Prime Medicine, Inc. · NASDAQ:PRME
Prime Medicine — Investor Presentation (September 2025)
37 slides from Prime Medicine's investor presentation, Sep 8, 2025
Slide 1: Delivering on the promise of Prime Editing
1 Delivering on the promise of Prime Editing September 2025 Corporate Presentation
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: Advancing Prime Editing to change the course of how diseases are treated
3 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.
Slide 4: Clinical proof of concept and poised for long-term value creation
4 Platform Validation Clinical Validation and Focused Execution Platform Discovery Sustained Growth and Commercial Success We Believe Prime Medicine has Demonstrated Clinical Proof of Concept and is Poised for Long-Term Value Creation 2020 - 2023 2024 - 2026 Breakthrough clinical data from first-in-
Slide 5: Entering a new era of gene editing: 2025, 2026, 2027+ milestones
5AATD = Alpha-1 Antitrypsin Deficiency; CF = cystic fibrosis; IND = investigational new drug; CTA = clinical trial application; CGD = chronic granulomatous disease Prime Medicine is Entering a New Era of Gene Editing: Generating Clinical Data for Multiple Programs, Leveraging Platform Modularity Sec
Slide 6: Prime Editing can edit, correct, insert and delete DNA sequences
6 We Believe Prime Editing is the Only Gene Editing Technology That Can Edit, Correct, Insert and Delete DNA Sequences in Any Target Tissue REPLACEMENT Prime Editor Deletion / Insertion Correction p47 Chronic Granulomatous Disease (CGD) Point Mutation Correction Wilson’s Disease, AATD Targeted Full
Slide 7: Clinical data in CGD with PM359
7 Successful manufacturing from single mobilizations Tolerable myeloablative conditioning at fully therapeutic exposures with expected adverse events (transitory mucositis, pancytopenia) No serious adverse events attributed to PM359 Rapid neutrophil and platelet engraftment confirmed within
Slide 8: Prime Editing has highly differentiated safety profile: no off-target activity
8 No detectable double strand breakage No detectable off-target deletions, chromosomal translocations or rearrangements No detectable off-target edits No detectable bystander edits 1Analysis of edited CD34+ cells from CGD program: Targeted in vitro Analysis of 550 potential off-target sites of off-t
Slide 9: Platform modularity de-risks efforts and accelerates program advancement
9 Prime Editing Platform Modularity Potentially De-Risks Ongoing Efforts and Accelerates Program Advancement Prime Editors Manufacturing Delivery Off-Target Assays Nonclinical Regulatory Clinical
Slide 10: Pipeline focused on value creating opportunities
10 Modular Platform Indication Delivery Discovery Lead optimization IND- enabling Phase 1/2 LIVER Wilson’s 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 Pipeline Focused on Value C
Slide 11: Liver
11| Liver
Slide 12: Liver franchise: Wilson's Disease and AATD
12 Prime Medicine’s Liver Franchise: Aspiring to Cure Two of the Largest Genetic Liver Diseases, Enabled by Platform Modularity Current Status IND-enabling activities ongoing Final stages of lead optimization Rapidly Advancing IND and/or CTA on track for H1’26 IND and/or CTA anticipated in mid-2026
Slide 13: Proprietary LNP-formulated Prime Editor components
13LNP = lipid nanoparticle; PEG = Polyethylene glycol; GalNAc = N-acetylgalactosamine; PE = Prime Editor; pegRNA = Prime Editing guide RNA; ngRNA = nicking guide RNA *May require slight modifications to mRNA depending on disease context Proprietary LNP–formulated Prime Editor is a Complex Multi-Comp
Slide 14: Modular LNP dosed in non-human primate with minimal safety concerns
14 Prime Medicine’s Modular LNP Dosed as a Single Administration in Cynomolgus Monkey (NHP) With Minimal, If Any, Safety Concerns LNP = lipid nanoparticle; LFT = Liver Function Tests; H&E = hematoxylin and eosin • Well-tolerated: no acute reactions, clinical observations, or body weight changes • Tr
Slide 15: Wilson's Disease
15Confidential | Wilson’s Disease
Slide 16: Wilson's Disease overview
16 Wilson’s Disease Advancing Prime Editors for Wilson’s 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 • Com
Slide 17: Prime Editors efficiently corrected the two most prevalent Wilson's mutations
17 Prime Editors delivered with Prime Medicine’s universal liver LNP administered at clinically relevant doses Prime Editors Efficiently Corrected the Two Most Prevalent Disease-Causing Mutations in Wilson’s Disease Editing efficiency in fully humanized homozygous p.H1069Q and R778L ATP7B mouse mode
Slide 18: H1069Q correction reduced liver copper and restored homeostasis
18IND = investigational new drug; CTA = clinical trial application * % Normalized to saline treated Prime has initiated IND-enabling activities planning for a H1’26 IND and/or CTA application(s) Efficient Correction of the H1069Q Mutation Reduced Liver Copper and Restored Copper Homeostasis In Vivo
Slide 19: Alpha-1 Antitrypsin Deficiency (AATD)
19Confidential | Alpha-1 Antitrypsin Deficiency (AATD)
Slide 20: Advancing Prime Editors for AATD: disease overview
20 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 21: AAT protein restoration targets
21 • Correcting circulating AAT protein to ≈20uM or more restored to healthy levels and predicted to prevent disease progression • 11uM is the current minimum level accepted by Regulators for replacement therapies • Decreased Z-AAT in the liver will potentially ameliorate the liver manifestations of
Slide 22: PM647 corrected mutation and restored M-AAT protein in vivo
22M-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 Prime has initiated IND-enabling activities planning for Mid’26 IND and/or CTA application(s)
Slide 23: Lung
23| Lung
Slide 24: Advancing Prime Editors for Cystic Fibrosis
24 We believe Prime Editing-based approaches could eventually benefit more than 93% of all people with CF CFTR = cystic fibrosis transmembrane conductance regulator Advancing Prime Editors for Cystic Fibrosis (CF), a Disease for Which There is No Curative Therapy Large genetically defined disease we
Slide 25: Parallel Prime Editing approaches to CF: Hotspot and PASSIGE
25CF = 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 In 2024, Prime Medicine entered into agreement with CF Foundation for up to
Slide 26: Progress developing Prime Editors for G542X mutated CF
26 Efforts towards 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 suite of assays in development to enable selection of development candidate and advance to IND enabling studies • Hum
Slide 27: Immunology and Oncology
Immunology and Oncology Delivering on the promise of Prime Editing [22
Slide 28: Strategic license and collaboration with Bristol Myers Squibb
28 • $110 million upfront • >$3.5 billion in potential milestones, including: ‒ $185 million in preclinical milestones ‒ $1.2 billion in development milestones ‒ More than $2.1 billion in commercial milestones ‒ Royalties on net sales • Multiple targets in immunological diseases and cancer, beyond t
Slide 29: CAR-T: PASSIGE and multiplex Prime Editing
29TRAC = 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 30: Multiplex editing of CAR-T cells
30 Maintained efficiency with five multiplex edits B2M = b2 macroglobulin; TRAC = T-cell receptor alpha constant 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 mod
Slide 31: Corporate
Corporate Delivering on the promise of Prime Editing | 31
Slide 32: Business development will continue to play a critical role
32 Prime Medicine remains active in sell-side business development, with the goal of accelerating our pipeline, bolstering our financial resources Beyond BMS, Business Development Will Continue to Play a Critical Role in Building Prime Medicine Within Our Core Outside Our Core Enabling Innovation Wi
Slide 33: Extensive, foundational intellectual property for Prime Editing
33 Prime Medicine Holds Extensive, Foundational Intellectual Property for Prime Editing Technologies • Multiple configurations of RNA-templated gene editing - Prime Editor protein configurations: fusion, separate and split configurations - pegRNA configurations: fusion, split, separate and engineere
Slide 34: Leader in gene editing positioned to create sustainable value
34 Prime Medicine is the Leader in Gene Editing Positioned to Create Sustainable Value Through Pipeline Execution and External Partnerships The Leader in Prime Editing Potential to address approximately 90% of genetic diseases and opportunities in non-genetic diseases Breakthrough initial clinic
Slide 35: Appendix
Appendix Delivering on the promise of Prime Editing | 35
Slide 36: Prime Editing wide range of genome editing capabilities
36BP = 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 37: PASSIGE technology enables insertion of gene-sized sequences
37*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
Prime Medicine — Investor Presentation (September 2025) on 6ix