Cellectis reports preclinical results for APOC3 gene editing candidate .HEAL-101
Cellectis presented data at the ESGCT 2026 congress showing 70% in vivo gene editing and a 76% reduction in triglyceride levels using its .HEAL-101 candidate in murine models.
Cellectis has released preclinical findings regarding .HEAL-101, an in vivo base editing therapy targeting severe hypertriglyceridemia. The data was presented during the European Society of Gene and Cell Therapy (ESGCT) 2026 annual congress in Hamburg, Germany.
.HEAL-101 utilizes a TALE-based editor delivered via mRNA within lipid nanoparticles, targeting the liver to permanently inactivate the APOC3 gene. Testing in murine disease models demonstrated that a single intravenous dose achieved 70% in vivo editing of the target gene, resulting in a 76% reduction in triglyceride levels.
This approach aims to address the clinical challenges of hypertriglyceridemia, which typically requires lifelong management. By providing a single-administration solution, the company seeks to offer an alternative to existing chronic therapies for patients with genetically validated elevated triglyceride drivers.