CTA101

The utilization of CRISPR/ Cas9 technology facilitated highly efficient and precise gene editing, resulting in the production of universal CAR-T cells. No genotoxicity or chromosomal translocation associated with gene editing was observed. Six patients received infusions of CTA101 at doses of 1 (3 patients) and 3 (3 patients) × 106 CAR+ T cells per kilogram of body weight.

CRISPR/Cas9-engineered universal CD19/CD22 CAR-T cells exhibited a manageable safety profile and prominent anti-leukemia activity. Universal dual-targeted CAR-T cell therapy may offer an alternative therapy for patients with r/r ALL.

Prior to CTA101 infusion, pts received pre-conditioning chemotherapy regimen consisting of cyclophosphamide, fludarabine and alemtuzumab...All pts experienced CRS (3 G1, 2 G2, 1 G3), and the G3 CRS recovered within 7 days with one dose of tocilizumab and glucocorticoids...Early data of cellular PK and efficacy illustrate that CRISPR gene editing does not curtail the expansion and anti-leukemia capacity of CAR-T. Updated data, including long term gene editing-related AEs, will be presented after further follow-up.