Editas Medicine (EDIT) Evercore ISI 8th Annual HealthCONx Conference summary

Strategic focus and program overview

• Pivoted to focus on LDLR upregulation using CRISPR, aiming for high-potency, low-cost therapies that could transform standard of care.

• Achieved a 6x increase in LDL receptor in non-human primates, resulting in a 90% reduction in LDL cholesterol, a level not seen with other modalities.

• Approach leverages natural human variants to identify and de-risk targets for safety and efficacy.

• The edit stabilizes LDLR mRNA, increasing protein levels without altering the receptor itself.

Mechanism of action and differentiation

• Directly increases LDL receptor levels, breaking through the efficacy ceiling of statins and PCSK9 inhibitors.

• Can potentially achieve cholesterol levels below 50 mg/dL with a single injection, regardless of baseline.

• Targets a broad population with hypercholesterolemia, with initial focus on heterozygous familial hypercholesterolemia (HeFH).

Patient populations and clinical development

• U.S. population with elevated cholesterol is about 70 million; HeFH represents about 1 million, with refractory cases as initial focus.

• Expansion planned into secondary and primary high-risk populations as safety is established.

• Human proof-of-concept targeted for end of 2026, with initial trials in HeFH and potential for rapid expansion.