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-approved gene therapies for hematological disorders
FDA approves two gene therapies for 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).
Programs and services
These clinical programs offer experimental and approved gene therapies for hematological disorders.