Cellectis Exits CAR-T and Reinvents Itself as an In Vivo Gene Editing Company

Sep 14 , 2026
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Sep 14, 2026 —

Cellectis is leaving cell therapy behind and remaking itself around in vivo gene editing. The company is discontinuing its two allogeneic CAR-T candidates, lasme-cel and eti-cel, citing a materially changed landscape for blood cancers as frontline regimens improve and bispecific antibodies and in vivo CAR-T crowd the later-line settings.

In their place, Cellectis is advancing two liver-directed programs built on its TALEN base-editing technology, formulated in lipid nanoparticles and given intravenously. The approach edits without cutting both strands of DNA, avoiding the double-strand breaks associated with conventional CRISPR nucleases.

HEAL-101 targets APOC3 for severe hypertriglyceridemia and reduced triglycerides by about 76% in mouse models, with first Phase 1 data expected in the second half of 2027. HEAL-201 targets the PCSK9 promoter for severe hypercholesterolemia and lowered plasma PCSK9 by more than 90% in mice, with Phase 1 data expected in the first half of 2028.

Source:

https://www.biopharmadive.com/news/cellectis-restructure-in-vivo-gene-editing-allogeneic-heart-disease/830281/

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