For children born with severe combined immunodeficiency, life is extremely challenging. SCID leaves them without a functioning immune system, so even minor infections can be life‑threatening. Without treatment, many children die within the first few years of life.

CIRM-funded clinical trials

CIRM‑funded stem cell and gene therapy trials at UCLA and UCSF/St. Jude offer promising options. An alternative—and sometimes curative—approach is a bone marrow or hematopoietic stem cell transplant, which replaces the child’s blood system with one free of the SCID mutation and restores immune function.

However, current transplant methods require chemotherapy or radiation to destroy unhealthy blood stem cells and make room for healthy ones. This process is toxic, complex, and available only at major medical centers, limiting access for poor and underserved communities.

To change that, Dr. Judy Shizuru at Stanford University, with CIRM funding, developed an antibody that directs the patient’s own immune cells to kill diseased blood stem cells, creating room to transplant new, healthy cells. The goal was to make stem cell transplants safer and more effective for treating many life-threatening blood disorders.

Safer bone marrow transplants

Jasper Therapeutics is now championing that approach, and it just got very good news from the Food and Drug Administration (FDA). The FDA granted JSP191 Fast Track Designation, which can speed review of therapies designed to treat serious conditions and address unmet medical needs.

In a news release, Ronald Martell, President and CEO of Jasper Therapeutics, said this is good news for the company and patients: “This new Fast Track designation recognizes the potential role of JSP191 in improving clinical outcomes for these patients and will allow us to work more closely with the FDA in the upcoming months to determine a path toward a Biologics License Application (BLA) submission.”

A BLA would allow Jasper to market the antibody in the US and make it available to those who need it.

This is the third boost Jasper has received from the FDA. Previously, the agency granted JSP191 both Orphan and Rare Pediatric Disease designations. Orphan drug designation qualifies sponsors for incentives such as tax credits for clinical trials. Rare Pediatric Disease designation means that if the FDA eventually approves JSP191, Jasper can apply for priority review of an application to use the product for a different disease, such as sickle cell disease or severe autoimmune diseases, in patients undergoing a bone marrow transplant.