FDA Grants Special Status to Cellectis' UCART22 for Acute Lymphoblastic Leukemia

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug (ODD) and Rare Pediatric Disease Designation (RPDD) Status to Cellectis' UCART22 product candidate for the treatment of Acute Lymphoblastic Leukemia (ALL). ALL, which accounts for 10% of all leukemia cases in the US, progresses rapidly and is typically fatal if left untreated. UCART22 is an allogeneic CAR T-cell product candidate targeting CD22 and evaluated in BALLI-01, a Phase 1/2 open-label dose-escalation and dose-expansion study. Clinical data presented at the American Society of Hematology in December 2023 showed that UCART22-P2 (fully manufactured at Cellectis) is more potent with a preliminary response rate of 67% at Dose Level 2, compared to a 50% response rate with UCART22-P1. Cellectis expects to provide updates on BALLI-01's progress by year-end 2024.