News

Penn Medicine at the American Society of Gene and Cell Therapy 27th Annual Meeting

Researchers from the Gene Therapy Program (GTP) at the Perelman School of Medicine at the University of Pennsylvania presented ten research abstracts, two invited talks, and a workshop presentation highlighting translational science and discovery research on gene therapy, gene editing, and adeno-associated virus (AAV) vector technology.

View Full Post

FDA Approval for New Attempt to Treat Liver Ailment in Babies

iECURE, a gene editing company started by University of Pennsylvania scientist Dr. Jim Wilson, has received FDA approval to test a form of gene editing in infants for the first time in the United States.

View Full Post

The Million Dollar Bike Ride for Rare Diseases

What is the power of the humble bicycle? For Dr. Jim Wilson, a rare disease researcher at the University of Pennsylvania, the bicycle unleashed inspiration. Wilson turned his love of cycling into an annual event that has channeled $17 million into rare disease research.

View Full Post

Companion Penn Medicine Studies on AAV-based Gene Therapies in Non-Human Primates Suggest Integration into Human DNA is Unlikely to Drive Cancer Mutations while Offering the Potential for Durable Expression of the Transgene

Gene therapy adeno-associated viruses (AAVs) - viruses that can be engineered to deliver DNA to target cells - are unlikely to cause cancer-triggering insertions in humans or monkeys and may contribute to long-term efficacy, according to new research from the University of Pennsylvania’s Gene Therapy Program (GTP).

View Full Post

The University of Pennsylvania’s Gene Therapy Program partners with the Foundation for Angelman Syndrome Therapeutics to advance gene therapy candidate for Angelman Syndrome

The Foundation for Angelman Syndrome Therapeutics (FAST), a non-profit organization, has entered into an exclusive global collaborative research and development agreement with the University of Pennsylvania to develop an investigational adeno-associated virus (AAV) gene therapy for Angelman syndrome.

View Full Post