Prime Medicine, Inc. · NASDAQ:PRME
Prime Medicine — Investor Presentation (January 2026)
41 slides from Prime Medicine's investor presentation, Jan 12, 2026
Slide 1: Delivering on the promise of Prime Editing
1 Delivering on the promise of Prime Editing Corporate Presentation January 2026
Slide 2: Forward-looking statements
2 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 prospects and our operati
Slide 3: Prime Editing: Emerging as the Predominant Gene Editing Technology
3Delivering on the promise of Prime Editing Prime Editing: Emerging as the Predominant Gene Editing Technology Gene editing permanently corrects genetic alterations Prime Editing is the most versatile gene editing technology Clinical data with Prime Editors support curative potential Prime Editing d
Slide 4: Prime Medicine is Strategically Delivering on the Promise of Prime Editing
4 Flawless execution on Wilson and Alpha-1 Antitrypsin Deficiency Initial efforts underway to expand liver franchise Prime Medicine is Strategically Delivering on the Promise of Prime Editing DISCIPLINED CLINICAL DEVELOPMENT, MAXIMIZE PLATFORM IMPACT AND ENSURE ACCESS TO BREAKTHROUGH THERAPIES Pursu
Slide 5: Near-Term, Focused Execution Positions Prime Medicine
5 Secure multiple additional strategic partnerships to accelerate our pipeline and bolster our financial resources Near-Term, Focused Execution Positions Prime Medicine to Capitalize on the Full Potential of Prime Editing 2026 2027+ WILSON DISEASE • File PM577 IND and/or CTA in 1H • Initiate Phase 1
Slide 6: Pipeline by therapeutic area and indication
6 Therapeutic Area Indication Delivery Discovery Lead optimization IND- enabling Phase 1/2 LIVER Wilson Disease LNP Alpha-1 Antitrypsin Deficiency (AATD) LNP LUNG Cystic Fibrosis1 (including PASSIGE ) LNP/AAV IMMUNOLOGY & ONCOLOGY Ex vivo CAR-T2 (with PASSIGE ) ex vivo Multi-target collaboration adv
Slide 7: Leveraging the Versatility of Prime Editing Across Diseases and Tissues
7 We Plan to Leverage the Versatility of Prime Editing to Address a Range of Diseases Across Target Tissues REPLACEMENT Deletion / Insertion Correction p47 Chronic Granulomatous Disease (CGD) Point Mutation Correction Wilson Disease, Alpha-1 Antitrypsin Deficiency Targeted Full Gene Insertion (PASSI
Slide 8: PM359 CGD clinical data and regulatory interactions
8 Prime Medicine is continuing regulatory interactions based on current data set Successful manufacturing from single mobilizations Tolerable myeloablative conditioning at fully therapeutic exposures with expected adverse events (transitory mucositis, pancytopenia) No serious adverse events att
Slide 9: No Detectable Off-Target Editing, Large Deletions, or Translocations
91Analysis of edited CD34+ cells from CGD program: Targeted in vitro Analysis of 550 potential off-target sites of off-target editing. 2Data from in vivo analysis from mouse bone marrow harvested 16 weeks after engraftment was complete. 3Cas9 nuclease-edited cells, generated by transfecting HEK293T
Slide 10: Modular Prime Editing Platform Flywheel Effect
10 Manufacturing Processes and Assays Regulatory Frameworks Clinical Development Consolidated safety and platform clinical trials Computational Design and Screening of Core Components PE proteins, RNAs, lipids Off-Target Analysis to Advance Highest Precision Guide RNAs Non-Clinical Development In-vi
Slide 11: Extensive, Foundational Intellectual Property for Prime Editing
11 Prime Medicine Holds Extensive, Foundational Intellectual Property for Prime Editing Technologies CRISPR-Cas Enzyme Any programmable nuclease to specifically identify and cut DNA sequences Guide RNA Engineered RNAs that locate the exact editing site and provide the template for transcription Reve
Slide 12: Liver
Liver Delivering on the promise of | 22 Prime Editing
Slide 13: Prime Medicine's Initial Liver Franchise: Wilson Disease and AATD
13 PM647: AATD Prime Medicine’s Initial Liver Franchise: Aspiring to Cure Two of the Largest Genetic Liver Diseases, Enabled by Platform Modularity WD = Wilson Disease; AATD = Alpha-1 Antitrypsin Deficiency; IND = investigational new drug; CTA = clinical trial application PM577: Wilson Disease >20,0
Slide 14: Proprietary LNP-Formulated Prime Editor Drug Product
14 Proprietary LNP-Formulated Prime Editor is a Complex Multi-Component Drug Product Designed to Support Current and Future Liver Programs Control particle size and stability, stealth coating reduces serum interactions and increases half-lifePEG Lipids Stabilize and improve LNP pharmacokinetics, fac
Slide 15: Wilson Disease
15Delivering on the promise of Prime Editing Wilson Disease
Slide 16: Advancing Prime Editors for Wilson Disease: Disease Overview
16 Wilson Disease Advancing Prime Editors for Wilson Disease: Disease Overview Large genetically defined disease well suited for Prime Editing ceruloplasmin Blood Bile (fecal excretion) ATP7B ATP7B Copper CTR1 ceruloplasmin Blood (urinary excretion) Bile ATP7B ATP7B Copper CTR1 X X Healthy • Common
Slide 17: Wilson Disease Programs Address Multi-Billion Dollar Market
17 Prime Medicine’s Wilson Disease Programs Have Potential to Address Multi- Billion Dollar Market H1069Q R778L Other Common Mutations All Other Mutations Prime Addressable Patients Up to 7k Six most common mutations account for up to 26,000 patients in addressable markets (US, Europe, Japan) with
Slide 18: Efficient correction of H1069Q and R778L mutations in mouse models
18 Efficient correction of the H1069Q and R778L mutations in fully humanized mouse models Prime Editors Efficiently and Precisely Corrected the Two Most Prevalent Disease- Causing Mutations in Wilson Disease No detectable off-target editing identified in patient-derived cells for PM577 LNP = lipid n
Slide 19: PM577 Efficiently Corrects the H1069Q Mutation and Restores Copper Concentration
19 >80% hepatocytes edited Hepatic copper concentration returns to wild-type levels at eight weeks Copper excreted normally through the feces at four weeks PM577 Efficiently Corrects the H1069Q Mutation and Completely Restores Wild- Type Copper Concentration In Vivo
Slide 20: Prime Edited Mice Demonstrated Normal Copper Clearance
20*Copper challenge and PET imaging performed 4 weeks post PE treatment, PET imaging performed 24hrs post copper challenge Prime Edited Mice Challenged with Radiolabeled Copper Demonstrated Normal Copper Clearance Treated (H1069Q Mut)Wild Type Control Untreated (H1069Q Mut)
Slide 21: PM577 Clinical Development: On Track for 1H'26 IND and/or CTA
21IND = investigational new drug; CTA = clinical trial application PM577 Clinical Development: On Track for H1’26 IND and/or CTA with Proof-of- Concept Data Anticipated in 2027 Anticipated Enrollment Criteria Primary Safety Endpoints Primary Efficacy Endpoints Adult patients maintained on standard o
Slide 22: Leverage Platform Modularity to Advance Wilson Disease Prime Editors
22IND = investigational new drug; CTA = clinical trial application; LNP = lipid nanoparticle; DC = development candidate; PMDA = Japan’s Pharmaceuticals and Medical Devices Agency We Plan to Leverage Platform Modularity to Rapidly Advance Prime Editors for a Majority of Wilson Disease Patients Goal
Slide 23: Alpha-1 Antitrypsin Deficiency (AATD)
23Delivering on the promise of Prime Editing Alpha-1 Antitrypsin Deficiency (AATD)
Slide 24: Advancing Prime Editors for AATD: Disease Overview
24 Advancing Prime Editors for AATD: Disease Overview AATD = Alpha-1 Antitrypsin Deficiency; A1AT = alpha-1 antitrypsin • 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
Slide 25: AATD Program Objective: Normalize AAT Levels in PiZZ Patients
25 • Restore SERPINA1 gene to wild type, without bystander or other unwanted edits • Increase M-AAT levels minimally above protective threshold (~11 µM), ideally into healthy human range (>20 µM) • Maintain wild-type protein under endogenous control to protect patients during acute episodes (M-AAT l
Slide 26: PM647 Corrected the Mutation In Vivo Restoring M-AAT Protein
26M-AAT = healthy A1AT protein; Z-AAT = mutant A1AT protein; IND = investigational new drug; CTA = clinical trial application *Performed by LC-MS; **Analysis at day 7 positive control = healthy human serum PM647 Efficiently Corrected the Mutation In Vivo Resulting in M-AAT Protein Restoration at Cli
Slide 27: PM647 Clinical Development: On Track for Mid'26 IND and/or CTA
27IND = investigational new drug; CTA = clinical trial application PM647 Clinical Development: On Track for Mid’26 IND and/or CTA with Proof-of- Concept Data Anticipated in 2027 Anticipated Enrollment Criteria Primary Safety Endpoints Primary Efficacy Endpoints Group A: adult patients with pulmonary
Slide 28: Lung
Lung Delivering on the promise of Prime Editing | 28
Slide 29: Advancing Prime Editors for Cystic Fibrosis
29CFTR = cystic fibrosis transmembrane conductance regulator 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 • Progressive, genetic diseas
Slide 30: Progress Developing Prime Editors for G542X Mutated Cystic Fibrosis
30 Prime Medicine has Made Significant Progress Developing Prime Editors for G542X Mutated Cystic Fibrosis Efforts towards LNP and AAV in vivo delivery to humanized mice and large animals ongoing • We believe primary human lung progenitor data is most predictive of in vivo efficacy • Comprehensive s
Slide 31: Parallel Prime Editing Approaches to CF: Hotspot and PASSIGE
31CF = cystic fibrosis; attP = complimentary recognition sequence for attB; SA = splice acceptor; 3’ UTR = 3’ untranslated region; *Exons in gray introns in black Parallel Prime Editing Approaches to CF: Hotspot and PASSIGE Hotspot PASSIGE Eight hotspot Prime Editors could address the “high unmet ne
Slide 32: Immunology and Oncology
32| Immunology and Oncology
Slide 33: Strategic License and Collaboration Agreement with Bristol Myers Squibb
33 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 billio
Slide 34: CAR-T: PASSIGE and Multiplex Prime Editing Foundation of BMS Collaboration
34TRAC = T-cell receptor alpha constant; Data presented at ASH, December 2022, ASGCT, May 2023 and ASH, December 2023 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-
Slide 35: Simultaneously and Efficiently Multiplex Edit CAR-T Cells
35 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 Maintained efficiency with five multiplex editsHigh efficiency multiplex editing of
Slide 36: Corporate
36| Corporate
Slide 37: Our Strategy to Maximize the Broad Therapeutic Potential of Prime Editing
37 Our Strategy to Maximize the Broad Therapeutic Potential of Prime Editing Within Our Core Outside Our Core Enabling Innovation By pairing our internal resources with strategic partners and best-in-class enabling technologies, we aim to accelerate platform development and extend the reach of Prime
Slide 38: Prime Medicine is Leading the Next Generation of Gene Editing
38AATD = Alpha-1 Antitrypsin Deficiency; IND = investigational new drug; CTA = clinical trial application Prime Medicine is Leading the Next Generation of Gene Editing Preeminent Editing Technology Permanently corrects genetic alterations, without causing double-strand breaks or bystander edits
Slide 39: Appendix
Appendix the pro
Slide 40: Prime Editing is Designed with a Wide Range of Genome Editing Capabilities
40BP = base pair; KB = kilobase 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 Approach Small edits (e.g., all 12 bp swaps, 1-bp to 20- bp ins or del, combinations thereof) Mid-si
Slide 41: PASSIGE Technology Enables Prime Editing to Insert Gene Sized Sequences
41*Not part of Prime Medicine’s current pipeline 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
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