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Philly Cyclists Ride to Raise Awareness for Rare Diseases

Bicyclists in Philadelphia are riding to raise awareness and money for rare diseases that affect some 200,000 Americans.


Passage Bio Announces Third Gene Therapy Development Program in Krabbe Disease and Supports Million Dreams Fundraising Gala

Check out this exciting news from our collaborators at Passage Bio!


Amicus Therapeutics and the University of Pennsylvania Announce Major Expansion of Gene Therapy Collaboration

Amicus Therapeutics and the Penn Gene Therapy Program markedly expand their research collaboration with plans to include most lysosomal storage disorders, 12 additional rare diseases, and $50M in Next Generation discovery efforts!


Amicus, Penn expand gene therapy collaboration in a big way

Our amazing expanded collaboration with Amicus Therapeutics has been featured in Philadelphia Business Journal! #raredisease #genetherapy


Newly Approved Spinal Muscle Atrophy Treatment Zolgensma is Based on Delivery System Discovered by Penn Gene Therapy Pioneer

Approval Marks a New Milestone in Quest to Treat Rare Diseases


Q&A with Dr Jim Wilson Featured in Benchmarks Magazine

Benchmarks spoke with Jim Wilson, MD, PhD, director of the Gene Therapy Program and the Orphan Disease Center, about how the Philadelphia region has grown to become an engine for the development of gene-based therapies for patients with inherited, rare diseases.