Skip navigation

Catalyzing the development of gene therapeutics from concept to clinic

The Gene Therapy Program is a complex R&D organization with the internal capacity to perform gene transfer studies from basic research through phase 1/2 proof-of-concept human trials.

About Us

Providing investigators access to state-of-the-art vector technology

The Vector Core is a full service core facility with over a decade of experience in the production of viral-based vectors.

Vector Core Services

Partnering with industry leaders to advance the field of genetic therapeutics

The Gene Therapy Program aims to discover transformative genetic-based therapeutics, which are translated to proof-of-concept in clinical trials and commercialized through partnerships to assure access to patients in need.

Partnered Programs

Leading the field in AAV vector innovation

Next Generation AAV Vectors

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
Learn More

Swipe to view

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

smaneuron
Explore Programs

Spinal Muscular Atrophy (SMA)

A rare neuromuscular disorder

Spinal muscular atrophy (SMA) is a rare genetic disorder caused by mutations in the SMN1 gene. These mutations lead to a deficiency of the survival motor neuron (SMN) protein, which results in the loss of specialized nerve cells in the spinal cord and brainstem such that information cannot be passed from the brain to the muscle. This leads to severe muscle weakness and loss of muscle control, as well as impaired breathing and swallowing in severe cases. As part of a collaborative alliance with Biogen, GTP is developing an AAV gene therapy approach to elicit long-term expression of SMN1 in motor neurons for the treatment of SMA.

immunology services
Explore Programs

Crigler-Najjar Syndrome

A rare, congenital autosomal recessive monogenic disease

Crigler-Najjar Syndrome (CN) is a rare genetic disorder caused by a defect of the UGT1A1 gene, which encodes the protein responsible for metabolizing bilirubin. The resulting toxic accumulation of bilirubin can cause severe neurological impairment and death. Phototherapy can help manage bilirubin levels, but requires 10-12 hour treatments each day and becomes less effective with age, at which point a liver transplant is required. GTP and Audentes Therapeutics are collaborating to advance AAV8-UGT1A1 (AT342) for the treatment of CN. A phase 1/2, multinational, open-label, ascending-dose, delayed-treatment concurrent control clinical study is planned (VALENS; NCT03223194).

2/12/2020

CRISPR “Minigene” Approach Stops Genetic Liver Disease in Mice

A new CRISPR gene-editing technique prevented a genetic liver disease known to be driven by hundreds of different mutations and improved clinical symptoms in mice, Penn Medicine researchers reported in new proof-of-concept study published online in Science Advances. The findings suggest a promising CRISPR tool that could potentially treat patients with a rare metabolic urea-cycle disorder caused by a deficiency the enzyme, ornithine transcarbamylase (OTC), as well as other hereditary diseases triggered by different mutations on the same gene.

 View All News

1/28/2020

FDA Continues Strong Support of Innovation in Development of Gene Therapy Products

This is a pivotal time in the field of gene therapy as the FDA continues its efforts to support innovators developing new medical products for Americans and others around the world. To date, the FDA has approved four gene therapy products, which insert new genetic material into a patient’s cells. The agency anticipates many more approvals in the coming years, as evidenced by the more than 900 investigational new drug (IND) applications for ongoing clinical studies in this area. The FDA believes this will provide patients and providers with increased therapeutic choices.

 View All News