Cellectis Presents Preclinical Data for .HEAL-101 Triglyceride Reduction

NEW YORK, Oct. 27, 2026 /CourierPR/ -- Cellectis, a genome engineering company, has presented preclinical data at the Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) in Hamburg, Germany, highlighting the potential of its in vivo base editing product candidate, .HEAL-101, for treating severe hypertriglyceridemia (sHTG). The congress, held from October 27 to 30, 2026, featured Cellectis' presentation of expanded preclinical data supporting .HEAL-101, which was first disclosed on September 14, 2026.

The company's .HEAL-101, designed to permanently inactivate the APOC3 gene after a single intravenous administration, achieved a 70% in vivo editing rate in a normolipidemic humanized liver mouse model. This figure was presented as part of a broader set of preclinical data that Cellectis first disclosed on September 14, 2026. In a hypertriglyceridemic mouse model, .HEAL-101 demonstrated a significant reduction in triglyceride levels, with a 76% decrease observed after a single dose. The data also showed that the editing was liver-restricted, with no significant activity detected in other tissues.

Key findings presented at ESGCT include: - Efficient and highly specific editing of APOC3 in vitro: In a human hepatic cell line, .HEAL-101 achieved a mean APOC3 editing rate of approximately 90%, without production of DNA double strand break at the target site. - Efficient editing of human APOC3 in vivo: In a normolipidemic humanized liver mouse model, a single intravenous injection of LNP-mRNA edited the human APOC3 gene at a mean rate of approximately 65%, reaching up to 70%. - Marked efficacy in a disease model: In a hypertriglyceridemic human APOC3 transgenic mouse model of hypertriglyceridemia, .HEAL-101 reduced APOC3 by 67% ± 12%, and triglycerides by 75% ± 11% (mean ± standard deviation). - Liver-restricted activity: Editing was detected in the liver, with no significant activity in the other tissues analyzed.

These preclinical results support the continued development of .HEAL-101. Cellectis plans to initiate a Phase 1 investigator-initiated trial (IIT) in China in the second half of 2027, with the aim of generating preliminary clinical data for the .HEAL-101 program. CET. It is available on Cellectis' website under the poster number P0208.

The press release also notes that forward-looking statements in the document are subject to various risks and uncertainties, including the potential for the preclinical data not to translate into further preclinical development or clinical results.