Cellectis quits CAR-T and pivots to ‘in vivo’ gene editing

An article from Dive Brief Cellectis quits CAR-T and pivots to ‘in vivo’ gene editing Long a frontrunner in donor-derived cell therapy research, the French biotech cited a shifting landscape in changing course and choosing to offload its lead programs. Published Sept. 14, 2026 Jonathan Gardner Senior Reporter Share Copy link Email LinkedIn X/Twitter Facebook Print License Add us on Google Dive Brief: Cellectis is giving up on donor-derived cell therapies and shifting its focus to gene editing medicines, stating in a Monday announcement that the “commercial and clinical landscape” for combatting blood cancers with so-called CAR-T treatments has “changed materially.” The decision dramatically lengthens Cellectis’ road to a marketed medicine, as the company is ending work on treatments that were in clinical development and turning to therapies that haven’t been tested in humans.

One CAR-T program it discovered and out-licensed has advanced as far as Phase 2 testing. Cellectis claimed that its new strategy represents the “most effective use of its financial and operational resources.” Investors expressed disappointment, however, as company shares fell as much as 43% in morning trading and, at around $2 apiece, trade just above all-time lows. Dive Insight: Founded around the turn of the century with gene editing technology developed by Institut Pasteur, Cellectis has long been a frontrunner in advancing “allogeneic” therapies built from the cells of donors.

It raised $228 million in an initial public offering in 2015 and, since then, has worked to prove its treatments could become a simpler alternative to the complicated, personalized CAR-T therapies now available for multiple blood cancers. Yet like many of its peers, Cellectis has struggled. Individualized, “autologous” cell therapies have set a high bar for winning approvals in malignancies like leukemia, lymphoma and multiple myeloma.

Their allogeneic counterparts have had difficulty matching those results, leading many developers in recent years to shift resources to other areas, such as autoimmune disease research. As recently as early August, Cellectis was touting data for two clinical-stage cell therapies called lasme-cel and eti-cel in leukemia and lymphoma. But treatment protocols in many blood cancers have changed drastically as new types of medicines — from cell therapies to bispecific antibodies and more — have been approved.

Those developments have heightened Cellectis’ competition, reduced the addressable patient populations for each therapy and slowed study enrollment. “We believe these trends are likely to continue and further constrain the commercial opportunity for both product candidates,” the company said in a statement. Cellectis will seek “strategic partnering opportunities” to maximize the value of lasme-cel and eti-cel.

The company will also “realign its organization” and support existing partnerships with Allogene, Servier, AstraZeneca and Iovance Biotherapeutics, the company said. The new drugs it’ll work on, meanwhile, are prospective cardiovascular disease treatments that are still in preclinical development. One, called HEAL-101, uses base editing technology to target a gene, APOC3, linked to high triglycerides.

Another known as HEAL-201 harnesses an epigenetic editing approach to lower levels of the cholesterol-regulating protein PCSK9. Cellectis will face an uphill battle in both cases, as multiple medications aimed at APOC3 and PCSK9 are already available. Cellectis’ strategic shift comes in the wake of multiple recent high-profile failures in cardiovascular disease studies.

The latest setback happened just over a week ago, when a therapy from Novartis and Ionis Pharmaceuticals fell short in a major study that had widely been expected to succeed. Add us on Google Share Copy link Email LinkedIn X/Twitter Facebook Print License Filed Under: Gene Therapy