Editas Medicine (EDIT) Corporate presentation summary

Transformative gene editing strategy

• Utilizes CRISPR nuclease-based technology to upregulate protein expression by editing non-coding regulatory regions, enabling genotype-agnostic treatments for serious diseases.

• Functional upregulation approach increases disease-mitigating protein levels without altering the natural protein sequence.

• Differentiated strategy includes genetic knockdown, correction, and non-genetic knockdown, aiming for one-time, curative potential.

EDIT-401: Hyperlipidemia program highlights

• EDIT-401 targets hyperlipidemia with a single-dose therapy, achieving over 90% mean LDL-C reduction in non-human primates and mice.

• At least 6- to 8-fold mean increase in LDLR protein observed in preclinical studies, with no adverse clinical observations and transient, resolved liver enzyme elevations.

• Human genetics data from Icelandic families with partial LDLR 3' UTR deletions show significant LDL-C reduction and increased LDLR without adverse health impacts.

Unmet need and market opportunity

• Approximately 70 million US patients have hyperlipidemia, with 1.2 million affected by HeFH and 15 million with ASCVD; most do not meet LDL-C goals.

• Current standard of care is inadequate, with ~75% of ASCVD patients not achieving LDL-C targets and high discontinuation rates due to burdensome regimens.

• Healthcare expenditures for ASCVD are projected to exceed $300 billion by 2035.