Duplicate report for OM#3 (was 4)
Jasper has developed a first-in-class monoclonal antibody (mAb) to target and deplete recipient hematopoietic stem cells (HSCs) to permit donor HSC engraftment in SCID patients without chemotherapy. The mAb, JSP191, targets CD117, a receptor tyrosine kinase present on the HSC surface that provides essential survival signals to HSC via its ligand, stem cell factor (SCF). CIRM supported the translation from preclinical studies through the IND filing to the current Phase I/II clinical trial in SCID. JSP191 has been shown to be safe, and is well-tolerated and efficacious in facilitating HSC engraftment. As a result, patients demonstrate immune reconstitution and clinical benefit from the JSP191-conditioned hematopoietic cell transplantation (HCT). Funding from this grant has successfully supported enrollment / dosing of the first 21 patients in the trial.