Cellectis gene editing candidate .HEAL-101 reduced triglycerides in a mouse disease model, the company said on October 27, 2026, as it presented preclinical data for a potential treatment for severe hypertriglyceridemia.

Key Takeaways

  • The company reported a mean triglyceride reduction of 75% in a mouse disease model.
  • Cellectis plans an investigator-initiated Phase 1 trial in China.
  • Preliminary clinical data are expected in the second half of 2027.

Cellectis gene editing results in preclinical studies

In mice engineered to carry human APOC3 and model hypertriglyceridemia, .HEAL-101 reduced triglycerides by a mean of 75%, with a standard deviation of 11%. APOC3 fell by 67%, with a standard deviation of 12%.

In a separate mouse model with humanized livers and normal blood lipid levels, a single intravenous injection edited human APOC3 at a mean rate of approximately 65%, reaching as high as 70%. In a human liver cell line, the mean editing rate was approximately 90%.

The company said editing was detected in the liver, with no significant activity in the other tissues analyzed. These are preclinical findings; animal results may not translate to people.

How the candidate works and what comes next

.HEAL-101 uses a TALE base editor delivered as messenger RNA in liver-directed lipid nanoparticles. It is designed to permanently inactivate APOC3, a driver of elevated triglycerides, after one intravenous administration.

Cellectis plans to initiate a Phase 1 investigator-initiated trial in China and expects preliminary clinical data in the second half of 2027. The trial and its results remain prospective.

The expanded dataset builds on findings first disclosed on September 14, 2026. Julien Valton, Cellectis vice president of gene therapy, is scheduled to present the poster on October 29 at the European Society of Gene and Cell Therapy congress in Hamburg.

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