2024年生物技術(shù)“猛”公司Fierce TOP15（英文版）-醫(yī)藥資源云

fX①

SPECIALREPORTFIERCE15

IntroducingFierceBiotech's2024Fierce15

ByAnnaleeArmstrong

Aug5,202406:00am

Fierce15EmergingBiopharmastartupsAbderaTherapeutics

OneofourFierce15honoreesdovedeepintotheoceanforinspirationfromtheseasquirttodevelopnewhumantherapeutics.Anotherthoughttheadorable13-linedgroundsquirrel,withitspolkadotsandhibernationhabits,mightprovideacluetonewtreatments.

Andthere'sthebiotechthatlookedattheHumanGenomeProjectandthought,whatelsecanitdo?

There'sacompanylookingatpsychedelicsinawholenewway,risingontherecentinterestinrepurposingtheillicitsubstancesforsomethingbetter,likeimprovingmentalhealth.

Andthen,overinNewZealand,onetinycompanyislevyingafightagainstviralinfections.Ithasbeentolditsmissionisoutrageous,butwedon'tthinkso.

Welcometothe2024Fierce15.TheFierceBiotecheditorialteamisproudtopresentthisclassofthebestinbiotech.These15companieswerecarefullyselectedfromhundredsofnominees.

Thisisthehardestprojectwedoallyear-topickjust15companiesbreakingbarriersanddefiningthefutureoftheindustry.Butit'salsothemostfulfllingwhenwe'redoneandthemostexcitingtopublish.Soreadontofindoutwhowepickedthisyear.

β

Abdera

Therapeutics

LoriLyons-Williams

CEO

AbderaTherapeutics

ByDarren/ncorvaia

Targetingtough-to-treatcancerswithversatileradiopharmaceuticals

Founded:2020

Based:Vancouver,BritishColumbiaandSouthSanFrancisco,California

Clinicalfocus:Smallcelllungcancerandlargecellneuroendocrinecarcinoma

WhatmakesAbderafierce:WhenGreekphilosopherDemocrituswasdevisinghisatomictheoryalmost2,500yearsago,henevercouldhaveguesseditwouldonedayleadtoaradiopharmacompanybearingthenameofhishometown.Butthat'sexactlywhat

happenedin2023,whenAbderaTherapeuticsemergedfromstealth(/biotech/abdera-skyrockets-out-stealth-142-million-biotechs-radiopharmaceutical-ambitions-take)withawarchestofS142millionandaplantousethepowerofradioactivedecaytodestroycanceroustumors.

RadiopharmaceuticalshavebeenlightingupthebiotechindustryeversinceSwispharmaNovartishititbigwithcancerdrugsPluvicto

-whichin2023narrowlymissedblockbusterstatusinitsfirstfullyearonthemarket-andLutathera.AflurryofacquisitionsfollowedsuitasotherBigPharmastriedtomimicthatsuccess.

"There'sbeen,Ithink,fourtransactionstotalingcloseto$10billioninjustthelastcoupleofquartersalone,"AbderaCEOLoriLyons-WilliamstoldFierce."There'salotofexcitementintargetedradiotherapeutics."

Mostoftheresearchinradiopharma,Lyons-Williamssaid,hasfocusedonjustoneortwotargets,thesamemoleculestargetedbyLutatheraandPluvicto.Lutatherahomesinonsomatostatinreceptors,whichareheavilyexpressedbyneuroendocrinetumors;

Pluvictoaimsforprostate-specificmembraneantigenonthesurfaceofprostatecancercells.

“WereallybelievethatourROVErplatformisenablingustogettonextgenerationtargets,sothatwecanhopefullydifferentiateandexpandintonewtypesofcancer,"Lyons-Williamssaid.

ROVErstandsforRadioOptimizedVectorEngineering,andit'sthetechniqueattheheartofAbdera'sambitions.Co-foundersAdamJudge,Ph.D.,andMichaelAbrams,Ph.D.,withbackgroundsinbiologicsandradiopharma,respectively,anticipatedthatnuclear

medicinewasabouttotakeoffanddevelopedamodularantibodyplatformthatcouldbeeasilymodifiedtotargetnewtypesofcancers.Fellowco-founderLanaJanesledthecompany'sincubationandspin-outofadMareBioinnovations.

Thegoalwastocreatetheidealsystemfordeliveringradioactiveisotopestotumors,lettingthemdecayanddestroycancercell,and

thenclearingthemfromthebodyasquicklyaspossibletoavoidcollateraldamage.ThedatasofarsuggestAbdera'santibodies"maybeabletodeliverbest-in-classtumoraccumulation,"Lyons-Williamssaid,andthen“getoutofthesystemmorerapidlyandultimately

mitigatedamagetohealthytissue."

Abdera'sflagshipdrugisABD-147,whichtargetsaproteinfoundonneuroendocrinetumorcellcalleddelta-likeligand3.ABD-147recentlyscoredafast-trackdesignation(

/press-releases/abdera-therapeutics-announces-fda-fast-track

-

designation-for-abd-147-a-next-generation-precision-radiopharmaceutical-therapy-for-the-treatment-of-patients-with-extensive-stage-small-cell-lung-cancer/)fromtheFDAforextensive-stagesmallcellungcancer(SCLC),withtheclinicaltrialsettobeginenrollingpatientslaterthisyear.

ExtensiveSCLChasanenormousunmetneed,withsurvivaltypicallyonlysevento11monthsafterdiagnosisevenwithtreatment;

Lyons-WilliamsthinksthissoberingfactplayedaroleinAbdera'sfast-tracksuccessalongwiththepreclinicaldataitpresentedtotheagency.

"Theresponsesthatwesawintheanimalmodelswerequitecompelling,"shesaid."Inadose-dependentfashion,withjustasingledoseofourmedicine,wesawtotalablationofthetumor."

IfABD-147provessafeandeffective,Lyons-WilliamssaidAbderawillbeabletoquicklymoveotherdrugsthroughthedevelopment

pipeline,too.Thisisthankstothemodularstructureoftheirantibodies,wheremuchofthemoleculeremainsthesamenomatterwhatthetargetis,aswellasAbdera'sstrategiccapacitybuilding.

ThefrstexecutivehireLyons-WilliamsmadeafterjoiningthecompanywasChiefTechnicalOfficerKawaChiu,theCEOremembers.

"Thatwouldn'tnecessarilybethefirstexecutiverolethatmostpeoplemighthire,but,inthiscase,thesupplychainofisotopesisnot

completelystable."Becausetheisotopesstartdecayingassoonasthedrugisconjugated,sheadded,"theabilitytobuildajust-in-timemanufacturingprocess"iscriticalforsuccess.

Withitsversatileplatformandmanufacturingcapabilities,Abderaisonamissiontoexpandintoothersolidtumortypesandprovidefirst-in-classtreatment."Therearesomerealltough-to-treatcancersouttherewhere]othermodalitieshavejustnotworkedsupereffectively,"Lyons-Williamssaid.“Atourcore,whatwecareaboutthemostisbeingabletoimpactmorepatients."

Ifupcomingdatalookcompelling,there'soneplacetheyalltalkaboutvisitingtocelebrate:Abdera,Greece."It'soneofouraspirations,"shesaid.

Investors:VersantVentures,AmplitudeVentures,venBioPartners,adMareBioInnovations,VikingGlobalInvestors,QimingVenturePartnersUSA,RTWInvestments,AbCellera,NorthviewVentures

AscidianTherapeuticsfX①

ByAnnaleeArmstrong

Developingexon-editingRNAtherapeuticswithinspirationfromseasquirts

Founded:2020Based:Boston

Clinicalfocus:RNAtherapeutics,eyediseases

WhatmakesAscidianfierce:Isitweirdtobefierceandfun?Orfiercelyfun?AscidianTherapeuticsCEOMichaelEhlers,M.D.,Ph.D.,wantstoknow.Becausehislatestbiotechis"oneofthemostfunworkenvironments"he'severbeenapartof.

Yes,hemeansthecompany'ssocialengagementsareoffthechain,buthealsospeaksofacompetitivespiritamongthestaffthatispushingthemtobethebestintheRNAfield.

“Werefercelycompetitive,"EhlerssaidinaninterviewwithFierceBiotech."It'sagroupthatreallywantstobethebestanddothe

bestatthethingsthattheyredoing…Everythingfromthewaythegroupmightcompeteinasocialactivityonsomething,whetherit'striviaorshuffleboardorwhateveritmightbe,tojustchallengingeachothertowardstheverybestexperiment,therighttechnology

thatcanbeappliedtothatexperiment."

Theblueascidian,atypeofseasquirt(johnandersonphoto/iStock/GettyImagesPlus)

It'snotsurprisingthatacompanyinspiredbythehumbleseasquirtwouldbefun.Ascidian-anothernameforseasquirts-is

developingexon-editingRNAtherapeuticsinthehopesofaddressingtheunderlyingcauseofretinalandneurologicaldiseases.TheideaistouseRNAsplicingcombinedwithlarge-scaleDNAandRNAsynthesisanddeep-sequencingtechnologiestorestorenormalproteinfunction.

Todothat,theteamlookedtonature.AndthesmallorganismbestatRNAsplicingjustsohappenedtobetheseasquirt.

“Thebiologyitselfisinteresting,becausetheseseasquirtsundergothissignificantmetamorphosisduringtheirdevelopment,andthatinvolvesthistranssplicing,"Ehlerssaid.

Researchonthisprocesshappenedabout20yearsago,butthetoolsandknowledgeavailableatthetimehadn'tquitecaughtuptothescience.Now,AscidianisheretobringtheideaofRNAsplicingintothemodernageandisadvancingthetechnologyquicklytoward

theclinic.

Ascidianhasaclutchofearly-stagecandidatesalreadyonthego.ThemostadvancedisaretinaldiseaseprogramforABCA4

retinopathy,agroupofdiseasesthatincludesStargardtdisease,ageneticdiseasegenerallycausedbymutationsintheABCA4gene.Aphase1trialwillsoontestwhetherAscidian'sACDN-01issafeandlookforsignsofslowingthedamagetotheretina

Ehlerssaidthegeneinquestionistoolargetobepackagedintoatypicaladeno-associatedvirusthatcurrentgenetherapiesuse,soaddressingthediseaseviaRNAisabetterwaytogo.RNAsplicingcouldapplytoanygeneeditingcasewheremoresubstantive

changesareneeded.

ThetechnologyhasalreadyattractedRoche,whichpaidS42millionupfront(/biotech/roche-wades-

deeper-rna-waters-paying-ascidian-42m-develop-exon-editing-drugs)inJunetosecureexclusiverightstoAscidian'stechnologyfor

undisclosedneurologicaltargets.ThedealincludesuptoS1.8billioninresearch,clinicalandcommercialmilestonepayments.

AscidianwillworkwithRocheondiscoveryandcertainpreclinicalactivitiesbeforehandingfullcontroltotheSwissdrugmakerforfurtherpreclinicalworkandbeyond.

"IthinktheysawinthiscoreRNAexoneditingtechnologytheabilitytodothingsthatsimplycan'totherwisebeaccomplishedbyothertechnologiesinsomeveryimportantdiseaseareasforthem,"EhlerssaidoftheRochepartnership.

Meanwhile,Ascidiancankeeplookingforitsowninternalprogramswithinneurology.Ehlerssaidthecompanyisonthelookoutfor

morepotentialpartnerships,andtalksarecurrentlyongoing.TheStargardtprogramisAscidian'sleadandwillstaythatway,butEhlerssaidtherearesomediseaseareasthetechnologycantacklethatmaynotbeagreatftforthebiotech.Hepromisedmorenewsonthatinthecomingmonths.

Asforfinancing,EhlerssaidAscidianhasstrongbackingfromfoundinginvestorAppleTreePartners,whichhasensuredthatthebiotechcanridethroughtheratherturbulentmarketsoflate.

Thekeychallengehasbeenmaintainingscientificmomentumthroughallofthat.Ehlerssaidit'sbeeneasytoattractateamofdedicatedscientiststothecause-agrouphecalls"theverybestonethatI'veeverseen."

“It'snotjustyourfather'sOldsmobile,thethingsthatweredoing.Thisissomethingreallyquitedifferentanduniquewithuniquepotentialanduniqueimpact,"Ehlerssaid.ThatiswhathasattractedtheteamofveryfunpeopletoAscidian,hesaid.

Thecompanycurrentlysitsaround50people,includingacropofstudentswhojointhecrewtemporaril,andisheadquarteredintheBrightonneighborhoodofBoston.HesaysAscidianevenhasaccesstotheBostonBruins'practicerink.

Withthepotentialforstaffpartiesthatcool,theFierceeditorialteamwillkeepaneyeoutfortheFierce15party.Wewon'tforgetourskates.

Investor:AppleTreePartners

BigHat

MarkDePristoCEO

BIOSCIENCES

BigHatBiosciences

fX①

ByAnnaleeArmstrong

Usingmachinelearningandsyntheticbiologytofindbetterantibodies

Founded:2019

Based:SanMateo,California

Clinicalfocus:Antibodydrugdiscovery

WhatmakesBigHatfierce:Design,build,test,repeat.That'swhattheteamatBigHatBiosciencesdoesweekinandweekout.It'salpartofthebiotech'smissiontodesignmoreeffectiveantibodieswithacombinationofmachinelearningandsyntheticbiologytech.

BigHatco-founderandCSOPeytonGreenside,Ph.D.(BigHatBiosciences)

ThisweeklycycleallowsBigHattobuildmoleculesthatkeepgettingbetterandbetterproperties,asfastaspossible.It'salotofdataandalotofrepetition,whichanycompanytoilinginAIthesedaysknowsplentyabout.ThedifferenceatBigHatisthecombinationofwetlabimprovementspairedwiththecomputersystemstoensurethebreakthroughscanapplytothebiotechworld.

“Wehaveoneofthefastestdesign-build-testlabs,andwereusingthatnotjusttogofaster,but,importantly,todesigntherapeuticsthatareoutoftherangeofotherplatforms,"saidPeytonGreenside,Ph.D.,co-founderandchiefscientificofficer.

CoretoBigHatistheMilinerplatform,whichcombinesmachinelearningandsyntheticbiologytechinaclosed-loopiterativeprocesstodesignandengineerantibodiestotreatdisease.

CEOMarkDePristo,Ph.D.-analumnusofGoogleBrainandtheBroadInstitute-andGreensidemetwhile"wearingaverydifferenthat,"theCEOsaidwithalaugh.GreensidewaspreviouslyacomputationalbiologistattheBroadInstituteandgotherPh.D.at

Stanford.Theywerebothtryingtofigureoutwhethergenomicdatawerereliableandeventuallybegantocollaborate.

"WethoughtthatnotenoughpeopleweretryingtocombinetheAItechnologieswithlabtechnologysothatyoucouldnotonlylearnfromexistingdata,butthenvalidatethosemodelsinnoveldomains,andthenlearnfromthatnoveldatatoimprovethemodels,"

DePristoexplained.

Theysoonrealizedtheneedtochangethewetlabcomponenttoencouragemorefrequentexperimentsthatgeneratehigher-qualitydata.

“Weactuallyturnthecrankinourlabeveryweek.Wereabletogofromadesignedantibodytoactualreal-worldmeasurementsofthatdesignedmoleculeonceaweek,andwecandothatweekinandweekoutonnow1,600moleculeseveryweek,"DePristoexplained."It'sthatspeedanddataqualitythatreallygivesusessentiallythefuelfortheAItechnologiestomoveatthespeedtheyare."

TheMilinerplatformhashelpedBigHatdevelopaninternalpipeline,withassetsunderwayinoncologyandsicklecelldisease,aswell

asatractmajorpartnershipsfromthelikesofJohnson&Johnson,Merck&Co.andAmgen.DePristocallstheplatform"wildlyproductive"andsaysitisa"value-creatingcapability."

Thislabworkisessentialtosolvingthekeyquestion:Isittranslatabletohumans?

"There'snoalgorithmorsimulatorthattellsyou….howamoleculeisgoingtoperforminapatient,"Greensidesaid.“Youreallyhavetomakeitandtestthingsrightinthelab,andsoIthinkweveputanenormousamountofeffortintomachinelearningqualitydata.”

Alsoimportantishavingthelabtechnicianscommunicatewiththeteamdoingthedatacrunching.Greensidesaidtheyestablishedthatcultureearlytoensuresmoothcollaboration.

“Asyougoaroundthekindofdesign-build-testloop,everyoneisbothaconsumerandaproducer.Andthisleadseveryonetosortoffeellikethey….ultimatelygettheconsequencesoftheproductsthatare[beingproduced]oneweeklater,"DePristosaid.

Withtheplatformchurningoutpotentialprograms,DePristoandGreensidesayBigHatisveryfocusedonthetherapeuticspartoftheequation.Theteamofabout90peoplewantstodeveloptreatmentsforrealpatients,andtheCEOexpectstohaveacandidateintheclinicby2026.

Tofundthatwork,DePristoconfrmedthatthecompanyissearchingforwaystoaddmorecapitalbutcalledthefinancingenvironment“complex.”

"Wehaventreallyshakenoffthebiotechwinterof22.Itscoming.It'sgettingbetter,butit'snot100%yet,"hesaid."Wedon'tneedtoraise,butwedothinkthatmorecapitalwillhelpusgoevenfaster."

Thatcapitalcouldcomeinseveralways,heexplained,suchasthroughanadditionalround,alicensingdeal,abiggerpartnershiportappingexistinginvestors.

“Wecertainlyarereadytogrowmore,andwe'llbethinkingaboutexactlyhowtodothat,”DePristosaid.

Investors:8VC,AMECloudVentures,InovationEndeavors,AndressenHorowitz,Section32,AmgenVentures,BristolMyersSquibb,QuadrilleCapital,Gaingels,GRIDSCapital

CourPharmaceuticalsfX①

ByConorHale

Retrainingtheculpritsatthecenterofautoimmunediseases

Founded:2013

Based:Skokie,Illinois

Clinicalfocus:Autoimmunediseases

WhatmakesCourfierce:CourPharmaceuticalshopestobringmorethanfourdecades'worthofresearchintotheimmunesystemtofruitionwithanoveltypeofreversevaccinethataimstoreprogramthebody'saggressive-ifatouchoverzealous-whitebloodcells

andultimatelyconvincethemtonotattackotherwisehealthyorgans.

Thestartup'sgoalistobuildatargeted,platform-basedapproachthatcanaddressdebilitatingautoimmunediseaseswithoutbluntingapatient'sabilitytofightoffaninvadinginfection-andonethatcouldpotentiallybetailoredforavarietyofdifferentconditions.

WithtechnologyoriginallydevelopedattheNorthwesternUniversityFeinbergSchoolofMedicineinChicago,Couremploys

biodegradablenanoparticlesthatencapsulatecertainantigens-specifically,whicheverpeptidesorproteinsareneededtomatchtheonesresponsiblefortriggeringthechainreactionthatdrivesTcellstodestroythebody'sowntissues.

Thesenanoparticleshitcharideonwhitebloodcellsknownasmonocytesastheytraveltothespleenandliver.Whenthosecellsarerecycledbythebody,theantigensarereleasedinawaythatmakesthemlooklikenormaldeadcellulardebris,whichtheimmune

systemthenlearnstorecognize.

Whereatraditionalvaccinewouldinstructthesetypesofcellstoattackaparticularintruder,Cour'sstrategyistoassuretheimmunesystemthatthey'reallowedtopassbyunharmed.

“Itwasa40-yearendeavortryingtofigureouthowyougettheimmunesystemtoberetrainedsothatitwon'tattacktheantigenof

interest,"CEOJohnPuisissaidinaninterviewwithFierce."Wearejustveryfortunatetohitthecrossroadsofaserendipitousscientificmedicaldiscovery."

Intheearlieststagesofthecompany,thetacticwasstudiedasawaytotampdownthedangerousinflammatoryresponsesseenin

peoplewithseverepeanutallergies.Today,Courisfocusedsolelyonautoimmunity,withapipelinethatincludescandidatestargetingType1diabetes,celiacdiseaseandthemuscle-weakeningconditionmyastheniagravis.And,sofar,earlyresultshavebeenpromising,accordingtotheCEO.

“Wecanactuallyabrogatethedisease,"Puisissaid."That'swhatourdatashows,whichisreallyprofound.”

InType1diabetes,whereTcellassaulttheinsulin-producingisletcellofthepancreas,Courplanstohalttheprogressionofthedisease.

Thebiotech'sCNP-103programaimstopresentthebodywith120antigenepitopesbyencapsulatingfourdifferentrecombinantproteinsatonce-andit'sslatedtomakeafirst-in-humandebutnextyearthroughCour'sphase1b/2atrial.

Theplanistostartthestudywithparticipantsintheadvancedstagesofthedisease-namelythosewithestablishedlossesof

pancreaticbetacellswhomaybeconsideringatissuetransplant,despitestillhavingtheautoimmunity-beforedevelopingitintoatreatmentforearlydiagnoses.

COODannielleAppelhans(CourPharmaceuticals)

"TypeIdiabetesisstillnotterriblywellmanagedtoday,despiteimprovementsandcontinuousglucosemonitoring,"saidChiefOperatingOfficerDannielleAppelhans."Butforaddressingtheunderlyingcause,there'sreallynothingavailable."

Meanwhile,Cour'sCNP-106treatmentformyastheniagravishasclearedtheproof-of-conceptstageandisenrolingadultsinasimilar

earlytomidphasestudy(

/study/NCT06106672

).

Forceliacdisease,thecompanyhasfoundapartnerinTakeda,withagloballicensingagreementinkedin2019thatincludesa

commitmentofupto$420millioninpotentialmilestoneandroyaltypayments.Thattherapy-knownasCNP-101orTAK-101,

dependingonwhoyouask-smugglesinthegliadinproteinsthatserveasoneofthetwomajorcomponentsofglutencompounds.

Inapreviousfirst-in-humanclinicaltrialofabout30patients,thecompanydemonstratedthatwithintheweeksfollowingtwo

infusions,measuresofinlammatorycytokinebiomarkersaswellasT-cellactivationandiniltrationintothesmalintestineweregreatlydiminished,evenwhenstudyparticipantsweresubsequentlygivenlargedosesofgluten.

"Doctorswhodiagnosepatientswithceliacdiseasegivethemaglutenchallenge,andthentheygolookattheTcels,"Puisissaid.

“Whatafewdoctorstolduswasthat,withourtreatment,thatpersonwouldnolongerhavebeendiagnosed.Sotakethatforwhatit's

worth,butthat'saprettyprofoundchange,anditwassignificantwithaverysmallpopulation,"hesaid.“I'mbeingcautiouslyexuberant."

Atthesametime,Courexpectsitsproductswillbecheapercomparedtogenetherapies,despitefallingunderthesameregulatorycategoryasothercell-programmingtech.

“What'sreallyinterestingaboutwhatwe'redoingisthatwehavethisplatform,but98%ofitisapolymer,andthentheproteinsareonly1%to2%,"saidAppelhans,whopreviouslyservedasCoOandCEOofcelltherapydeveloperRubiusTherapeuticsandaschieftechnicalofficerofNovartis'genetherapydivision.AppelhanswilltakeovertheCEOrolefromPuisiseffectiveSept.1.

“Sowhatreallyexcitesmeisthepotentialtoreachmanypatientsacrosstheworld,because,whilebeinganadvancedmodality,itdoesn'thavethesamecoststructureassociatedwiththose."

Investors:LumiraVentures,BristolMyersSquibb,RocheVentureFund,PfizerBreakthroughGrowthInitiative,AlphaWaveVentures

ExcisionBioTherapeutics

fX①

ByEricSagonowsky

UsingCRISPRtechtoexciseviralgenomes

Founded:2015

Based:Watertown,Massachusetts

Clinicalfocus:ViralinfectionssuchasHIV,hepatitisBandherpessimplexvirus

WhatmakesExcisionfierce:Antiviraldrugshaveadvancedingreatwaysovertheyearsanddecades,turningHIVintoalargelymanageablediseasethatcanbeeffectivelyprevented.

Atthesametime,vaccinesagainstotherinfectionssuchashepatitisBandrespiratorysyncytialviruscontinuetoimprovepatientoutcomes.

ButExcisionistakingthefightagainstviralinfectionsintoanentirelydifferentrealm.ByusingCRISPRgeneeditingtechnology,thecompanyisworkingtodevelopdrugsthatcompletelyremoveviralgenomesfrominfectedindividuals.

Foundedin2015,Excision'stechplatform(https:/www.excision.bio/technology)islicensedfromthelabofJenniferDoudna,Ph.D.,attheUniversityofCalifornia,BerkeleyandtheKhaliliLabatTempleUniversity.In2019,theExcisionteamcelebratedamajorscientificmilestonewhenitstechenabledscientiststoremoveHIVDNAfromthegenomesoflivingmice

(/news-release/2019/07/02/1877432/0/en/HIV-Eliminated-from-the-Genomes-of-Living-Animals-Excision-BioTherapeutics-Advancing-the-Innovation-to-Clinical-Development.html).Atthetime,thecompanyhailedthe

developmentasavalidationofitsapproach.

AsExcisionCEODanielDornbuschexplainedinarecentinterview,scientistshavelongknownhowtoidentifyviralinfectionsand

inhibitviralreplication.Muchmorechallenging,though,hasbeenthelong-runningeffortto“targetthesourceoftheinfection”andpotentiallycureinfectedindividuals.

That'swhereExcisionhopesitsplatformcanintroduceadramaticparadigmshift.

ThecompanyistestingitsCRISPRviralremovalapproachagainstHIV,ofcourse,butit'salsoworkingonhepatitisBand

otherinfections.There'salreadya“greatvaccine"againsthepatitisB,theCEOexplained,butthat“doesn'tdoanything"forthehundredsofmillionsworldwidewhocurrentlyhavethedisease.

Besidesthosetwoviruses,herpes,herpeskeratitisandBKvirusareamongExcision'sexistingandpotentialtargets.

"There'salitanyofthesevirusesthatunfortunatelyplaguepatientsaroundtheworldthatwewouldlovetohaveanewmodalitytogetasclosetoacureaspossible,"Dornbuschsaid.

Thecompanywasfoundedin2015andreorganizedin2019tolocateitsheadquartersinSanFrancisco,Dornbuschsaid.

Afterraising$60millionthroughaseriesinAin2021(

/news

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release/2021/02/17/2177053/0/en/Excision-BioTherapeutics-Completes-60-Million-Financing-to-Advance-CRISPR-Based-Infectious-

Disease-Programs.html),ExcisionestablishedalabinCambridge,Massachusetts,andhassincemovedthatresearchbasetoWatertown,Massachusetts,onasitethatnowservesasthebiotech'sHQ.

Despiteoperatingwithits"bicoastal"profile,thecompanyemploysjust18people,Dornbuschsaid.Beyondtheinternalstaff,the

biotechworkswithan"extensivenetwork"ofconsultants,advisersandCROs,theCEOadded.Dornbusch,forhispart,joinedin2019.

Overtheyears,Excisionhasadedtop-tierteammemberstoheadupR&D,clinicaloperationsandbioinformatics,Dornbuschsaid.Thecompany'sseniorvicepresidentofR&D,JenniferGordon,Ph.D.,spentdecadesinacademicresearch,includingatTempleUniversity,

beforejoiningExcision.

Thecompany'sleadprogramhashititsprimaryandsecondaryendpoints(https:/www.excision.bio/news/press-

releases/detail/43/excision-biotherapeutics-announces-data-from-the-phase-12)initsfirstclinicaltrial.Inthenextseveralyears,Excisionplanstorunmoretrialsforits"next-generationHIVprogram,"itsherpesprogramanditsHBVprogram,Dornbuschsaid.

Aroundfiveyearsfromnow,thecompanycouldbethinkingaboutpivotaltrialsor"possiblyeveninitiallaunches,"theCEOsaid.Besidestheclinicaleffort,thecompanysoonintendstoraiseaseriesBround.

"Inthelasttwoorthreeyears,Ithinkthefieldhaschanged180degrees,"Dornbuschsaidoftheindustry'sCRISPRandgenetherapyadvances.“I'mgettingphonecallsbythesamepeoplewhosaid,'Thiswillneverwork'[who]now[say]'Thisistheway.”

AsExcisionworksonitsearly-stagepipeline,thebiotechhasalreadystarteddiscussionswithlargepharmasaboutpotential

collaborationstogrowitstech'sglobalreach.Thecompanybelievespartnershipsare“absolutelytherightwaytocommercializethesetherapiesaroundtheworld,"Dornbuschsaid.

Investors:ARTISVentures,GreatPointVentures,NorwestVenturePartners,AdjuvantCapital,CotaCapital,WRVICapital,IndusAgePartners,LoredaHoldings,Oli