Exa-cel shows 95% TDT transfusion-free; CTX130 achieves 70% ORR in T-cell lymphoma

CRISPR Therapeutics AG

42 of 44 TDT patients transfusion-free; all 31 SCD patients free of VOCs; follow-up up to 37.2 months.
Exa-cel primary endpoints amended: TDT target Hb ≥9 g/dL without transfusions; SCD target no severe VOCs.
Pediatric exa-cel studies CLIMB-141/151 now enrolling patients ages 5-11 with TDT or SCD.
CTX130 at DL3+: ORR 70% (30% CR) in PTCL/CTCL; 90% DCR; responses across all disease compartments.
CTX130 well tolerated: no DLTs, no GvHD, all CRS/ICANS grade 1-2; no treatment-related deaths.

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