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OptimizingImmunotherapies
CORPORATEOVERVIEW
(Pleasecontactustorequestconfidentialversionofpresentationwithsupportingdata)
www.Vycellix.com 2
CertainstatementscontainedinthispresentationorinotherdocumentsofVycellix,Inc.(the“Company”),alongwithcertainstatementsthatmaybemadebymanagementoftheCompanyorallyinpresentingthismaterial,maycontain“forward-lookingstatements”asdefinedinthePrivateSecuritiesLitigationReformActof1995.Thesestatementscanbeidentifiedbythefactthattheydonotrelatestrictlytohistoricorcurrentfacts.Theyusewordssuchas"estimate,""expect,""intend,""believe,""plan,""anticipate,"“projected”andotherwordsandtermsofsimilarmeaninginconnectionwithanydiscussionoffutureoperatingorfinancialperformanceorcondition.ThesestatementsarebaseduponthecurrentbeliefsandexpectationsoftheCompany'smanagementandaresubjecttosignificantrisksanduncertainties.
ForwardLookingStatements:
www.Vycellix.com 3
§ Closely-held biotech advancing innovative small moleculesthat optimize and enhance human immune responseagainst disease with a focus on cancers
§ Founded in 2015 on IP developed by medical researchers atSweden’s Karolinska Institutet (KI)
§ Pipeline of novel, targeted small molecules for ex vivo andin vivomodulation of cells
§ Lead products have demonstrated unrivaled enhancementof cytotoxic lymphocytes
§ Scientific founders include KI’s current Head of Gene & CellTherapy Group and KI’s former Dean of Research
CorporateOverview
4
EvrenAlici,M.D.,Ph.D.:CEO&Chairman•Head,GeneandCellTherapyGroup,DivisionofHematology,DepartmentofMedicine,Karolinska Institutet,Karolinska UniversityHospital,Stockholm
Hans-Gustaf Ljunggren,M.D.,Ph.D.:ChiefMedicalOfficer• FormerDeanofResearch,Karolinska Institutet andFounderoftheCenterforInfectiousMedicine,DepartmentofMedicine,Karolinska Institutet,Karolinska UniversityHospital,Stockholm
DouglasW.Calder:President• StrategicAdvisortotheNSUCellTherapyInstitute;formerExecutiveVP&OfficerrolesattheVaccine&GeneTherapyInstitute(VGTI),AccentiaBiopharmaceuticals,Viragen andBiovest International
SamuelDuffey,Esq.:ExecVP,GeneralCounsel•CEOofViraCell,ahollow-fibermanufacturingVLPvaccinecompanybasedatTexasA&M;formerrolesincludePresident&CEOofAccentiaBiopharmaceuticalsandBiovest International,andattorneyatSEC
MichaelJ.Keller,J.D.:ExecVP,IntellectualPropertyOfficer•CEO,KellerLifeScienceLaw;formerrolesincludeChiefPatentCounselforIVAX,partneratleadingIPlawfirms,andtechnologytransferrolesattheNationalCancerInstitute(NCI)
Vycellix FoundingBoardofDirectors:
www.Vycellix.com 5
§ Optimizinggenemodification
UnrivaledEnhancementofNKcells&TcellsLeadproductcandidates:VY-OZ&VY-X
§ Rapidlyincreasingexvivoand invivoimmuneresponse
ExVivoApplications
InVivoApplications
ExVivoApplications
Small Molecule Immunomodulator
Small Molecule Immunomodulator
InVivoApplications
Pre-INDCandidates:APipelineinEachMolecule
GeneticModificationEnhancement
Platform
OptimizesCRISPR-Cas9Gene
EditingRates
UnrivaledTransductionRatesforLymphocytesandStemCells
PlatformtoBoostSerialKillingfor
CytotoxicLymphocytes
UnrivaledIncreasedLevelsofPerforin &GranzymeB
SUPRESSINGCERTAINRNAPATHWAYS
OPTIMIZINGADOPTIVECELLTHERAPIES
IMPROVINGGENEEDITINGEFFICACY
DecreasesgRNARecognition&
IncreasesEditingEfficacy
OPTIMIZINGADOPTIVECELLTHERAPIES
Myositis,Myocarditis,Encephalitis,Meningitis
AdjuvantTherapyforDampening
ViralInflammation
HIGHLYSPECIFICANTI-TUMORAGENT
AdjuvantTherapytoOncolyticViralVectorTherapies
SignificantlyInhibitsTumorDevelopmentinMyelomaModels
EnhancesDistributionofOncolyticVirusWithinTumor
TargetedExpressionofPerforinandGranzymeB
www.Vycellix.com 7
ProductDevelopmentStrategy:
ü Platformtechnologyfortheefficient,cost-effectivemodulationofcellandgenetherapies
ü Monotherapyand/oradjuvanttherapeuticstargetingcancersandinfectiousdisease
ü EnhancersofgeneeditingforCRISPR-Cas9systems
www.Vycellix.com 9
DespiteNKcellsbeinginherentlyresistanttoretroviralinfections,VY-OZ:
§ Enablesretroviralandlentiviral genedeliverytoNKcells,targetingintracellularantiviraldefensemechanisms
§ Providesdosedependenteffectwithnoobservedtoxicity
VY-OZ:ExVivoGeneticModification
www.Vycellix.com 10
§ OptimizesgeneticmodificationofTcells&NKcells
§ Supportsdesignof:- NKcellsexpressingcytokines- Silencedinhibitoryreceptors- Activatingreceptors- CARs
§ IncreasesgeneeditingratesforCRISPRsystems
VY-OZExVivoPlatformSummary
www.Vycellix.com 12
Standardpracticeforadoptivecelltransferislimitedbyprocesses,includinginvitrocytokineexposure,thatfailtoobtainsufficientserialkillingbyeachcytotoxiclymphocyte.
Incontrast,VY-X:§ IsanovelRNAconstructthatishighlyspecificandleadstoa
significantlyincreasedloadofperforin andgranzyme BinNKcellsandTcells
§ OvercomestheobstaclesoflimitedefficacyofadoptivetransferofNKcellsandTcellsbyincreasingcytotoxicactivity
VY-XExVivoPlatform:EnhancesSerialKilling
www.Vycellix.com 13
§ 1600-2000x increaseinPerforin§ 800-1100x increaseinGranzyme B§ FirstknownreportofaRNAconstructleading
tosuchadramaticdifferenceintheproteomicprofileofcytotoxiclymphocytes
VY-XExVivoPlatform:IncreasestheKillingPowerofCytotoxicCells
www.Vycellix.com 14
BecauseVY-X isanovelRNAconstructthatishighlyspecificandleadstoasignificantlyincreasedloadofperforin andgranzyme BinNKcellsandcytotoxicTcells,itisbeingadvancedasadirecttherapeutic.
§ Showssignificantdelayintumordevelopmentcomparedtoactivecontrols inexperimentaltumorsystems
§ Safewithnooff-targeteffectsorsevereadverseeventsobserved
§ Believedtoimproveclinicaloutcomeswhencombinedwithotherimmunotherapies
VY-XInVivo:DirectCancerTherapeutic
www.Vycellix.com 15
IPProtectedTechnologyPlatform§ Assets are not encumbered by any external ownership
rights, royalty obligations or other restrictions§ Market exclusivity strategy includes patents, trade
secrets and planned orphan drug protections§ IP filing: “Enhanced Gene Delivery to Natural Killer Cells,
Hematopoietic Stem Cells and Macrophages”§ IP filing: “Novel RNA Construct”
www.Vycellix.com 16
InvestmentHighlights:§ VY-OZ&VY-X (exvivo)representimmediatemarketopportunities
asplatformstooptimizeadoptiveimmunotherapyby:- enhancinggeneticmodification- improvingserialkillingcapacity
§ VY-X (in vivo) represents a highly-specific anti-cancer agentenhancing T cell/NK cell mediated immune response
§ VY-OZ (ex vivo) represents a genome editing optimization agent toincrease gene editing rates for CRISPR-Cas9 systems using viralvectors
§ Other market opportunities for pipeline: oncolytic viral vector uses,targeting viral inflammation and veterinary applications
§ IP provides long-term market exclusivity protections
§ Significant corporate partnering/licensing interest
§ World-class scientific leadership
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OptimizingImmunotherapiesTorequestConfidentialslidedeck
withsupportingdata,pleasecontact:DouglasW.Calder,President
Phone:772-418-6302/Email:[email protected]