CRISPR-Edited HLA Donor Kidney Transplant to Reduce Rejection Risk

PHASE1/PHASE2RecruitingINTERVENTIONAL
Enrollment

90

Participants

Timeline

Start Date

June 1, 2025

Primary Completion Date

December 18, 2027

Study Completion Date

December 28, 2028

Conditions
End-Stage Renal DiseaseEnd Stage Renal Disease on DialysisEnd Stage Renal Disease With Renal TransplantKidney Transplant RejectionKidney TumorKidney FailureKidney Ischemia
Interventions
BIOLOGICAL

Ex Vivo CRISPR-Cas9 Gene Editing of Donor Kidney

The donor kidney is treated outside the body with CRISPR-Cas9 ribonucleoprotein complexes targeting the genes HLA-A, HLA-B, and CIITA. This genetic intervention knocks out HLA-A/B on donor cells and disables expression of HLA-DR, -DQ, -DP by disrupting CIITA (essential for class II antigen presentation). The goal is to create a transplanted organ with greatly reduced immunogenic surface proteins. (This is a one-time genetic manipulation applied to the donor organ prior to transplantation; no direct gene therapy is given to the patient's own cells.)

PROCEDURE

Kidney Transplantation with Standard Care

After gene editing, the donor kidney is implanted into the recipient in a surgical transplant procedure. Standard peri-operative care is provided. All patients will receive standard immunosuppressive therapy post-transplant (such as tacrolimus, mycophenolate, and prednisone, per center protocol) to prevent rejection, though the regimen may be tailored based on the edited graft's expected lower immunogenicity. Patients will be hospitalized for transplant and monitored closely during the immediate post-op period, then followed in clinic frequently for transplant aftercare.

Trial Locations (1)

102206

RECRUITING

Peking University Health Science Center (PKUHSC), Beijing

All Listed Sponsors
lead

AMERICAN ORGAN TRANSPLANT AND CANCER RESEARCH INSTITUTE LLC

OTHER