BioCentury
ARTICLE | Product Development

Dec. 7 ASH Quick Takes: CRISPR, Vertex update durability for edited cell therapy; plus J&J- Legend, Kura, Fate, Pfizer, Autolus and Affimed

December 8, 2020 2:40 AM UTC

Vertex, CRISPR update long-term data for edited cell therapy
CRISPR Therapeutics AG (NASDAQ:CRSP) and Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) showed seven transfusion-dependent β thalassemia patients in the CLIMB-Thal-111 study remain transfusion-free for up to 18 months following CTX001 infusion; and three sickle cell disease patients in the CLIMB-SCD-121 study remain free of vaso-occlusive crises for up to 15 months after receiving the ex vivo CRISPR-edited cell therapy. The data presented at the virtual  American Society of Hematology update what were in an abstract released ahead of the conference: transfusion independence for up to 15 months in five thalassemia patients, and no vaso-occlusive crises for up to a year in two SCD patients.

97% ORR in MM for Legend, J&J BCMA CAR T
Johnson & Johnson (NYSE:JNJ) and  Legend Biotech Corp. (NASDAQ:LEGN) reported that ciltacabtagene autoleucel, an anti-BCMA CAR T cell therapy, led to a 97% overall response rate in the Phase Ib/II CARTITUDE-1 study for relapsed or refractory multiple myeloma patients at a median of 12.4 months. The readout improves on the 95% ORR revealed last month in the abstract, which was among the standouts in BioCentury’s review of BCMA data to be presented at ASH...