Drs. Katherine High, Jean Bennett and Albert Maguire honored for trailblazing work on the first FDA-approved gene therapy for an inherited condition

KJ is celebrating a big year after becoming the first patient in the world to receive a personalized CRISPR gene editing therapy to treat his rare disease.

Certification recognizes excellence in clinical care and research and highlights CHOP’s commitment to advancing care for children living with muscular dystrophy.

Landmark study from CHOP and Penn Medicine showcases the power of customized gene editing therapy to treat patient with rare metabolic disease.

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.

Made possible with a $4 million grant from the Brazilian Ministry of Health, CHOP cell therapy specialists will offer their expertise and resources to oversee this initiative.

Evan Weber will use this “high risk, high reward” grant to fund research into improving CAR-T therapies.

The findings from the clinical trial were published in the New England Journal of Medicine.

CHOP researchers find exposing cells to a small molecule drug improves cell fitness during hematopoietic stem cell transplants, which could improve ex vivo gene therapy.

A Penn Medicine and CHOP team will seek to develop treatments for three rare, incurable genetic diseases with the help of a $26 million grant from the National Institutes of Health.