Patient no longer needs regular blood transfusions for her inherited blood disorder, and her future has been transformed.

CHOP announced that its first patients outside of clinical trials have begun the treatment process for the newly FDA approved sickle cell gene therapies.

The life and legacy of the late Dr. Kwaku Ohene-Frempong was celebrated by family, friends and colleagues at his portrait unveiling ceremony on CHOP’s campus on June 20.

Earlier this month, Children’s Hospital of Philadelphia (CHOP) hosted the Cure Sickle Cell Walk & Family Fun Day presented by West Pharmaceutical Services at Citizens Bank Park raising more than $256,000 (and counting!) to support sickle cell warriors.

In a landmark study, an international consortium led by researchers at CHOP published the final results of a key clinical trial of the gene therapy CASGEVY for the treatment of sickle cell disease.

The Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel) and LYFGENIA™ (lovotibeglogene autotemcel), the first two gene therapies for the treatment of sickle cell disease  in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). 

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

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.

A group of researchers that includes CHOP presented new data on CRISPR-edited gene therapy for blood disorders at an international meeting.