Prime Medicine (PRME) investor relations material

Technology differentiation and platform strategy

• Prime Editing is described as the most versatile and safest gene editing technology, enabling precise DNA edits without unwanted off-target effects or bystander edits.

• The platform allows for large, specific DNA insertions and correction of mutations not addressable by CRISPR or base editing.

• Universal LNP technology uses a novel ionizable lipid, showing favorable preclinical safety and potential for higher dosing and potency.

• The company is open to broadening access through business partnerships and collaborations.

Wilson's disease program

• Targeting transversion mutations in Wilson's disease, which are not addressable by other gene editing technologies.

• IND/CTA filing planned for the first half of next year, with clinical data expected in 2027.

• Biomarkers include copper PET scans, fecal and urinary copper, and seroplasmin to assess copper homeostasis and guide withdrawal from standard of care.

• Pre-study underway to genotype and screen patients, aiming to expedite enrollment and cover 30%-50% of the U.S. patient population initially.

• Initial clinical focus on patients with compensated liver disease, with plans to expand to decompensated cases after safety is established.

Alpha-1 antitrypsin deficiency (AATD) program

• Prime Editing aims to restore wild-type protein, potentially offering superior function compared to base editing approaches.

• Preclinical data show normalization of alpha-1 levels in serum and high editing efficiency.

• Clinical entry planned for mid-next year, with data readout in 2027; study design will follow established approaches in the field.

• Early studies will focus on AAT levels and percentage correction, with a goal of rapid patient enrollment and dose escalation.