Editas Medicine (EDIT) Strategy Update summary

Strategic objectives and clinical progress

• Achieved preclinical in vivo proof of concept for sickle cell disease and beta thalassemia using a proprietary targeted lipid nanoparticle (tLNP) platform for gene editing in humanized mouse models, with 29% editing efficiency in HSPCs and 20% HbF induction in red blood cells after a single dose, outperforming public domain data.

• Advancing reni-cel (EDIT-301) toward BLA and commercialization, with ongoing enrollment in RUBY and EdiTHAL trials, 28 adult patients dosed, and adolescent dosing scheduled.

• Planning to present substantial clinical data for sickle cell patients in RUBY and EdiTHAL trials at the ASH Annual Meeting in December 2024 and by year-end 2024.

• The in vivo upregulation strategy targets rare and orphan diseases, with plans to expand to larger patient populations in the future.

• Initiated process to partner or out-license reni-cel, engaging Moelis & Company to lead the global process.

In vivo gene editing strategy and technology

• In vivo approach leverages clinically validated AsCas12a enzyme, proprietary guide RNA, mRNA, and tLNP delivery for functional gene upregulation, differentiating from knockdown strategies.

• Proprietary tLNP platform enables delivery to extrahepatic tissues and efficient gene editing in hematopoietic stem cells, with potential to address additional hematologic and non-hematologic disorders.

• Demonstrated ability to deliver gene editing cargo to HSCs and potential for delivery to other tissues.

• Optimization of tLNP and editing parameters continues, with plans to advance to non-human primate studies.

• Durability of in vivo editing is expected to match ex vivo approaches, with permanent genomic edits.

Financial and business development update

• Secured over $80 million in non-dilutive financing in the past nine months, including a $57 million deal with DRI Healthcare Trust for future Cas9 license payments, strengthening the balance sheet to $320 million after the DRI payment.

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

• Announced a collaboration with Genevant for LNP technology to target additional tissues beyond HSCs.

• Demonstrated ability to leverage foundational IP estate for non-dilutive capital and future licensing opportunities.

• Focusing on capital-efficient pipeline development, business development, and IP monetization.