CIRM Funded Clinical Trials

A Phase I, Open-Label Study To Assess The Safety, Feasibility and Engraftment of Zinc Finger Nucleases (ZFN) CCR5 Modified Autologous CD34+ Hematopoietic Stem/Progenitor Cells (SB-728MR-HSPC) with Escalating Doses of Busulfan In HIV-1 (R5) Infected Subjects with Suboptimal CD4 Levels on cART


John Zaia
Disease Area: 
Investigator:
Institution:
CIRM Grant:
Award Value:
$5,583,438
Trial Stage: 
Phase 1
Trial Status: 
Recruiting
Targeted Enrollment:
12
ClinicalTrials.gov ID:
Details: 

Autologous hematopoietic stem cells gene edited ex vivo to eliminate expression of HIV entry co-receptor CCR5. Cells carrying disrupted CCR5 provide a renewable, long-lasting source of HIV-1 resistant immune cells.

Design: 

Single arm, non-randomised study.

Goal: 

Safety. Efficacy - engraftment.

Status: 

Enrolling.