Cellectis reports 90% gene editing in preclinical study
Key points
- Cellectis (NASDAQ:CLLS) reported that its .HEAL-101 candidate achieved approximately 90% mean APOC3 editing in human liver cells.
- In a separate mouse disease model, the company reported a 67% reduction in APOC3 and a 76% reduction in triglycerides after a single dose.
- Cellectis plans a Phase 1 investigator-initiated trial in China, with preliminary clinical data expected in the second half of 2027.
Cellectis (NASDAQ:CLLS) presented preclinical data for HEAL-101, a gene-editing candidate for severe hypertriglyceridemia, reporting approximately 90% mean editing in human liver cells.
The candidate targets APOC3, a gene involved in regulating triglyceride levels, and the company reported no DNA double-strand breaks at the target site in the human liver cell experiments.
In mice with humanized livers and normal lipid levels, a single intravenous injection produced approximately 65% mean human APOC3 editing, reaching up to 70%.
In a separate mouse disease model, APOC3 levels fell by 67% ± 12%, while triglyceride levels decreased by 76% following a single dose.
The company reported that editing was detected in the liver, with no significant activity in other tissues analyzed, and plans to evaluate the candidate in a Phase 1 investigator-initiated trial in China.
The results form part of Cellectis' broader strategy to develop in vivo gene-editing treatments, with the company targeting preliminary clinical data for HEAL-101 in the second half of 2027.
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