Cellectis (ALCLS) investor relations material

Strategic focus and technology platform

• Transitioned to prioritize in vivo gene editing, leveraging a differentiated TALE-based platform for high specificity and versatility in gene targeting.

• Two lead preclinical candidates, .HEAL-101 (APOC3) and .HEAL-201 (PCSK9), target severe hypertriglyceridemia and hypercholesterolemia, respectively, with single IV injections designed for durable effects.

• TALE base editors and epigenetic modulators enable precise gene modification or silencing without DNA breaks, minimizing off-target effects and genotoxicity.

• Partnerships with major industry players remain unchanged, and internal resources are now focused on these two priorities.

• Cash runway extends into the second half of 2028, supporting ongoing development and global clinical plans.

Preclinical and clinical development highlights

• .HEAL-101 demonstrated up to 76% reduction in triglycerides in humanized mouse models by introducing a stop codon in APOC3, effectively disabling the protein.

• .HEAL-201 uses epigenetic silencing to shut down PCSK9 expression, resulting in durable LDL-C lowering in preclinical humanized liver models.

• Both programs are advancing to investigator-initiated trials in China, with .HEAL-101 starting in Q1 2027 and .HEAL-201 following four months later.

• First-in-human data for .HEAL-101 expected in H2 2027, with .HEAL-201 readouts anticipated in H1 2028; durability of response will be a key focus.

• Parallel IND and CTA activities are ongoing to enable seamless transition to global phase I-b/II studies targeting high-risk patient populations.

Market positioning and unmet needs

• Severe hypertriglyceridemia and hypercholesterolemia represent large, underserved populations, with 1–2 million patients in the US and EU for each indication.

• Existing therapies require lifelong redosing and carry significant costs; gene editing offers a potential one-time, durable solution.

• Market research indicates strong clinician and patient interest in a "gene surgery" approach, especially for younger, high-risk patients.

• PCSK9 and APOC3 are well-validated targets, and the approach is positioned as potentially best-in-class if efficacy and durability are confirmed.

• No changes to ongoing cell therapy partnerships; focus is now on gene editing for cardiometabolic diseases.