Briquilimab (JSP-191) is a non-toxic humanized monoclonal antibody targeting CD117 (c-Kit) to deplete hematopoietic stem cell (HSC). Briquilimab has safety to clear host marrow niche space to enable sufficient donor HSC engraftment and immune reconstitution as primary method of severe combined immunodeficiency (SCID)[1].
[1]. Agarwal R, et al. First report of non-genotoxic conditioning with JSP191 (anti-CD117) and hematopoietic stem cell transplantation in a newly diagnosed patient with severe combined immune deficiency[J]. Blood, 2020, 136: 10.