Prime Medicine, Inc. · NASDAQ:PRME
Prime Medicine — Investor Presentation (October 2026)
44 slides from Prime Medicine's investor presentation, October 2026
Slide 1: Corporate Presentation
Delivering on the promise of Prime Editing Corporate Presentation October 2026 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: Prime Editing: Emerging as the Predominant Gene Editing Technology
Prime Editing: Emerging as the Predominant Gene Editing Technology Prime Medicine’s Gene editing Prime Editing is strong IP position permanently the most versatile covers any corrects genetic gene editing permutation of alterations technology Prime Editing Prime Editing New regulatory Clinical data
Slide 4: Prime Medicine is Strategically Delivering on the Promise of Prime Editing
Prime Medicine is Strategically Delivering on the Promise of Prime Editing Focused execution on Initial efforts Pursue Make PM359 Wilson disease underway to complementary available for and Alpha-1 expand liver partnerships to patients with Antitrypsin franchise expand reach CGD Deficiency DISCIPLINE
Slide 5: Near-Term, Focused Execution Positions Prime Medicine to Capitalize on Prime Editing
Near-Term, Focused Execution Positions Prime Medicine to Capitalize on the Full Potential of Prime Editing 2026 2027+ ✓PM577a CTA and IND cleared • Announce PM577a initial clinical data WILSON ✓Initiate clinical trial • Capitalize on platform modularity vis-a-vis additional DISEASE common mutations
Slide 6: Prime Medicine's Pipeline: Focused on Value Creating Opportunities
Prime Medicine’s Pipeline: Focused on Value Creating Opportunities Therapeutic Lead IND- Pivotal / Indication Delivery Discovery Phase 1/2 Area Optimization enabling Registration Wilson Disease LNP LIVER Alpha-1 Antitrypsin LNP Deficiency (AATD) Cystic Fibrosis 1 LUNG (including PASSIGE ) LNP Plan t
Slide 7: Leveraging the Versatility of Prime Editing to Address a Range of Diseases
We Plan to Leverage the Versatility of Prime Editing to Address a Range of Diseases Across Target Tissues Deletion / Insertion Correction p47 Chronic Granulomatous Disease (CGD) Point Mutation Correction Wilson Disease, Alpha-1 Antitrypsin Deficiency REPLACEMENT Hotspot Correction Cystic Fibrosis, R
Slide 8: Modular Prime Editing Platform Flywheel Effect
Modular Prime Editing Platform Flywheel Effect: Industrialized Engine is Reused Across Programs OPTIMIZE Computational Design Off-Target Analysis to Non-Clinical and Screening of Core Advance Highest Development Components Precision Guide RNAs In-vitro, in-vivo PE proteins, RNAs, lipids LEARN REUSE
Slide 9: Extensive, Foundational Intellectual Property for Prime Editing Technologies
Prime Medicine Holds Extensive, Foundational Intellectual Property for Prime Editing Technologies CRISPR-Cas Enzyme Any programmable nuclease to specifically identify and cut DNA sequences Any combination or permutation of a CRISPR-Cas Guide RNA enzyme, template Guide Engineered RNAs that locate the
Slide 10: Liver
Liver Delivering on the promise of Prime Editing
Slide 11: Prime Medicine's Initial Liver Franchise
Prime Medicine’s Initial Liver Franchise: Aspiring to Cure Two of the Largest Genetic Liver Diseases, Enabled by Platform Modularity PM577: Wilson Disease PM647: AATD Opportunity (Patients) Opportunity (Patients) >20,000 US and EU 200,000 US and EU 7,500-15,000+ Japan 20,000-30,000 Diagnosed Clinica
Slide 12: Proprietary LNP-Formulated Prime Editor Multi-Component Drug Product
Proprietary LNP-Formulated Prime Editor is a Complex Multi-Component Drug Product Designed to Support Current and Future Liver Programs LNP Modularity: Ionizable Nucleic acid encapsulation and endosomal escape Lipid Majority of components in the LNP are the same across liver programs Helper Stabiliz
Slide 13: Wilson Disease
Wilson Disease 13
Slide 14: Advancing Prime Editors for Wilson Disease: Disease Overview
Advancing Prime Editors for Wilson Disease: Disease Overview Large genetically defined disease well suited for Prime Editing Disease Severity and Opportunity Healthy Blood • Common liver and systemic disease presenting in teens to 20s • Leads to liver failure, neurocognitive decline and premature de
Slide 15: 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 Prime Addressable Prime Addressable geographic mutational Patients Patients distributio
Slide 16: Prime Editors Efficiently and Precisely Corrected Prevalent Wilson Disease 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 for PM577a Prime Editors d
Slide 17: PM577a Efficiently Corrects the H1069Q Mutation and Restores Copper Concentration
PM577a Efficiently Corrects the H1069Q Mutation and Restores Wild-Type Copper Concentration at Multiple Doses ~45% editing restored copper levels to near wild-type levels Precise correction of ATP7B Increase in wild-type ATP7B protein Liver copper restored to wild-type levels* p.H1069Q in mice expre
Slide 18: Prime Edited Mice Demonstrated Normal Copper Metabolism
Prime Edited Mice Challenged with Radiolabeled Copper Demonstrated Normal Copper Metabolism At Multiple Doses Untreated Untreated Treated (H1069Q Mut) (H1069Q Mut) (Wild Type) 0.4 mg/kg 0.8 mg/kg Copper challenge and PET imaging performed 4 weeks post PE treatment, PET imaging performed 24hrs post c
Slide 19: PM577a Clinical Trial Overview
PM577a Clinical Trial Overview Trial Overview Dose Escalation Dose Expansion Design H1069Q adult patients stable on SOC Phase 1/2, open-label Dose Cohort 4+ Population (if required) Adults and adolescents with Wilson Part 1 (Extension) Disease and at least one ATP7B Adults stable on SOC p.H1069Q all
Slide 20: Leveraging Platform Modularity for a Majority of 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 Follow-on formalize DC H1069Q (PM577a) programs to leverage Goal to incorporate into exi
Slide 21: Alpha-1 Antitrypsin Deficiency (AATD)
Alpha-1 Antitrypsin Deficiency (AATD) 21
Slide 22: Advancing Prime Editors for AATD: Disease Overview
Advancing Prime Editors for AATD: Disease Overview Disease Severity and Opportunity • AATD is an inherited genetic disorder that causes low levels of AAT protein • Low levels of AAT protein increases the risk of lung disease (emphysema) • Patients are also at risk of liver disease (cirrhosis) caused
Slide 23: AATD Program Objective: Normalize AAT Levels in PiZZ Patients
AATD Program Objective: Normalize AAT Levels in PiZZ Genotype Patients to Healthy Human Levels Serum A1AT Concentration 50 40 30 Normal Human Range (µM) 20 11uM is minimum 10 threshold for benefit 0 Genotype MM MS SS MZ SZ ZZ NULLNULL Target patient Healthy Human Low Risk population** • Restore SERP
Slide 24: PM647 Efficiently Corrected the Mutation In Vivo
PM647 Efficiently Corrected the Mutation In Vivo Resulting in M-AAT Protein Restoration at Clinically Relevant Doses Efficient correction Restoration M-AAT Protein** % Corrected AAT 100 % Mutant AAT % Human AAT Isoform* 75 50 25 0 Negative Dose Dose Positive Control Level #1 Level #2 Control M-AAT =
Slide 25: PM647 Clinical Trial Overview
PM647 Clinical Trial Overview Trial Overview Dose Escalation Dose Finding & Expansion Design Group A: adults with lung-only AATD (Pi*ZZ) Phase 1/2, open-label Cohort 4+ Population (if required) Adults with PiZZ AATD: lung-only Group A (Extension) (Group A) or lung + liver / liver-only Lung-only AATD
Slide 26: Lung
Lung Delivering on the promise of Prime Editing
Slide 27: Advancing Prime Editors for Cystic Fibrosis
Advancing Prime Editors for Cystic Fibrosis (CF), a Disease for Which There is No Curative Therapy Prime Medicine’s efforts in Cystic Fibrosis funded through multiple grants from the Cystic Fibrosis Foundation Disease Severity and Opportunity Healthy • Progressive, genetic disease that affects the l
Slide 28: Progress Developing Prime Editors for G542X Mutated Cystic Fibrosis
Prime Medicine has Made Significant Progress Developing Prime Editors for G542X Mutated Cystic Fibrosis Phenotypic restoration of G542X mutated CFTR in G542X In vitro Prime Editor in Primary HBEs differentiated ALI culture CFTR C band B band -actin Prime Healthy Unedited Edited Patient Efforts towa
Slide 29: Parallel Prime Editing Approaches to CF: Hotspot and PASSIGE
Parallel Prime Editing Approaches to CF: Hotspot and PASSIGE Hotspot PASSIGE Eight hotspot Prime Editors could address the “high unmet Potential to address nearly all CF patients with a single need” mutations; these same Prime Editors could address super exon insertion strategy >93% of all CF patien
Slide 30: Immunology and Oncology
Immunology and Oncology Delivering on the promise of Prime Editing
Slide 31: Chronic Granulomatous Disease (CGD)
Chronic Granulomatous Disease (CGD) 31
Slide 32: Advancing Prime Editor for CGD: Disease Overview
Advancing Prime Editor for CGD: Disease Overview Rare genetic disease, characterized by defective neutrophil function Healthy Disease Severity and Opportunity gp91phox (NOX2) Extracellular Space • Serious life-threatening disease presents in childhood; life expectancy approximately 40 years Membrane
Slide 33: Breakthrough Clinical Data with PM359 in CGD
Breakthrough Clinical Data with PM359 in CGD Provide Proof-of-Concept for Prime Editing as the Foundation for a New Class of Curative Therapies Observed rapid engraftment, restored DHR positivity and improvement in inflammatory markers, with acceptable safety Successful manufacturing from single m
Slide 34: No Detectable Off-Target Editing, Large Deletions, or Translocations
Prime Medicine Has Not Observed Any Detectable Off-Target Editing, Large Deletions, or Translocations in Any Lead Program Prime Medicine uses CRISPR-Cas9 editors as a positive control in off-target analyses Examples from CGD program used to support IND/CTA filings No off-target editing detected in N
Slide 35: Ex-Vivo CAR-T
Ex-Vivo CAR-T 35
Slide 36: CAR-T: PASSIGE and Multiplex Prime Editing and BMS Collaboration
CAR-T: PASSIGE and Multiplex Prime Editing is the Foundation of Prime Medicine’s Collaboration with BMS Platform modularity has potential to accelerate development of additional CAR-T Programs Existing Limitations Prime Editing Solution ꭗ Low payload integration efficiency ✓ >80% integration efficie
Slide 37: Strategic License and Collaboration Agreement with Bristol Myers Squibb
Strategic License and Broad Collaboration Agreement with Bristol Myers Squibb (BMS) to Develop Prime Edited ex Vivo CAR-T Products First broad, multi-target collaboration advancing Prime Editors for the treatment of complex oncology and autoimmune indications • $110 million upfront • >$3.5 billion i
Slide 38: Simultaneously and Efficiently Multiplex Edit CAR-T Cells
Beyond Precisely Inserting a Chimeric Antigen Receptor, We Can Simultaneously and Efficiently Multiplex Edit CAR-T Cells Prime Editors can be multiplexed to introduce multiple genomic modifications in CAR-T cells High efficiency multiplex editing of B2M and TRAC Maintained efficiency with five multi
Slide 39: Corporate
Corporate Delivering on the promise of Prime Editing
Slide 40: Our Strategy to Maximize the Broad Therapeutic Potential of Prime Editing
Our Strategy to Maximize the Broad Therapeutic Potential of Prime Editing Within Our Core Outside Our Core Enabling Innovation Partner at the right time to Form strategic partnerships to access expertise and expand into Access best-in-class enabling fund, advance and therapeutic areas beyond our tec
Slide 41: Prime Medicine is Leading the Next Generation of Gene Editing
Prime Medicine is Leading the Next Generation of Gene Editing Pipeline Wilson disease clinical trial underway; AATD IND cleared; initial clinical data from both programs in 2027 Positioned Focused on programs in large genetic diseases, with clear path to value and multi billion -dollar opportuni
Slide 42: Appendix
Appendix Delivering on the promise of Prime Editing
Slide 43: Prime Editing is Designed with a Wide Range of Genome Editing Capabilities
Prime Editing is Designed with a Wide Range of Genome Editing Capabilities Flexibility to select right approach for each indication based on editing need Prime Editing Small edits Mid-sized edits Large deletions Large insertions or (e.g., all 12 bp swaps, 1-bp to 20- (e.g., hotspot corrections, del
Slide 44: PASSIGE Technology Enables Insertion of Gene Sized Sequences
PASSIGE Technology Enables Prime Editing to Insert Gene Sized Sequences Precisely, Potentially Addressing Large Markets PASSIGE: Prime-Assisted Site-Specific Integrase Gene Editing: One step non-viral multi-kilobase-size gene editing approach with no double-stranded breaks Where we are working today
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