CIRM Funded Clinical Trials
Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency Using a Lentiviral Vector to Transduce Autologous CD34 Hematopoietic Stem Cells
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:
Institution:
CIRM Grant:
Award Value:
$12,000,000
Trial Sponsor:
University of California, San Francisco
Trial Stage:
Phase 1
Trial Status:
Recruiting
Targeted Enrollment:
25
ClinicalTrials.gov ID:
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.
