Cellectis (ALCLS) investor relations material

Strategic transformation and pipeline focus

• Announced a major pivot to focus exclusively on in vivo gene editing, leveraging expertise from ex vivo CAR T leadership and over 25 years in gene editing technologies to advance a simpler, scalable therapeutic approach.

• Two lead preclinical candidates, .HEAL-101 (APOC3 for severe hypertriglyceridemia) and .HEAL-201 (PCSK9 for severe hypercholesterolemia), will move into clinical development, targeting large populations with high unmet need.

• Exiting internal cell therapy programs (lasme-cel and eti-cel) to concentrate resources on in vivo gene editing, while seeking strategic partnerships and maintaining existing collaborations.

• Cash runway extended into the second half of 2028, supported by cost-saving initiatives, operational realignment, and organizational restructuring.

Lead program details and preclinical results

• .HEAL-101 targets APOC3 for severe hypertriglyceridemia, showing >70% base editing and significant reduction in APOC3 protein and triglycerides in preclinical models.

• .HEAL-201 targets PCSK9 for severe hypercholesterolemia, demonstrating >90% epigenome editing and deep reduction in PCSK9 transcript and protein levels in preclinical studies.

• Both programs use mRNA and lipid nanoparticle (LNP) delivery for a one-time, durable gene-editing effect, aiming for best-in-class efficacy and safety.

• Preclinical data show high specificity, durable gene silencing or editing, and strong efficacy in animal models, supporting clinical translation.

Clinical development and timelines

• .HEAL-101 will enter first-in-human trials in China in Q1 2027, with data readouts expected in H2 2027; .HEAL-201 will follow with first patients in Q2 2027 and data in H1 2028.

• Both programs plan Phase 1 investigator-initiated trials in China, with global expansion to follow.

• Accelerated IND and CTA preparations are ongoing for global phase I-B/II trials, with early regulatory engagement in the US and Europe.

• Phase 1 studies will focus on safety, tolerability, and dose optimization, with surrogate markers accepted for regulatory approval.