Cellectis scraps CAR-T pipeline in gene editing pivot
French biotech Cellectis is abandoning development of its two internal allogeneic CAR-T programs and repositioning itself as an in vivo gene editing company focused initially on cardiovascular disease.
Why it matters: The move is a dramatic reset for one of Europe’s pioneers of off-the-shelf CAR-T. As recently as January, Cellectis described itself as a late-stage allogeneic CAR-T company and expected pivotal Phase 2 data from its lead program this year. It will now replace clinical-stage oncology assets with two programs that have yet to enter human testing.
What changed: Cellectis is stopping development of lasme-cel in relapsed or refractory B-cell acute lymphoblastic leukemia and eti-cel in non-Hodgkin lymphoma, while looking for partners for both assets.
- The company said improving frontline treatments have reduced the number of patients reaching later treatment lines, while bispecific antibodies and emerging in vivo CAR-T therapies have increased competition.
- “Together, these dynamics have reduced the addressable patient population for lasme-cel and eti-cel, resulting in slower enrollment, a potentially longer and more costly development pathway, and therefore a delayed timeline to potential registration,” explained Cellectis in a press release.
- The reversal comes only about a month after Cellectis reiterated plans for a Phase 2 interim analysis of lasme-cel and full Phase 1 data for eti-cel in Q4.
Zoom in: The new strategy centers on two preclinical cardiovascular programs:
- HEAL-101 uses a lipid nanoparticle-delivered TALE base editor targeting APOC3, with the goal of treating severe hypertriglyceridemia. Cellectis plans a Phase 1 investigator-initiated trial in China, with preliminary data in H2 2027.
- HEAL-201 uses epigenetic editing to suppress PCSK9 in severe hypercholesterolemia. A Chinese Phase 1 trial is planned, with preliminary data expected in H1 2028.
- Both approaches are designed to alter gene activity without creating double-strand DNA breaks.
The bigger picture: Cellectis is joining a wider industry shift toward editing or engineering cells directly inside the body. Gilead’s Kite, AbbVie, Bristol Myers Squibb, AstraZeneca and Eli Lilly have all made major investments or acquisitions in the in vivo cell and gene therapy field.
- Cellectis is also entering a cardiovascular gene editing race that already has clinical validation. Lilly-owned Verve Therapeutics reported Phase 1 data this year showing its PCSK9 base editor VERVE-102 reduced LDL cholesterol by as much as 62% after a single infusion and plans to move into Phase 2.
The reset: Cellectis plans to realign its workforce and operations while maintaining cell therapy partnerships with AstraZeneca, Allogene, Servier and Iovance. The restructuring is expected to extend its cash runway from the previously projected Q4 2027 into H2 2028.
- Investors were not immediately convinced: Cellectis shares fell as much as 43% in Monday trading following the announcement.
Bottom line: Cellectis spent years trying to prove that donor-derived CAR-T could offer a simpler alternative to personalized cell therapy. Its decision to walk away from its own clinical programs shows how quickly that opportunity has narrowed and how strongly biotech investment is now moving toward therapies that perform the engineering directly inside the patient.
