Editas Medicine (EDIT) TD Cowen 46th Annual Health Care Conference summary

Company overview and strategic direction

• Focused on in vivo CRISPR gene editing, aiming for human proof of concept for EDIT-401 by end of year, 2026.

• Differentiated by targeting non-coding DNA to upregulate disease-mitigating proteins.

• Pivoted from ex vivo to in vivo editing for greater patient accessibility and lower costs.

• Funded into Q3 2027, with disciplined resource management.

• Key challenge is regulatory clarity in the CRISPR space, but recent FDA guidance is positive.

EDIT-401 program and scientific rationale

• EDIT-401 targets LDLR gene to increase LDL receptor protein, reducing LDL cholesterol by up to 90% in preclinical models.

• Editing strategy informed by human genetics, specifically an Icelandic family with beneficial LDLR variants.

• Non-human primate studies show rapid, durable, and well-tolerated LDL-C reduction.

• Mouse models confirm efficacy across various baseline LDL-C levels, including familial hypercholesterolemia genotypes.

• Human proof of concept data expected by year-end, with initial trials in heterozygous familial hypercholesterolemia patients.

Clinical development and market opportunity

• Initial trial aims to demonstrate safety (especially liver tolerability) and robust LDL-C reduction.

• Targeting patients refractory to current therapies, including those with familial hypercholesterolemia and established cardiovascular disease.

• European guidelines now recommend even lower LDL-C targets, increasing the addressable market.

• A 70%-90% LDL-C reduction is considered highly competitive and commercially attractive.

• Outpatient infusion and scalable LNP delivery offer payer and healthcare system advantages.