Award
National Heart, Lung, and Blood Institute 5R01HL170629-04
Chemotherapy-free cure of hemoglobin disorders through base editing
Recipient
Boston Children's Hospital
Award Amount
$730,175.00
Ceiling
$730,175.00
Awarded
August 03, 2026
Identifier
5R01HL170629-04
Award to Boston Children's Hospital for developing a chemotherapy-free gene therapy for hemoglobin disorders using base editing, with a funding amount of $730,175.
Description
This project aims to develop a chemotherapy-free gene therapy approach using base editing to treat severe blood disorders such as sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). The objectives include generating “stealth” HSPCs by multiplex CRISPR-Cas base-editing, validating efficacy on pre-clinical models, and optimizing manufacturing for clinical application.