Editas Medicine (EDIT) Wells Fargo 21st Annual Healthcare Conference summary

Company strategy and focus

• Focused on developing in vivo CRISPR-edited medicines targeting highly differentiated efficacy and exploiting unique CRISPR properties.

• Prioritizes diseases with accessible biomarkers for rapid proof of concept in humans.

• Shifted away from cell-based therapies to control costs and maximize public health impact.

• Lead asset EDIT-401 targets cardiovascular disease by reducing LDL cholesterol, Lp(a), and ApoB by up to 90% in non-human primates.

• Clinical entry for EDIT-401 is planned this year, with top-line data expected next year.

EDIT-401 program and scientific rationale

• EDIT-401 aims to address high-risk cardiovascular patients unable to reach LDL targets despite current therapies.

• Uses CRISPR to upregulate LDL receptor expression in liver cells, based on human gain-of-function variants.

• Achieved a six-fold increase in LDLR expression and unprecedented LDL cholesterol reduction in preclinical models.

• Editing strategy mimics natural beneficial mutations, with optimized guide RNAs yielding greater potency.

• Significant LDL reduction achieved with editing as few as 15% of alleles, mainly monoallelic edits.

Clinical development and trial design

• Phase I trial to begin in Australia, targeting heterozygous familial hypercholesterolemia patients.

• Dose escalation design (3+3) with at least four cohorts; part two will expand patient numbers.

• Confident in dose translation from non-human primates to humans, expecting similar efficacy regardless of baseline LDL.

• Safety data expected in Q1 2027, with top-line part one data later in 2027.

• U.S. trial entry anticipated in 2027, pending manufacturing documentation.