Cellectis presented expanded preclinical data on October 27 for .HEAL-101, an in vivo base editing candidate targeting APOC3 for severe hypertriglyceridemia, at the European Society of Gene and Cell Therapy congress in Hamburg. The candidate is a TALE base editor delivered as messenger RNA in liver directed lipid nanoparticles, designed to permanently inactivate APOC3 after a single intravenous dose.
In a human hepatic cell line the editor achieved a mean APOC3 editing rate of approximately 90 percent without producing a DNA double strand break at the target site. In a humanized liver mouse model, a single injection edited the human APOC3 gene at a mean rate of approximately 65 percent, reaching up to 70 percent. In a transgenic mouse disease model, APOC3 fell by 67 percent and triglycerides by about 75 to 76 percent. Editing was detected in the liver, with no significant activity in the other tissues analyzed.
The company plans an investigator initiated Phase 1 trial in China, with preliminary clinical data expected in the second half of 2027. The distance between this dataset and a medicine is the standard one for in vivo editing: mouse lipid biology, nanoparticle delivery at human scale, and durability all remain unproven in people. What the data establish is editing efficiency high enough, on the company’s own measures, to justify the human test.