Editas Medicine (EDIT) Barclays 28th Annual Global Healthcare Conference summary

Platform and differentiation

• Focuses on in vivo CRISPR therapeutics delivered via IV infusion using lipid nanoparticles, targeting edits in non-coding DNA to upregulate disease-mitigating proteins.

• Differentiates by using CRISPR for edits not achievable by other modalities, such as upregulating LDL receptor for cholesterol reduction.

• Collaborates with Genevant for liver delivery and has proprietary LNP technology for extrahepatic tissues.

• Emphasizes cost and capital efficiency in execution.

Safety and durability

• CRISPR editing offers high specificity, aided by machine learning to minimize off-target effects; robust off-target safety data presented to regulators.

• Lipid nanoparticle delivery avoids hepatotoxicity risks seen with AAV vectors and enables edits that are inherited by daughter cells, supporting long-term durability.

• Preclinical studies show only transient, mild transaminase elevations with no significant safety concerns.

• Mouse data demonstrate durable LDL-C lowering effects for at least three months.

EDIT-401 program and clinical plans

• EDIT-401 targets LDL receptor upregulation, achieving 90% mean LDL cholesterol reduction in multiple animal models, including those mimicking familial hypercholesterolemia.

• The approach is based on human genetic variants that naturally upregulate LDLR, with evidence from an Icelandic family showing significant LDL-C reduction and good health.

• Consistent efficacy observed regardless of baseline LDL-C; safety profile in animals is favorable.

• Phase 1 human proof-of-concept study is planned by year-end, with rapid LDL-C response expected and initial dosing in high-risk HeFH patients.

• Broader addressable population includes up to 10 million high-risk hyperlipidemia patients in the US.