Editas Medicine (EDIT) Strategy Update summary

Strategic transition to in vivo gene editing

• Announced a full pivot to focus exclusively on in vivo gene editing, ending ex vivo programs and development of reni-cel after failing to secure a commercial partner.

• Achieved in vivo proof of concept with ~40% HBG1/2 promoter editing and 20% fetal hemoglobin induction in a humanized mouse model for sickle cell disease and beta thalassemia.

• Demonstrated high-efficiency in vivo liver editing in non-human primates through a collaboration, expanding the platform's reach.

• Strategic shift includes a 65% workforce reduction to optimize costs and extend cash runway into Q2 2027.

• Plans to provide additional preclinical data and program timelines in Q1 2025.

Financial and operational guidance

• Cash runway extended into Q2 2027, supported by $320 million pro forma cash at Q3 and reduced operating expenses.

• Manufacturing scale-up for in vivo programs is included in current financial guidance.

• Cost optimization measures include significant headcount reduction and resource reallocation to discovery and early development.

Scientific and competitive positioning

• In vivo approach leverages clinically validated gamma-globin upregulation, considered more potent for anti-sickling than wild-type beta-globin restoration.

• Ongoing optimization of LNP delivery and guide RNA potency to further improve editing efficiency and HbF induction.

• Differentiation from competitors is based on validated targets and delivery technology, with confidence in plug-and-play platform potential.