Cellectis Shifts Focus to In Vivo Gene Editing

Cellectis, a pioneer in gene editing, has announced a strategic transformation to focus on in vivo gene editing, marking a significant shift in its focus from its previous cell therapy programs. The company aims to advance two lead in vivo programs, .HEAL-101 and .HEAL-201, which target severe dyslipidemias. .HEAL-101, designed to treat severe hypertriglyceridemia, and .HEAL-201, targeting severe hypercholesterolemia, are expected to provide long-lasting treatments for chronic diseases.

The .HEAL-101 program, which targets the human APOC3 gene, has shown promising results in preclinical studies. In a liver humanized normolipidemic murine model, intravenous injection of .HEAL-101 successfully edited the human APOC3 gene, leading to a significant reduction in plasmatic APOC3 levels and circulating triglycerides. The company plans to initiate a Phase 1 investigator-initiated trial (IIT) in China and share preliminary data in the second half of 2027.

Similarly, .HEAL-201, targeting the human PCSK9 gene, demonstrated high on-site epigenome editing activity in preclinical studies. Intravenous injection of PCSK9 TALEM mRNA LNPs in a liver humanized murine model resulted in a significant decrease in plasmatic PCSK9 levels and triglyceride levels. The company intends to share preliminary clinical data from this program in the first half of 2028.

The strategic realignment comes after a thorough assessment of the evolving therapeutic landscape. Despite the company's continued belief in the potential of allogeneic CAR T-cell therapies, the commercial and clinical landscape for B-ALL and NHL has changed significantly. Advances in frontline treatment regimens and the emergence of bispecific antibodies and in vivo CAR-T approaches have reduced the addressable patient population for lasme-cel and eti-cel, leading to slower enrollment and a potentially longer development pathway. As a result, Cellectis has decided to exit the development of these programs while seeking strategic partnering opportunities to maximize their value.

To support its new strategic direction, Cellectis will realign its organization and resources to focus on its in vivo gene editing pipeline and existing cell therapy partnerships with AstraZeneca, Allogene, Servier, and Iovance. These actions are designed to extend the company's cash runway into the second half of 2028, providing financial flexibility to advance its in vivo gene editing programs through key development milestones.

Cellectis' Chief Executive Officer, André Choulika, commented, "Gene surgery has the potential to transform the treatment of high-risk metabolic diseases by delivering long-lasting benefits through a single IV injection. Our decision to focus Cellectis on in vivo gene editing reflects the progress we have made with .HEAL-101 and .HEAL-201 and our assessment of where our gene editing capabilities can be most effectively deployed."

The company will host a conference call and webcast on September 14, 2026, to discuss the strategic transformation and provide more details on the .HEAL-101 and .HEAL-201 programs. The call will be available in both English and French, with webcast links provided for participants.