Prime Medicine (PRME) Jefferies Global Healthcare Conference 2026 summary

Technology and platform overview

• Prime Editing is described as a highly versatile and safest gene editing technology, capable of a broad range of genome edits, including large insertions and complex mutations not addressable by other technologies.

• The platform is positioned to address high unmet needs in genetic diseases, with ongoing efforts to improve editing efficiency and therapeutic index.

Wilson's disease program

• IND/CTA filing for PM577 in Wilson's disease is on track for the first half of the year, with the next update expected upon application acceptance.

• The initial phase I/II study will be global, with strong interest from KOLs and patients, aiming to expedite enrollment and development.

• Over 30 U.S. patients have been pre-screened to fast-track enrollment, focusing on the H1069Q mutation.

• Sites are expected to open and initial patients may be dosed by year-end, with no patient-by-patient updates planned.

• Preclinical data suggest that 40-45% hepatocyte editing may normalize copper metabolism, and clinical dosing will aim for biologically active levels.

• Biomarkers such as urinary and fecal copper, ceruloplasmin, and radiolabeled copper PET will be used to assess efficacy, with regulatory engagement ongoing.

• Safety measures include limited prophylactic drugs and preclinical benchmarking indicating a potentially wide therapeutic index for moderate liver disease patients.

• Initial clinical data are expected in 2027, with preference for reporting more comprehensive data sets.

Alpha-1 antitrypsin deficiency (AATD) program

• IND/CTA filing is planned for mid-year, with initial data targeted for 2027; guidance remains unchanged.

• Phase I/II will likely enroll lung and liver cohorts separately, focusing on biomarker endpoints such as AAT for accelerated approval.

• Dose escalation is expected to start near the lower efficacious preclinical dose, pending regulatory agreement.

• No major preclinical data updates are planned before 2026.

• Arbitration with Beam regarding AATD is expected to resolve by end of July.

• The program leverages synonymous editing to improve efficiency, with the collaboration agreement distinguishing between transition and non-transition edits.