Provided by M. Agbandje-McKenna