Editas Medicine (EDIT) investor relations material

Strategic positioning and technology differentiation

• Completed pivot to a fully in vivo CRISPR editing focus, prioritizing efficient capital use and rapid human value generation.

• Differentiates by upregulating therapeutic proteins using CRISPR, leveraging large human genetic datasets to de-risk targets.

• In vivo approach chosen for lower cost, scalability, and ease of use compared to cell therapies, enabling global accessibility and better margins.

• Identified over five liver-based opportunities for in vivo upregulation, with further tissue exploration planned.

Lead program and clinical development

• Selected EDIT-401, an LDLR-targeting program, as lead asset, showing 90% LDL cholesterol reduction in preclinical models.

• Preclinical efficacy demonstrated across multiple animal models, including those mimicking human familial hypercholesterolemia.

• Human proof of concept targeted by 2026, with cash runway extending into Q2 2027.

• Development milestones include GMP manufacturing, dose range finding, and off-target editing package completion, aiming for IND/CTA submission in 2026.

• Additional nonclinical data to be shared at upcoming scientific meetings.

Commercialization and financial outlook

• EDIT-401 positioned as a one-time, durable treatment with potential for significant healthcare savings and value creation.

• Pricing strategy aims for pharmaceutical margins, leveraging scalable off-the-shelf manufacturing and lower cost of goods.

• Early commercial focus on refractory hypercholesterolemia patients, with potential expansion and partnerships for broader impact.

• Payers show interest in the transformative potential and cost-effectiveness of the therapy.

• No need for new manufacturing infrastructure due to established LNP and RNA supply chains.