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.

CHOP and Penn researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders directly within the body.

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.

CHOP researchers will develop gene therapies for multiple sulfatase deficiency, a devastating lysosomal storage disorder, with the goal of first-in-human trials.

CHOP gene therapy expert Dr. Lindsey George reacts to a phase 3 hemophilia gene therapy study in an editorial in the New England Journal of Medicine.

CHOP will be a provider in Independence Blue Cross’s new Advanced Network for Gene-Based Therapeutics, effective March 1, 2023.

Researchers from Children’s Hospital of Philadelphia (CHOP) have found that economically disadvantaged children treated with CAR T-cell therapy for relapsed/refractory acute lymphoblastic leukemia (ALL) fair just as well as children from more socioeconomically advantaged backgrounds.

In a short documentary by the New England Journal of Medicine, Dr. Alexis Thompson discusses the early successes of gene therapy, as well as its risks and benefits.

A leading study site for nearly a decade, CHOP will be part of a network of treatment centers in the country offering the therapy to patients who require regular blood transfusions.

Four CHOP researchers are part of a world-class research team that will receive $25 million to study solid tumors in children, funded by Cancer Grand Challenges.