Cellectis ends development of two cancer cell therapy programs and shifts resources to gene editing inside the body

Cellectis ends development of two cancer cell therapy programs and shifts resources to gene editing inside the body

On September 14, Cellectis announced that its board of directors had approved a strategic shift on September 11. The company will end its own development of two CAR-T programs. This therapy engineers T-cells to recognize tumors; these programs used donor cells. The resources freed up will support two preclinical programs that edit genes inside the body, with initial preliminary data expected in 2027 and 2028.

Before this shift, Cellectis was independently developing lasme-cel and eti-cel. In its September 14 press release, the company says it will continue seeking partners for both programs. It attributes the decision to improvements in earlier lines of treatment, which have reduced the number of patients needing later-line therapy and slowed trial enrollment. Competition is also increasing from bispecific antibodies, drugs that bind two biological targets, and CAR-T therapies generated inside the patient's body. Cellectis will maintain its existing cell therapy partnerships.

The two new programs deliver mRNA, a temporary set of genetic instructions for the editing system, to the liver in lipid nanoparticles, tiny fatty shells. .HEAL-101 targets the APOC3 gene to treat severe hypertriglyceridemia, or very high blood triglyceride levels. In a mouse model with human liver cells, Cellectis reports approximately 55% editing at the target site and an approximately 45% reduction in triglycerides. In a separate model of severe hypertriglyceridemia, it reports an approximately 76% reduction in triglycerides.

.HEAL-201 is intended to treat severe hypercholesterolemia, or very high cholesterol levels. Its editing system targets the PCSK9 promoter, a DNA region that regulates gene activity. In a mouse model with human liver cells, Cellectis reports a reduction in plasma PCSK9 levels of more than 90%.

The company plans exploratory trials in China, with preliminary data for .HEAL-101 expected in the second half of 2027 and for .HEAL-201 in the first half of 2028. Cellectis estimates that the reorganization will extend its cash runway into the second half of 2028.