Editas Medicine (EDIT) Strategy Update summary

Strategic objectives and progress

• Achieved pre-clinical in vivo proof of concept for sickle cell disease and beta thalassemia using a proprietary targeted lipid nanoparticle (tLNP) for gene editing in humanized mouse models, reaching 29% editing efficiency and 20% HbF induction after a single dose.

• Reni-cel (EDIT-301) continues to show strong clinical data, with 28 adult patients dosed in the RUBY trial and adolescent dosing scheduled, aiming for BLA and commercialization.

• Presenting substantive clinical data for sickle cell patients in the RUBY study by mid- and year-end 2024.

• Actively pursuing global partnership or out-licensing for reni-cel to focus resources on in vivo pipeline development and commercialization.

• Collaboration with Genevant announced for LNP technology targeting non-HSC tissues, expanding delivery capabilities.

In vivo gene editing platform and pipeline

• In vivo strategy centers on functional upregulation of gene expression using indel CRISPR technology, focusing on HSCs and other tissues.

• Lead discovery leverages proprietary guide RNA modifications, AsCas12a enzyme, mRNA, and programmable tLNP delivery.

• Pre-clinical data show competitive editing and functional outcomes, supporting potential for first- and best-in-class in vivo therapies for rare and orphan diseases.

• Proprietary LNP platform enables delivery to extrahepatic tissues and outperforms current public data.

• Ongoing optimization of tLNP and cargo, with plans to advance to non-human primate studies.

Financial and business development update

• Secured over $80 million in non-dilutive financing in the past year, including a $57 million agreement with DRI Healthcare Trust, extending cash runway into 2026.

• Ended Q3 with $265 million in cash, increasing to $320 million after DRI payment.

• Focused on capital-efficient pipeline development, IP monetization, and anti-dilutive financing, with recent deals including collaborations with Vertex, Vor Bio, and Genevant.

• Reni-cel development and commercialization costs are high; partnering is preferred to avoid significant dilution and ensure broad patient access.

• In vivo medicines expected to offer larger market opportunities with lower cost of goods and clinical trial expenses compared to ex vivo therapies.