Prime Medicine, Inc. · NASDAQ:PRME
Prime Medicine — Technical Presentation (November 2025)
27 slides from Prime Medicine's technical presentation, Nov 12, 2025
Slide 1: Restoring Copper Homeostasis: Prime Medicine's Path to Developing Transformative Therapies for Wilson Disease
Delivering on the promise of Prime Editing Restoring Copper Homeostasis: Prime Medicine’s Path to Developing Transformative Therapies for Wilson Disease November 12, 2025 1
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation contains forward-looking statements of Prime Medicine, Inc. ("Prime", "we" or "our") within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These forward-looking statements contain information about our current and future
Slide 3: Today's Agenda
Today’s Agenda S P EAKERS SETTING THE STAGE ‒ Welcome and Introduction Allan Reine, M.D. Chief Executive Officer ‒ Why Prime Editing for Wilson Disease WILSON DISEASE OVERVIEW ‒ Global Market Opportunity Michael Schilsky, M.D. ‒ Wilson Disease Treatment Landscape and the Professor of Medicine, Yale
Slide 4: Key Takeaways from Today's Event
Key Takeaways from Today’s Event Wilson Disease is an area of tremendous unmet need: no curative therapies 1 available; physicians and patients dissatisfied with current standards of care. Prime Editing has the potential to provide a durable cure, by precisely and 2 permanently correcting the causat
Slide 5: We are advancing Prime Editing to change the course of how diseases are treated
We are advancing Prime Editing to change the course of how diseases are treated. We aim to provide safe, effective and curative treatments, which offer lifelong benefit to patients. 5
Slide 6: Prime Editing is the Only Technology That Can Edit, Correct, Insert and Delete DNA
We Believe Prime Editing is the Only Technology That Can Edit, Correct, Insert and Delete DNA Sequences in Any Target Tissue Deletion / Insertion Correction p47 Chronic Granulomatous Disease (CGD) Point Mutation Correction Prime Editing is designed with a wide Wilson Disease, Alpha-1 range of genome
Slide 7: We Are Focused on Value Creating Opportunities
We Are Focused on Value Creating Opportunities: Substantial Need, Clear Biology, Potential for Meaningful Commercial Impact Modular Lead IND- Phase Indication Delivery Discovery Platform optimization enabling 1/2 Wilson Disease LNP LIVER Alpha-1 Antitrypsin Deficiency LNP (AATD) Cystic Fibrosis 1 LU
Slide 8: Our Initial Efforts Are Focused on Two of the Largest Genetic Liver Diseases
Our Initial Efforts Are Focused on Two of the Largest Genetic Liver Diseases Wilson Disease Alpha-1 Antitrypsin Deficiency CURRENT STATUS IND-enabling studies ongoing IND-enabling studies ongoing UPCOMING IND and/or CTA on track for 1H 2026 IND and/or CTA on track for mid-2026 MILESTONES Clinical da
Slide 9: Franchise of Programs for Genetic Liver Diseases Sharing Proprietary Universal LNP
We are Advancing a Franchise of Programs for Genetic Liver Diseases, Which Share Our Proprietary, Universal LNP Vast majority of components can be used across liver programs, allowing potential benefit of shared toxicology, manufacturing and regulatory experience Accelerates, derisks and reduces cos
Slide 10: Wilson Disease Programs Have Potential to Address Multi-Billion Dollar Market
Prime Medicine’s Wilson Disease Programs Have Potential to Address Multi-Billion Dollar Market Six most common mutations account for up to 26,000 patients in addressable markets (US, Europe, Japan) with unique geographic Prime Addressable Patients Prime Addressable Patients mutational distribution
Slide 11: Leverage Platform Modularity to Rapidly Advance Prime Editors for Wilson Disease Patients
We Plan to Leverage Platform Modularity to Rapidly Advance Prime Editors for a Majority of Wilson Disease Patients R778L Large commercial opportunity in Japan >90% editing efficiency, minimal preclinical work to formalize DC H1069Q (PM577) Follow-on Goal to incorporate into existing regulatory filin
Slide 12: A Clinician's Perspective on Wilson Disease
A Clinician’s Perspective on Wilson Disease Michael Schilsky, M.D. Professor of Medicine, Yale University Confidential | 12
Slide 13: Wilson Disease: A Large, Genetically Defined Disease With No Curative Options
Wilson Disease: A Large, Genetically Defined Disease With No Curative Options Autosomal-recessive disorder due to mutations in the ATP7B gene, which encodes a copper -transporting ATPase Mutations in ATP7B impair copper excretion into bile and its incorporation into ceruloplasmin, leading to patholo
Slide 14: Phenotype at Presentation of Wilson Disease
Phenotype at Presentation of Wilson Disease From Ott et al Designing clinical trials in Wilson disease. Hepatology 2021 Michael L Schilsky MD FAASLD 14
Slide 15: Treatment Goal Depends on Phase of Disease
Treatment Goal Depends on Phase of Disease PREVENTION TREATMENT RESCUE Neuropsychiatric symptoms Hepatic Inflammation End-stage Liver Failure Cirrhosis Time Death Asymptomatic elevation of Nonspecific Complications of portal hypertension serum aminotransferases symptoms • Ascites of liver disease •
Slide 16: Current Standard-of-Care: High Rates of Non-Adherence, Side Effects
Current Standard-of-Care: High Rates of Non-Adherence, Side Effects Require multiple daily doses with large pill burden under fasting conditions; therapies bring adverse side effects that can f orce discontinuation or exacerbate other disease symptoms Penicillamine Trientine Zinc Liver Transplant (C
Slide 17: Prime Medicine's Approach to Wilson Disease
Prime Medicine’s Approach to Wilson Disease Confidential | 17
Slide 18: Prime Editing Has the Potential to Change the Treatment Paradigm in Wilson Disease
Prime Editing Has the Potential to Change the Treatment Paradigm in Wilson Disease TODAY OUR VISION Chronic treatment burden: standard- One time therapy that of-care agents and low copper diets precisely and permanently are burdensome and often hard to tolerate; long-term compliance is restores wild
Slide 19: Preclinical Data Strongly Support Potential for PM577 as Transformative Therapy
Preclinical Data Strongly Support Potential for PM577 as Transformative Therapy DELIVERY Optimized Prime Editors achieve high levels of hepatocyte editing Favorable safety profile in mice and NHPs, with no evidence of off-target editing or SAFETY meaningful LFT elevations at clinically relevant dose
Slide 20: Prime Editors Efficiently and Precisely Corrected the Two Most Prevalent Mutations
Prime Editors Efficiently and Precisely Corrected the Two Most Prevalent Disease-Causing Mutations in Wilson Disease Efficient correction of the H1069Q and R778L No detectable off-target editing identified in mutations in fully humanized mouse models patient-derived cells Prime Editors delivered wit
Slide 21: Prime Editors Correct H1069Q and Restore Wild-Type Copper Concentration In Vivo
Prime Editors Efficiently Correct the H1069Q Mutation and Completely Restore Wild-Type Copper Concentration In Vivo Using optimized Prime Editor in partially humanized homozygous p.H1069Q ATP7B mouse model >80% hepatocytes Hepatic copper concentration* returns to Copper** excreted normally through t
Slide 22: Prime Edited Mice Demonstrated Normal Copper Clearance 24h Post Injection
Prime Edited Mice Challenged with Radiolabeled Copper Demonstrated Normal Copper Clearance 24h Post Injection Wild Type Control Untreated (H1069Q Mut) Treated (H1069Q Mut) 22 *Copper challenge and PET imaging performed 4 weeks post PE treatment
Slide 23: PM577 Clinical Development: On Track for H1'26 IND and/or CTA
PM577 Clinical Development: On Track for H1’26 IND and/or CTA with Proof-of-Concept Data Anticipated in 2027 ANTICIPATED ENROLLMENT CRITERIA: • Adult patients who are maintained on standard of care (chelators, zinc salts) Ultimate goal of the Phase 1/2 PRIMARY SAFETY ENDPOINTS: study is to demonstra
Slide 24: Prime Medicine is Entering a New Era of Gene Editing
Prime Medicine is Entering a New Era of Gene Editing: Generating Clinical Data for Multiple Programs, Leveraging Platform Modularity 2025 2026 2027+ PM577 for Wilson Disease: PM577 for Wilson Disease: PM577 for Wilson Disease: Prepare for 2026 clinical entry, advance File IND and/or CTA in 1H 2026;
Slide 25: Q&A
Q&A Delivering on the promise of Prime Editing
Slide 26: Prime Medicine is the Leader in Gene Editing Positioned to Create Sustainable Value
Prime Medicine is the Leader in Gene Editing Positioned to Create Sustainable Value Through Pipeline Execution and External Partnerships Potential to address approximately 90% of genetic diseases and opportunities in non-genetic diseases The Leader in Breakthrough initial clinical data in CGD de
Slide 27: Thank You!
Thank You! Delivering on the promise of Prime Editing
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