Cellectis: its gene editor reduces triglycerides by 75% in mice
At the annual congress of the European Society of Gene and Cell Therapy (ESGCT), held in Hamburg from October 27-30, 2026, Cellectis unveiled an expanded dataset on its in vivo base editing candidate targeting the APOC3 gene. In mice, a single administration reduced triglycerides by 75% on average, and the group indicated it plans to initiate a Phase 1 trial in China.
Up to 70% editing of the human APOC3 gene in animals
.HEAL-101 is an APOC3-specific TALE base editor, administered as messenger RNA in lipid nanoparticles targeting the liver. It is designed to permanently inactivate APOC3, which Cellectis presents as a genetically validated factor in triglyceride elevation, following a single intravenous injection. The candidate targets severe hypertriglyceridemia. These results extend an initial dataset published by the group on September 14, 2026. In a human hepatic cell line, the average APOC3 editing rate reached approximately 90%, without double-strand breaks in DNA at the targeted site. In a normolipidemic mouse with a humanized liver, a single injection edited the human APOC3 gene at an average rate of approximately 65%, with a maximum of 70%. In a transgenic mouse model of hypertriglyceridemia, .HEAL-101 reduced APOC3 by 67% ± 12% and triglycerides by 75% ± 11% (mean ± standard deviation). Editing was detected in the liver, with no significant activity in other tissues analyzed.
Phase 1 trial planned in China
According to Julien Valton, Vice President of Gene Therapy at Cellectis, these data "strengthen the potential" of the TALE base editing approach to achieve efficient and specific APOC3 editing following a single administration. He believes that the reduction in triglycerides observed supports the development of .HEAL-101 as a potential one-time treatment for patients with severe hypertriglyceridemia. Cellectis plans to launch an investigator-initiated Phase 1 trial in China, with initial clinical data expected in the second half of 2027. The group itself recalls, in its forward-looking statements disclaimer, that data from animal models may not translate into the clinic, and that acceptance by international regulatory authorities of data generated in a trial conducted in China constitutes an uncertainty. The poster (number P0208) will be presented on October 29, 2026, from 2 p.m. to 4 p.m. (Paris time), and is already available on the company's website.
