Editas Medicine (EDIT) investor relations material

Strategic focus and innovation

• Focused on developing therapeutics that surpass current standards of care using a unique CRISPR editing approach to upregulate beneficial proteins for disease mitigation.

• CRISPR editing is formulated into lipid nanoparticles, enabling scalable, off-the-shelf, single-infusion therapies with low cost of goods.

• EDIT-401 is positioned as a transformative therapy for hyperlipidemia, targeting unprecedented reductions in LDL cholesterol and Lp(a).

Clinical need and patient populations

• Atherosclerotic cardiovascular disease remains the leading cause of morbidity and mortality globally, with LDL-C and Lp(a) as key risk factors.

• Recent guidelines push for even lower cholesterol targets, leaving millions unable to reach goals despite current therapies.

• High-risk groups include patients with familial hypercholesterolemia and those with established cardiovascular disease who remain at risk despite standard care.

Preclinical data and therapeutic potential

• EDIT-401 achieved a mean 90% reduction in LDL cholesterol and Lp(a) in non-human primates, with effects durable through six months.

• Reductions in LDL cholesterol and Lp(a) are highly correlated and rapid, with a six-fold increase in LDLR protein expression in the liver.

• The approach is based on human genetic data identifying a naturally occurring gain-of-function LDLR variant associated with low cholesterol and good tolerance.