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1、1Delivering on the promise of Prime EditingJanuary 2024JP Morgan Healthcare Conference2This 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 c
2、ontain information about our current and future prospects and our operations,which are based on currently available information.All statements other than statements of historical facts contained in this presentation,including statements regarding our strategy,projects and plans are forward-looking s
3、tatements.In some cases,you can identify forward-looking statements by terminology such as“aim,”“anticipate,”“assume,”“believe,”“contemplate,”“continue”“could,”“design,”“due,”“estimate,”“expect,”“goal,”“hope,”“intend,”“may,”“might,”“objective,”“opportunity,”“plan,”“predict,”“positioned,”“possible,”“
4、potential,”“project,”“seek,”“should,”“strategy,”“target,”“will,”“would”and other similar expressions that are predictions of or indicate future events and future trends,or the negative of these terms or other comparable terminology.These forward-looking statements include,but are not limited to,expr
5、ess or implied statements about Primes beliefs and expectations regarding:the initiation,timing,progress and results of our research and development programs,preclinical studies and future clinical trials,and the release of data related thereto;our ability to demonstrate,and the timing of,preclinica
6、l proof-of-concept in vivo for multiple programs;our ability to pursue our four strategic indication categories:immediate target indications,differentiation target indications,“blue sky”indications and“march up the chromosome”approaches;our ability to quickly leverage programs within our initial tar
7、get indications and to progress additional programs to further develop our pipeline;the potential of Prime Editors to reproducibly correct disease-causing genetic mutations across different tissues,organs and cell types,and the capacity of our PASSIGE technology to edit CAR-T cells for the treatment
8、 of certain cancers and immune diseases;the timing of our regulatory filings,including our anticipated initial IND submission for CGD as early as 2024 with additional filings anticipated in 2025;our ability to demonstrate superior off-target profiles for Prime Editing programs;the further advancemen
9、t of Prime Editors to maximize their versatility,precision and efficiency;the continued development and optimization of various non-viral and viral delivery systems,including our universal liver-targeted LNP delivery approach;the expansion of Prime Editings therapeutic potential to extend the reach
10、and impact of Prime Editing to areas beyond our current areas of focus;the potential of Prime Editing to offer curative genetic therapies for a wide spectrum of diseases;the scope of protection we are able to establish and maintain for intellectual property rights covering our Prime Editing technolo
11、gy;developments related to our competitors and our industry;our ability to leverage the clinical,regulatory,and manufacturing advancements made by gene therapy and gene editing programs to accelerate our clinical trials and approval of product candidates;the research collaboration with Cimeio to com
12、bine our and Cimeios respective technologies,including our Prime Editing platform and Cimeios SCIP platform,and the goals of such collaboration,the potential benefits of such collaboration and technology thereunder,including the ability to cure various diseases and replace existing treatments such a
13、s transplantation;the implementation of our strategic plans for our business,programs and technology,including our ability to identify and enter into future license agreements and collaborations;regulatory developments in the United States and foreign countries;our ability to attract and retain key
14、scientific and management personnel;and our estimates of our expenses,capital requirements,and needs for additional financing as well as our cash runway into 2024.Actual results or events could differ materially from the plans,intentions and expectations disclosed in the forward-looking statements w
15、e make due to a number of risks and uncertainties.These and other risks,uncertainties and important factors are described in the section entitled Risk Factors in our most recent Annual Report on Form 10-K,as well as any subsequent filings with the Securities and Exchange Commission.Any forward-looki
16、ng statements represent our views only as of the date of this presentation and we undertake no obligation to update or revise any forward-looking statements,whether as a result of new information,the occurrence of certain events or otherwise subject to any obligations under applicable law.We may not
17、 actually achieve the plans,intentions or expectations disclosed in our forward-looking statements,and you should not place undue reliance on our forward-looking statements.No representations or warranties(expressed or implied)are made about the accuracy of any such forward-looking statements.Certai
18、n information contained in this presentation relates to or is based on studies,publications,surveys and other data obtained from third-party sources and our own internal estimates and research.While we believe these third-party studies,publications,surveys and other data to be reliable as of the dat
19、e of this presentation,we have not independently verified,and make no representation as to the adequacy,fairness,accuracy or completeness of,any information obtained from third-party sources.In addition,no independent source has evaluated the reasonableness or accuracy of our internal estimates or r
20、esearch and no reliance should be made on any information or statements made in this presentation relating to or based on such internal estimates and research.Forward Looking Statements3“The age of human therapeutic gene editing isnt just coming.Its already here.”*David Liu,Ph.D.,Co-Founder of Prime
21、 Medicine4*David Liu,Ph.D.,Co-Founder of Prime Medicine“The age of human therapeutic gene editing isnt just coming.Its already here.”*5Now is our moment:Prime Medicine brings together the right people and the right technology at the right timewe are building on decades of progress to deliver the pro
22、mise of one-time,curative genetic therapies to address the widest spectrum of diseases6Delivering on the promise of Prime EditingPrime Medicine brings together the right people and the right technology at the right timeNow is our moment:we are building on decades of progress to deliver the promise o
23、f one-time,curative genetic therapies to address the widest spectrum of diseases OPERATIONAL EXECUTIONBROAD OPPORTUNITY TO ADDRESS LARGE MARKETSDIFFERENTIATED SAFETY PROFILEPLATFORM MODULARITYENTERING THE CLINICSTRATEGIC PIPELINE ALIGNED TO FOUR CORE PILLARS7Transferred technology from founding acad
24、emic lab and reproduced dataDeveloped initial strategic pipeline programsExecuted on key early hires to build out and scale operationsCompleted initial financing round20+Developed high throughput computational pegRNA and PE screening systemsEstablished universal assays for off-target ac
25、tivity and PE safetyBuilt delivery,CMC and platform innovation capabilitiesRobust and experienced management team in placeAccessed public markets via IPOFirst IND/CTA,with several more to follow,and first-in-human data on the horizonRobust infrastructure for CMC,Clinical and Regulatory executionPrep
26、aration for late-stage product development of lead programsPlatform modularity begins to enable rapid product cyclesPlatform GestationPlatform IndustrializationPlatform Translation:Entering the ClinicConsistent Operational Execution Sets Strong Foundation For Transition to Clinical-Stage Biotech Com
27、pany in 2024PE=Prime Editing;IPO=initial public offering;IND=investigational new drug;CTA=clinical trial application,CMC=chemistry,manufacturing and controls;pegRNA=Prime Editing guide RNA8Prime Editing is the only gene editing technology with the capability to edit,correct,insert and delete DNA seq
28、uences in any target tissuePrime Editings Versatility Can Unlock Broad Opportunity Across Wide Spectrum of Diseases REPLACEMENTDeletion/Insertion CorrectionChronic Granulomatous Disease(CGD)Point Mutation CorrectionWilsons DiseaseTargeted Gene Insertion(PASSIGE)CAR-TRepeat ExcisionFriedreichs Ataxia
29、Hotspot CorrectionRetinitis Pigmentosa/Rhodopsin(RHO adRP)Prime Editor9Recombinase enzymePASSIGE Technology Enables Prime Editing to Insert Gene Sized Sequences Precisely,Potentially Addressing Large MarketsPASSIGE:Prime-Assisted Site-Specific Integrase Gene Editing:One step non-viral multi-kilobase
30、-size gene editing approach with no double-stranded breaksCorrect inversion mutations(e.g.,Hemophilia A*)In vivo protein factory(e.g.,GLA enzyme for Fabrys disease*)Targeted whole gene replacement for rare liver diseases(e.g.,Phenylketonuria,*Tyrosinemia*)Targeted whole gene replacement for bone mar
31、row diseases(e.g.,Hereditary anemias,such as Fanconi Anemia)Non-viral,multiplex-edited CAR-T therapiesexpanding opportunity*Not part of Prime Medicines current pipeline101Analysis of edited CD34+cells from CGD program:Targeted in vitro Analysis of 550 potential off-target sites of off-target editing
32、.2Data from in vivo analysis from mouse bone marrow harvested 16 weeks after engraftment was complete.3Positive control generated by transfecting HEK293T with sgRNA targeting NCF1 and SpCas9 mRNA.HSC=hematopoietic stem cell;IND=investigational new drug;CTA=clinical trial applicationPrime Editing Has
33、 Highly Differentiated Safety Profile:No Off-Target Activity Detected in Any Lead Program*Prime Medicine uses a comprehensive suite of robust,IND-ready assays to evaluate Prime Editor safety risksExamples from CGD Program that are being used to support IND/CTA filings:No detectable off-target activi
34、ty in programs*for:Wilsons Disease CGD Glycogen Storage Disease 1b(GSD1b)RHONo double strand breakageNo detectable off-target editsNo detectable large deletions,chromosomal translocations or rearrangements No off-target editing detected in healthy human donor CD34+cells1Genomic locationTranslocation
35、 positive control:Cas9 nuclease-edited cells3No large deletions or translocations in bone marrow engrafted Prime-Edited LT-HSCs2On-target(NCF1)11Platform Modularity Accelerates and De-Risks Ongoing Efforts and Enables Rapid Generation of New Product Candidates Core components can be readily leverage
36、d to drive pipeline acceleration,efficiency and executionClinicalManufacturingDeliveryRegulatory Off-Target AssaysPrime Editors12Prime Medicine is Entering the Clinic at the Right Time:Evolving Landscape Favors Innovation in Cell and Gene Therapy Positive regulatory interactions in U.S.and globally
37、set stage for near-term clinic entryIn 2023,Prime Medicine:Engaged in multiple formal and informal interactions with global regulatory agencies on PM359 program and Prime Editing platform INTERACT and pre-IND meetings with the FDA Highly positive interactions with one ex-U.S.agency to-date;two addit
38、ional pending for early 2024Prime Medicine has aligned with FDA recommendations regarding:Preclinical data Toxicology CMC Off-target Clinical development plans In 2023,FDA:Established Office of Therapeutic Products under Dr.Nicole VerdunIntroduced novel initiatives for expediting development of gene
39、tic medicines Platform designation:allows companies to leverage data across programs using modular components START program:increased regulatory feedback for therapies targeting rare diseases with morbidity in first decade of lifeAllowed first clinical trials of base editing-and in vivo CRISPR-based
40、 therapies to proceedApproved first BLA of CRISPR-based therapy in Vertexs exa-cel BLA=biologics license application;IND=investigational new drugOn-track to file first IND in 1H 202413Prime Medicine is Focused Internally on Four Pillars,Each with Demonstrated High Efficiency,Precise in Vivo EditingB
41、usiness development can extend reach and impact,bolstering our financial resources and maximizing the potential of Prime EditingHematology&ImmunologyLiverOcularNeuromuscularBusiness DevelopmentStrong company foundationExpert at designing guide RNAs,mRNA and vector genome sequenceClinical and Regulat
42、ory know-howCMC expertise 14Strategic pillarIndicationDeliveryDiscoveryLead optimizationIND-enablingPhase 1/2HEMATOLOGY&IMMUNOLOGYChronic Granulomatous Diseaseex vivoOther programs in discovery:Fanconi Anemia,Cell Shielding LIVERWilsons DiseaseLNPGlycogen Storage Disease 1bLNPUndisclosedLNPOCULARRet
43、initis Pigmentosa/RhodopsinAAVOther programs in discovery:Retinitis Pigmentosa/Usher Syndrome,Fuchs Endothelial Corneal Dystrophy NEUROMUSCULARFriedreichs AtaxiaAAVOther programs in discovery:Amyotrophic Lateral Sclerosis,Huntingtons Disease,Fragile X SyndromeMyotonic Dystrophy Type 1viral/non-viral
44、Other programs in discovery:Oculopharyngeal Muscular Dystrophy,Duchenne Muscular Dystrophy ADDITIONAL PROGRAMS Advancing as potential partnership opportunities Cystic Fibrosis(lung)LNPCAR-T(oncology/autoimmune)ex vivoOther programs in discovery:Usher Syndrome(Type 3)(ear);Non-Syndromic Hearing Loss
45、GJB2(ear)Our Pipeline:Aligned to Four Core Modular Platforms,With Additional Programs Advancing as Potential Partnership Opportunities15NCF1 gene encodes the p47phox protein;HSCT=hematopoietic stem cell transplant;1P47phox mutation in 25%of patients 2Prevalence 1:100K-200KAdvancing PM359 to the Clin
46、ic for Chronic Granulomatous Disease,A Disease of Significant Unmet NeedRare genetic disease,characterized by defective neutrophil function Serious life-threatening disease presents in childhood;life expectancy 40 years Caused by mutation in the p47phox protein1-Found globally;100s of patients in U.
47、S.alone2 Results in recurrent,life-threatening infections-Difficult to eradicate-Frequent hospitalizations,IV antibiotics-Potentially deadly infections from normal exposures(gardening,swimming)Causes ongoing autoimmunity and inflammation-Deteriorating lung function-Inflammatory bowel-like syndromes-
48、Urinary and gastrointestinal obstruction Current treatment options-Lifelong anti-microbial therapy:ultimately fails due to evolution of antimicrobial resistance-Allogeneic HSCT,only curative option:complicated by GvHD,graft failure,limited availability We believe Prime Editing is uniquely well-suite
49、d to initially address this form of CGD16 DP manufacturing site GMP ready Prime Editing components GMP manufactured,QC tested and ready-for-use to make PM359 Global trial sites selected to maximize access to patients,expedite enrollmentWith PM359,Prime Medicine is Set to Become a Clinical-Stage Comp
50、any Poised to Deliver Data in Near-TermDP=drug product;1On-track to file first IND/CTA in 1H 2024Key eligibility criteria delGT mutation in NCF1 gene Dihydrorhodamine(DHR)c/w CGD Recent or on-going infectious/inflammatory CGD complicationsKey outcome measures DHR 20%normal neutrophil function Resolu
51、tion pre-existing infectious/inflammatory CGD complications Frequency new infectious/inflammatory CGD complications PM359 is comprised of autologous hematopoietic stem cells modified ex vivo using Prime EditingIND 1H 20241First clinical data expected in 2025EnrollMobilize Manufacture Condition6 mont
52、hsDP InfusionEngraftmentDHR Read-out1-3 monthsPrimary endpoint12 months Cohort 2:n=2-3,ages 12-17Cohort 3:n=2-3,ages 6-11Cohort 1:n=2-3,age 18Study Expansion into Pivotal:All ages 617Cell Shielding and In Vivo Delivery or Targeting Has Potential to Expand HSC Platform Beyond Rare DiseasesHSC=hematop
53、oietic stem cell Current efforts lay the foundation for wider range of rare and non-rare indications:benign conditioning with CD117 cell shielding enables non-toxic bone marrow transplant Platform ExpansionTechnology DevelopmentMultiplex EditingPASSIGENon-Viral HSC DeliveryEx Vivo EditingCGD ex vivo
54、Cell ShieldingAutologous HSC Transplant with Corrective EditFuture Program(s)In Vivo HSC Gene Editing TherapiesFuture Program(s)Low Intensity Allogeneic HSC TransplantFuture Program(s)Future Program(s)Low Intensity Autologous HSC TransplantFuture Program(s)Our Current Focus:Conditioning toxicity is
55、major bottleneck to HSC transplant.Combining Prime Editing with Cell Shielding:To improve safety and effectiveness of HSC transplant,significantly improving:Accessibility Eligibility Outcomes To enable selection of in vivo edited HSCs,allowing for treatment of genetic diseases without transplant18Pr
56、ecise and efficient editing of up to 83%of hepatocytes in NHPs Separately,no off-target editing detected in patient-derived iPSCsAdditional data showed repeat dosing of NHPs was generally well tolerated,and led to at least equal levels of precise editing1%Hepatocytes precisely edited is calculated f
57、rom NGS of whole liver biopsy,factored for 60%of cells in liver are hepatocytes(Based on PK/PD relationships and qualifications of cell types in liver:Wang et al Sci.Rep.(2021)11:19396;MacParland et al Nat Commun.(2018)9:4383;Hansel et al,Curr Protoc Toxicol(2014)62:14.12.1;Kmiec,Adv Anat Embryol Ce
58、ll Biol.(2001)161:IIIXIII.1151).Data presented at ESGCT 2023,October 2023.NHP=non-human primateProprietary LNP Platform is Advancing Toward the Clinic for the Treatment of Liver DiseasesUniversal targeted LNPProof-of-concept in GSD1b may accelerate development of all liver programs,including Wilsons
59、 Disease and other undisclosed programs in rare and non-rare liver diseasesPotential minimal threshold for patient benefitControl Day 7 Day 14%hepatocytes precisely edited1LNP delivered Prime Editors achieved high levels of precise editing in the livers of NHPsLNP delivery mechanism shown to precise
60、ly correct disease-causing mutations in the liver of NHPs19Precise and efficient correction of prevalent RHO mutations:up to 65-70%precise correction in photoreceptors in vivo Prime Editors prevented degeneration of retina in vivoSeparately,no off-target editing detected in human photoreceptorsNo de
61、tectable evidence of viral vector integration into retina cells1Editing within transduced area of mouse retina;25%is predicted to be therapeutically beneficial based on Cideciyan et al.,1998.PNAS 95,7103-7108.Mutations in RHO p.P23H and the two hotspot mutations p.V345L/p.P347L affect approximately
62、60%of patients with RHO adRP.Single Prime Editor corrects 18 different pathogenic mutations within single hotspot.Data presented at RD2023,October 2023Early Data with Proprietary Dual-AAV System:Key Step Toward Unlocking Opportunities in Retinal Diseases and Larger Indications Proof-of-concept achie
63、ved:demonstrated ability to correct pathogenic mutations in the eye with high efficiency and no off-target edits detectedProof-of-concept in RHO adRP potentially accelerates development of all retina programs,including Retinitis Pigmentosa/Usher Syndrome program,as well as other ophthalmological dis
64、easesProprietary dual AAVCorrection of prevalent P23H mutation1Correction of 1 of 18 mutations in a mutational“hotspot”1Minimal threshold for patient benefitControl%precise correctionPrime EditorControlPrime EditorIn RHO adRP,Prime Editors efficiently corrected a prevalent RHO mutation and all mutat
65、ions in a mutational“hotspot”20Prime Editors offer a potential curative therapeutic approach for repeat expansion diseases and other neuromuscular diseasesPrime Medicine is leading with Friedreichs ataxia and amyotrophic lateral sclerosisEfficient Prime Editing of neurons by local delivery to the CN
66、S observed in mice Current focus on modular AAV delivery system to CNS in large animal studiesPrime Editors offer genetic correction in patient-derived neurons and muscleEarly In Vitro and In Vivo Data Suggest Potential for Prime Editing To Address Many Neuromuscular Repeat Expansion Diseases toxic
67、RNA foci resolved RAN peptides resolved C9ORF72 level restored Transduced%of neurons transduced%precise correction in neuronsPrime EditedIsolate transduced neuronsMeasure editingPE restored motor neuron functionPE restored sensory neuron functionPE efficiently edited rodent neurons in vivo delivered
68、 by dual AAVPE restored dystrophin and cardiomyocyte functionFriedreichs AtaxiaAmyotrophic Lateral SclerosisDuchenne Muscular DystrophyDeliveryPE=Prime Editing;CNS=central nervous system21Prime Medicine will remain active in both sell-side and buy-side business development,with the goal of accelerat
69、ing our pipeline,bolstering our financial resources,and maximizing the potential of Prime EditingNHP=non-human primate;IND=investigational new drug;CTA=clinical trial application Business Development Remains Core Focus for Building Prime MedicineWithin the CorePartner at the right time with goal to
70、accelerate and globalizeOutside Our CoreCollaborate/license now(e.g.,CAR-T,ear,cardiovascular/cardiometabolic)Access Enabling InnovationAdvance delivery and manufacturing capabilitiesRecent accomplishments have built a strong foundation to facilitate execution of a multi-pronged business development
71、 strategy in 2024 and beyond NHP proof-of-concept achieved Murine proof-of-concept achieved across several programs and delivery modalities Expected first IND/CTA application following positive regulatory discussions Industrialization of Prime Editing platform,enabling the exploitation of modularity
72、 to rapidly develop product candidates Foundational patents issuedWithin Our CoreOutside Our CoreAccess Enabling Innovation22Data presented at ASH,December 2022,ASGCT,May 2023 and ASH,December 2023PASSIGE and Multiplex Prime Editing Create Potentially Best-in-Class Allogenic CAR-T Cell ProductModula
73、rity of platform has potential to accelerate development of additional CAR-T programs Existing LimitationsPrime Editing SolutionMultiplex EngineeringLow payload integration efficiencyConstrained to limited number of knock-outs and limited single base pair changes80%integration efficiency to date,aim
74、ed at TRAC locus to maintain endogenous controlCapable of multiple edits done safely,each with a full suite of functional modifications SafetyRandom or semi-random integrationHigh rate of translocations/chromosomal abnormalities Precise on-target transgene integrationNo detectable off-target edits,t
75、ranslocations,or unintended structural abnormalitiesManufacturing/Cost of GoodsDependence on viral componentsComplicated by multi-step engineeringEntirely non-viral manufacturing process Single-step editing and integration23Eight hotspot Prime Editors could address the“high unmet need”mutations;Thes
76、e same eight hotspot Prime Editors could address 98%of all CF patientsPrime Editors Correct“High Unmet Need”CF Mutations,For Example,the Prevalent G542X(null)MutationPrecise correction of the G542X mutation1Precise correction of both the I507del1 and prevalent F508del mutation2Prime Editors efficien
77、tly corrected the G542X mutation and mutations in the I507del/F508del“hotspot”One-time,non-viral delivery to patient intestinal organoids restored CFTR function Van Mourik et al.,2019.Actual time course:24 hours.TRIKAFTA is a registered trademark of Vertex Pharmaceuticals,Incorporated.1G542X and I50
78、7del are“high unmet need”mutations;F508del is one of the most prevalent CF mutations;2data show correction in patient iPSCs.Each dot shows a different Prime EditorHealthy controlG542X with mock treatmentG542X with Prime Editing correctionG542X with TRIKAFTA treatmentPrime Editing of G542X patient in
79、testinal organoids restored CFTR functionIntestinal organoids swelling assay for CFTR functionLNP delivered Prime Editors efficiently corrected patient Human Bronchial Epithelial(HBE)progenitors in vitroIdentified early LNP formulations to deliver Prime Editors to lung basal cells in vivo24Prime Med
80、icine Holds Extensive Intellectual Property for Prime Editing Technologies Prime Medicines IP includes:Seminal Prime Editing PublicationDual Flap Prime EditingPASSIGE SystemEngineered pegRNAspegRNA Enhancements PE4,PE5,and PEmaxNovel PE ProteinsImproved recombinases Multiple configurations of RNA-te
81、mplated gene editing-Prime Editor protein configurations:fusion,separate and split configurations-pegRNA configurations:fusion,split,separate and engineered configurations-Dual flap and dual guide RNA editing systems Broad diversity of RNA-templated gene editing systems-Large variety of nucleic acid
82、 programmable DNA binding proteins-Extensive range of RNA-dependent DNA polymerases(reverse transcriptases)PASSIGE:System using Prime Editing and recombinase to insert gene-sized DNA at chosen target location in genome-PASSIGE systems include various gene editing configurations and recombinases Addi
83、tional gene editing technology including DNA-dependent DNA polymerase editing Program-specific patent filings for pipeline programs Prime Medicine has 3 issued US patents and 1 allowed US application-Numerous pending applications worldwide with broad coverage-Aggressive filing strategy covering tech
84、nological advances25As of September 30,2023,Prime Medicine had cash,cash equivalents,and investments of$165.3 million,excluding restricted cash,or$178.8 million,including restricted cashKey Upcoming Events will Drive Prime Medicine Forward,Support Our Maturation into a Clinical-Stage CompanySummary
85、of key ongoing activities and planned next steps for Prime Medicine in 2024-2025PipelineHematology&Immunology-Open IND and/or CTA for Phase 1/2 study in Chronic Granulomatous disease in 1H 2024,with anticipated initial clinical data in 2025-Advance Shielded HSC and Immunotherapy Pairs(SCIP)technolog
86、y,establish proof-of-concept in HSC and immunotherapy,and identify first clinical program(s)with this approach in 2024-Advance Prime Medicines differentiated CAR-T program(using PASSIGE)into lead optimizationLiver-Continue to advance preclinical studies for our 3 liver programs,and initiate IND-enab
87、ling activities for at least one in 2024,leading to an IND/CTA in 2H 2025/1H 2026 Ocular-Nominate development candidate for Retinitis Pigmentosa/Rhodopsin(RHO)in 2024 and initiate IND-enabling activities in 2024Neuromuscular-Continue to advance Friedreichs Ataxia,and advance one other program into l
88、ead optimization in 2024PlatformDelivery-Nominate first development candidate using Prime Medicines liver-targeted universal LNP platform in 2024-In large animal studies,establish AAV delivery platform and a route of administration for neuromuscular programs in 2024Regulatory-Advance discussions with Regulatory agencies on platform strategy for streamlined developmentIND=investigational new drug;CTA=clinical trial application;HSC=hematopoietic stem cell Delivering on the promise of Prime EditingThank you!