Philippe Duchateau, PhD, Chief Scientific Officer at Cellectis, describes the company’s TALEN gene editing technology and its application in hematologic cancers.
TALEN is based on a class of proteins derived from transcription activator-like effectors (TALEs), which are highly specific DNA-binding proteins. TALEs can be engineered to bind to almost any DNA sequence and, when combined with a nuclease, can edit the DNA at specific locations.
As Dr. Duchateau explains, Cellectis most prominent use of TALEN is through engineered allogeneic CAR T-cells, which the company calls UCARTs, each of which target a specific cancer type. Three of these products are being investigated in phase 1 clinical trials. In these trials, the safety and efficacy of UCARTs are being evaluated in patients with acute myeloid leukemia (AML; NCT03190278), relapsed and refractory B-cell acute lymphoblastic leukemia (ALL; NCT04150497), and refractory/relapsed multiple myeloma (NCT04142619). Additionally, the use of UCARTs in the treatment of solid tumors is under investigation.
Preliminary clinical data on the use of the product, UCART22, in relapsed and refractory B-cell ALL patients were recently presented at The American Society of Hematology Meeting & Exposition (ASH 2021).
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