Release Date: 09-Sep-2021
Jasper Therapeutics, Inc., a biotechnology earns orphan drug designation from US FDA to JSP191, a monoclonal antibody targeting the CD117 (stem cell factor) receptor, for conditioning treatment prior to hematopoietic stem cell transplantation. JSP191 is a humanized monoclonal antibody in clinical development as a conditioning agent that blocks stem cell factor receptor signaling leading to clearance of hematopoietic stem cells from bone marrow, creating an empty space for donor or gene-corrected transplanted stem cells to engraft. It is currently enrolling in 2 clinical trials for myelodysplastic syndromes (MDS)/acute myeloid leukemia (AML) and severe combined immunodeficiency (SCID).
The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S and it also gives several benefits, like tax credits for qualified clinical trials, potential seven years of market exclusivity after approval and exemption from user fees.
Severe combined immune deficiency (SCID) is caused by mutations in genes involved in the development and function of infection-fighting immune cells. Infants having SCID appear healthy at the time of birth but are really vulnerable to severe infections. The condition is serious, usually within the first year or two of life, unless infants receive immune-restoring treatments, such as transplants of blood-forming stem cells, gene therapy or enzyme therapy.