News

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.

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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).

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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.

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Penn Medicine at the American Society of Gene and Cell Therapy 26th Annual Meeting

Nine researchers from the University of Pennsylvania’s Gene Therapy Program (GTP) presented abstracts highlighting their translational science and discovery research on gene therapy, gene editing, and adeno-associated virus (AAV) vector technology at the American Society of Cell and Gene Therapy (ASGCT)’s 26th Annual Meeting.

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Finding a Home for Orphan Diseases

Forty years after the Orphan Drug Act passed, researchers advance drug development for neglected rare conditions everywhere from the lab bench to backstage.

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