Current Status of Clinical Gene Therapy for Hemophilia and Globin Disorders - PubMed
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- #gene therapy
- #hemophilia
- #CRISPR
- Gene therapy has transitioned from experimental research to approved medicine for congenital blood disorders like hemophilia and hemoglobinopathies.
- Six gene therapies are now approved in the United States: Roctavian (hemophilia A), Beqvez and Hemgenix (hemophilia B), Lyfgenia (sickle cell disease), Zynteglo (β-thalassemia), and Casgevy (sickle cell disease or β-thalassemia).
- Therapies use various methods including in vivo and ex vivo approaches, lentiviral and AAV vectors, and CRISPR-Cas9 gene editing.
- Results are encouraging, often eliminating the need for coagulation factor or RBC infusions, but long-term effects and durability require further study.
- Improvements are needed in safety, therapeutic durability, and accessibility of these therapies.