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Cell and Gene Therapy Innovations News

Building on our proven track record in cell and gene therapy, CHOP is committed to discovering the next life-changing treatments for the most serious diseases. 

Trails

Children’s Hospital of Philadelphia Researchers Announce Promising Results from First-of-its-Kind, Multicenter, Phase 1 Gene Therapy Trial for Danon Disease

Nov 18, 2024

Researchers at Children’s Hospital of Philadelphia (CHOP) announced encouraging results from the first ever gene therapy trial for Danon disease (DD), a rare, X-linked heart condition caused by a single gene mutation. The data on the results of the RP-A501 Phase 1 trial, presented at a late breaking session today at the American Heart Association Scientific Sessions 2024 in Chicago, were also published in the New England Journal of Medicine.

CHOP Treats 500th CAR T Patient

Jul 17, 2023

CHOP has treated its 500th patient with CAR T-cell immunotherapy, a “living drug” that harnesses the power of a patient’s own immune system to fight cancer.

FDA Approves First Gene Therapy for Duchenne Muscular Dystrophy

Aug 22, 2023

For decades, doctors and researchers have been working diligently to discover new treatments and provide more options for patients with Duchenne muscular dystrophy (DMD), a debilitating genetic disease that ravages the muscles in the body and leads to a loss of movement and coordination.

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