瑞信-美股-生物科技行业-生物科技：AAV基因治疗的价值-2019.12.12-137页.pdfVIP免费

12December2019EquityResearchAmericasBiotechnologyFindingValueinAAVGeneTherapy:ACreditSuissePrimerMartinAuster,MD+1212-325-6573martin.auster@credit-suisse.comMarkConnolly+1212325-7576mark.connolly@credit-suisse.comTiagoFauth+1212325-7569tiago.fauth@credit-suisse.comThomasDeal+1212-325-3719thomasavery.deal@credit-suisse.comResearchAnalystsDISCLOSUREAPPENDIXATTHEBACKOFTHISREPORTCONTAINSIMPORTANTDISCLOSURES,ANALYSTCERTIFICATIONS,LEGALENTITYDISCLOSUREANDTHESTATUSOFNON-USANALYSTS.USDisclosure:CreditSuissedoesandseekstodobusinesswithcompaniescoveredinitsresearchreports.Asaresult,investorsshouldbeawarethattheFirmmayhaveaconflictofinterestthatcouldaffecttheobjectivityofthisreport.Investorsshouldconsiderthisreportasonlyasinglefactorinmakingtheirinvestmentdecision.2Source:CreditSuisseResearchWhyYouNeedtoReadThisPrimer,Including:oOursystematicAAVgenetherapyframework,whichweusetoguideourassessmentofcompaniesinthespace;oAdeep-diveanalysisofgenetherapymanufacturing,withaninvestigationofproductioncostsperdose,etc.;andoAdiscussiononhowgenetherapyispricedandwhatcouldchangeinthefutureasitspharmacoeconomicbenefitsarerealized.oWealsoreviewkeylearningsinthespaceacrossthepast20+yearstoframeourthesisonhowtoinvestinthisniche(includingnumerouscasestudiesonwhathas/hasnotworkedandwhatindications/programsmaybeinterestingmovingforward).oAbonus!Pleasecontactusifyou’dlikeourcomprehensiveAAVgenetherapydatabase,acomprehensivereferenceguidethatdetails>190AAVgenetherapyclinicaltrialsthathavebeenconductedovertheyears.(Weincludedetailsonconstructdesign–capsid,promoter,doses;results,ifapplicable;linkstopublications;etc.)ExecutiveSummaryWeHighlight3GeneTherapyNamesUnderCoveragewithMake-or-BreakCatalystsintheNext+/-1yr:oSarepta.Webelieveithasthepotentialtohavethelargeststockmovewithinourcoverageuniverseoverthenext~12-15months.SRP-9001(DMD)hashadencouragingefficacyinasmallPh1/2trialandfunctionalresultsareexpectedfromaplacebo-controlled40-patientPh3trial~late2020.WeexpectDMDgenetherapytosupplantSM...