CGT

7개 · ADC 882 · Antibody 4071 · CGT 2714 · 자동 수집 갱신 권장 · 9월 2일

PopularRecently updatedCuratedMost trialsNeeds refreshORRLatestPhase 3FDA approved7 programs

데이터 갱신 주기입니다. 갱신 대기 7,666개 보기 · Curated Core는 주간 자동 동기화됩니다.

CGTAAVCurated CoreFDAstaleNovartisApproved

onasemnogene abeparvovec (Zolgensma, AVXS-101, onasemnogene abeparvovec-xioi)

Spinal muscular atrophy (SMA) Type 1–3, pediatric · SMN1 (AAV9)

#SMN1AAV9#CGT#Approved#FDA-approved#BioJungboMoa-pick+5
Trials 28Status Approved
CGTAAVCurated CoreFDAstaleSarepta TherapeuticsApproved

delandistrogene moxeparvovec (Elevidys, SRP-9001, delandistrogene moxeparvovec-rokl)

Duchenne muscular dystrophy (DMD) — ambulatory / label-expanded populations · Micro-dystrophin (AAVrh74)

#Micro-dystrophinAAVrh74#CGT#Approved#FDA-approved#BioJungboMoa-pick+5
Trials 12Status Approved
CGTLimited DatastaleKevin FlaniganPhase 2

rAAVrh74.MCK.GALGT2 (rAAVrh74.MCK.GALGT2)

Duchenne Muscular Dystrophy · Duchenne Muscular Dystrophy

#DuchenneMuscularDystrophy#CGT#Phase2#cell-gene-therapy#CAR-T+2
Trials 2Status Active
CGTLimited DatastaleMuscular Dystrophy AssociationPhase 1

Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- Second cohort (Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- Second cohort)

Muscular Dystrophies · Muscular Dystrophies

#MuscularDystrophies#CGT#Phase1#cell-gene-therapy#CAR-T+2
Trials 1Status Active
CGTLimited DatastaleMuscular Dystrophy AssociationPhase 1

rAAV1.tMCK.human-alpha-sarcoglycan- First cohort (rAAV1.tMCK.human-alpha-sarcoglycan- First cohort)

Muscular Dystrophies · Muscular Dystrophies

#MuscularDystrophies#CGT#Phase1#cell-gene-therapy#CAR-T+2
Trials 1Status Active
CGTLimited DatastaleAskBio IncPhase 1

d3990 (rAAV2.5-CMV-minidystrophin, rAAV2.5-CMV-minidystrophin (d3990))

Duchenne Muscular Dystrophy · Duchenne Muscular Dystrophy

#DuchenneMuscularDystrophy#CGT#Phase1#cell-gene-therapy#CAR-T+2
Trials 1Status Active
CGTLimited DatastaleAudentes TherapeuticsPhase 2

scAAV9.U7.ACCA (scAAV9.U7.ACCA)

Duchenne Muscular Dystrophy · Duchenne Muscular Dystrophy

#DuchenneMuscularDystrophy#CGT#Phase2#cell-gene-therapy#CAR-T+2
Trials 1Status Active