Gene Therapy for Hematological Disorders

Children’s Hospital of Philadelphia (CHOP) has played a vital role in the development of gene therapies for hematological disorders. In these therapies, a vehicle called a vector delivers a functional gene to replace or do the work of a defective gene, or CRISPR technology is used to edit the defective gene.

CHOP investigators have been leaders since the 1990s in conducting basic, translational and clinical research efforts for hematological disorders. These research efforts have resulted in the realization of clinical trials for hemophilia gene therapies. For beta thalassemia and sickle cell disease, CHOP has played an instrumental role in clinical trials, including the trials that led to FDA approval of Zynteglo® for transfusion-dependent beta thalassemia and Casgevy™ for sickle cell disease.

FDA Approves Two Gene Therapies for Sickle Cell Disease

FDA-Approved Gene Therapies for Hematological Disorders 

  • Zynteglo® for adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions
  • Casgevy™ for patients between 12 and 35 years old with a history of severe sickle cell disease
  • Hemgenix® for adults with hemophilia B
  • Lyfgenia™ for patients between 12 and 35 years old with a history of severe sickle cell disease


Conditions We Treat 

At CHOP, we continually search for breakthroughs in the treatment of hematological disorders. There are four hematologic diseases we can treat with either an FDA-approved gene therapy or an experimental gene therapy being tested in a clinical trial.

Programs and Services 

These clinical programs offer experimental and approved gene therapies for hematological disorders.

Cell & Gene Therapy Supportive Programs

The path from an idea in the lab to a clinical trial and finally to an FDA-approved therapy winds through these support programs. It’s with the expertise of these programs that discoveries are ushered through the complicated but critical steps toward safe and effective treatments.