CIRM Funded Clinical Trials

Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency Using a Lentiviral Vector to Transduce Autologous CD34 Hematopoietic Stem Cells


Morton Cowan
Disease Area: 
Severe Combined Immunodeficiency, Artemis deficient (ART-SCID)
Investigator:
CIRM Grant:
Award Value:
$12,000,000
Trial Sponsor:
University of California, San Francisco
Trial Stage: 
Phase 1
Trial Status: 
Launching
Targeted Enrollment:
N/A
Details: 

UC San Francisco researchers aim to re­pair the damaged immune system of children born with severe combined immunodeficiency (SCID), a genetic blood disorder in which even a mild infection can be fatal. This trial will focus on SCID patients who have mutations in a gene called Artemis, the most difficult form of SCID to treat when using a standard bone marrow transplant from a healthy donor. The team will genetically modify the patient’s own blood stem cells with a functional copy of Artemis, with the goal of creating a new blood system and restoring the health of the immune system.

Design: 

Open label, single arm study.

Goal: 

Safety and efficacy. Multilineage engraftment persistence and B cell reconstitution.