波士顿咨询BCG2021细胞和基因治疗的未来之路英文版10页.pdfVIP

PeterMarksDiscussestheRoadAheadfor

CellandGeneTherapy

AUGUST27,2021

ByMichaelChoy

BCGManagingDirectorandPartnerMichaelChoyrecentlysatdownwithPeter

Marks,thedirectoroftheCenterforBiologicsEvaluationandResearch(CBER)at

theFDA.CBERisresponsibleforassuringthesafetyandeffectivenessofbiological

products,includingvaccines;allergenicproducts;bloodandbloodproducts;and

cellular,tissue,andgenetherapies.Belowareeditedexcerptsfromtheirdiscussion

ofseveraltopicsthatareonthemindsofcellandgenetherapy(CGT)executives

today.

©2021BostonConsultingGroup1

WhatexcitesyoumostaboutthefieldofCGTtoday?

Thereareacoupleofexcitingareas:cell-basedgenetherapiesanddirectly

administeredgenetherapies.Forcell-basedtherapies,primarilychimericantigen

receptorTcells,we’vereallyseenprogressinmanufacturinganddevelopment.

Theyaremoreroutinelyprogressingthroughthedevelopmentprocess,sonow

we’restartingtoseemultipleapprovalsperyear,andbranchingoutfromjustthe

onehematologictarget,CD19.Onthehorizonareotherhematologictargets,as

wellas,potentially,solidtumors—especiallyaswestarttolookatallogeneicCAR-

Tcells,whichcouldalsobetransformative.

We’vealsoseenalotofprogresswithdirectlyadministeredgenetherapies.We’re

startingtobumpintosometoxicities,butwe’realsoseeingprogressintermsof

positiveresults,improvementsinmanufacturingtechnologies,andstartingto

branchoutintothingsascuttingedgeasdirectlyadministeredorinvivogenome

editing,which,oncewegetovertheissuesofoff-targeteffects,couldbereally

transformative.

INNOVATIONSINCLINICALDEVELOPMENT

Whatkindsofopportunitiesarethereforinnovationinclinical

development?

There’soneforgenetherapieswithve