FDA Approves Gene Therapy for Epidermolysis Bullosa
Apr 29, 2025
ZEVASKYN™ is an ex vivo gene therapy approved for pediatric recessive dystrophic EB patients, where a genetically modified skin graft is applied to help wound healing.
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.
Apr 29, 2025
ZEVASKYN™ is an ex vivo gene therapy approved for pediatric recessive dystrophic EB patients, where a genetically modified skin graft is applied to help wound healing.
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.
Nov 7, 2024
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.
Oct 8, 2024
Evan Weber will use this “high risk, high reward” grant to fund research into improving CAR-T therapies.
May 6, 2024
The findings from the clinical trial were published in the New England Journal of Medicine.
Sep 26, 2023
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.
Aug 1, 2023
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.
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.
Jul 27, 2023
CHOP and Penn researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders directly within the body.
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.