CGT

1,163개 · ADC 890 · Antibody 4071 · CGT 2714 · 다음 갱신 D-5 · 마지막 9월 2일

PopularRecently updatedCuratedMost trialsNeeds refreshORRLatestPhase 3FDA approved1,163 programs
CGTAAVLimited DatastaleGenzyme, a Sanofi CompanyPhase 1

AAV2-sFLT01 (AAV2-sFLT01)

Macular Degeneration; Age-Related Maculopathies · Macular Degeneration

#MacularDegeneration#CGT#Phase1#cell-gene-therapy#CAR-T+3
Trials 1Status Active
CGTAAVLimited DatastaleeyeDNA TherapeuticsPhase 2

AAV2/5-hPDE6B (AAV2/5-hPDE6B)

Retinitis Pigmentosa · Retinitis Pigmentosa

#RetinitisPigmentosa#CGT#Phase2#cell-gene-therapy#CAR-T+2
Trials 1Status Discontinued
CGTAAVLimited DatastaleMedical Research CouncilPhase 2

AAV2/8-HLP-FVIII-V3 (AAV2/8-HLP-FVIII-V3)

Hemophilia A · Hemophilia A

#HemophiliaA#CGT#Phase2#cell-gene-therapy#CAR-T+2
Trials 1Status Recruiting
CGTAAVLimited DatastaleMeiraGTx UK II LtdPhase 2

AAV5-RPGR (AAV5-RPGR)

X-Linked Retinitis Pigmentosa · X-Linked Retinitis Pigmentosa

#X-LinkedRetinitisPigmentosa#CGT#Phase2#cell-gene-therapy#CAR-T+2
Trials 1Status Active
CGTAAVLimited DatastaleCSL BehringPhase 2

AAV5-hFIX (AAV5-hFIX)

Hemophilia B · Hemophilia B

#HemophiliaB#CGT#Phase2#cell-gene-therapy#CAR-T+2
Trials 1Status Active
CGTAAVLimited DatastaleCSL BehringPhase 2

AMT-061 (AAV5-hFIXco-Padua, AAV5-hFIXco-Padua (AMT-061))

Hemophilia B · Hemophilia B

#HemophiliaB#CGT#Phase2#cell-gene-therapy#CAR-T+2
Trials 1Status Active
CGTAAVLimited DatastaleSpark Therapeutics, Inc.Phase 1

AAV8-hFIX19 (AAV8-hFIX19)

Hemophilia B · Hemophilia B

#HemophiliaB#CGT#Phase1#cell-gene-therapy#CAR-T+2
Trials 1Status Discontinued
CGTAAVLimited DatastaleOpus Genetics, IncPhase 2

AAV8.hLCA5 (AAV8.hLCA5)

LCA5 · LCA5

#LCA5#CGT#Phase2#cell-gene-therapy#CAR-T+2
Trials 1Status Recruiting
CGTAAVLimited DatastaleAspa TherapeuticsPhase 2

AAV9 BBP-812 (AAV9 BBP-812)

Canavan Disease · Canavan Disease

#CanavanDisease#CGT#Phase2#cell-gene-therapy#CAR-T+2
Trials 1Status Recruiting
CGTAAVLimited DatastaleCelosia Therapeutics Pty LtdPhase 1

AAV9 Gene therapy (AAV9 Gene therapy)

ALS (Amyotrophic Lateral Sclerosis) · ALS (Amyotrophic Lateral Sclerosis)

#ALSAmyotrophicLateralSclerosis#CGT#Phase1#cell-gene-therapy#CAR-T+2
Trials 1Status Recruiting
CGTAAVLimited DatastaleUnlimited Biotechnology LLCPhase 2

AAV9-Follistatin gene therapy (AAV9-Follistatin gene therapy)

Age-related Muscle Decline · Age-related Muscle Decline

#Age-relatedMuscleDecline#CGT#Phase2#cell-gene-therapy#CAR-T+2
Trials 1Status Recruiting
CGTAAVLimited DatastaleUniQure Biopharma B.V.Phase 2

AAV9-hSyn1-miGRIK2 (AAV9-hSyn1-miGRIK2)

Mesial Temporal Lobe Epilepsy · Mesial Temporal Lobe Epilepsy

#MesialTemporalLobeEpilepsy#CGT#Phase2#cell-gene-therapy#CAR-T+2
Trials 1Status Recruiting
CGTAAVLimited DatastaleEmily de los ReyesPhase 2

AAV9.SLC6A1 Gene Therapy (AAV9.SLC6A1 Gene Therapy)

SLC6A1 · SLC6A1

#SLC6A1#CGT#Phase2#cell-gene-therapy#CAR-T+2
Trials 1Status Active
CGTAAVLimited DatastaleThe Children's Hospital of Zhej…Phase 1

AAV9.hMCOLN1co (AAV9.hMCOLN1co)

Mucolipidosis Type IV · Mucolipidosis Type IV

#MucolipidosisTypeIV#CGT#Phase1#cell-gene-therapy#CAR-T+2
Trials 1Status Active
CGTAAVLimited DatastaleBenjamin GreenbergPhase 1

AAV9/CLN7 (AAV9/CLN7)

CLN7 · CLN7

#CLN7#CGT#Phase1#cell-gene-therapy#CAR-T+2
Trials 1Status Recruiting
CGTLimited DatastaleAkouos, Inc.Phase 2

AAVAnc80-hOTOF via Akouos Delivery Device (AAVAnc80-hOTOF via Akouos Delivery Device)

Sensorineural Hearing Loss, Bilateral · Sensorineural Hearing Loss, Bilateral

#SensorineuralHearingLossBilatera#CGT#Phase2#cell-gene-therapy#CAR-T+4
Trials 1Status Recruiting
CGTLimited DatastaleAkouos, Inc.Phase 2

AAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism (AAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism)

Sensorineural Hearing Loss, Bilateral · Sensorineural Hearing Loss, Bilateral

#SensorineuralHearingLossBilatera#CGT#Phase2#cell-gene-therapy#CAR-T+4
Trials 1Status Recruiting
CGTLimited DatastaleJanssen Research & Development,…Phase 1

AAVCAGsCD59 (AAVCAGsCD59)

Dry Age-related Macular Degeneration · Dry Age-related Macular Degeneration

#DryAge-relatedMacularDegeneratio#CGT#Phase1#cell-gene-therapy#CAR-T+2
Trials 1Status Active
CGTAAVLimited DatastaleWeill Medical College of Cornel…Phase 2

AAVrh.10CUCLN2 (AAVrh.10CUCLN2)

Late Infantile Neuronal Ceroid Lipofuscinosis; Batten Disease · Late Infantile Neuronal Ceroid Lipofusci

#LateInfantileNeuronalCeroidLipof#CGT#Phase2#cell-gene-therapy#CAR-T+3
Trials 1Status Active
CGTAAVLimited DatastaleWeill Medical College of Cornel…Phase 1

AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies (AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies)

Batten Disease; Late-Infantile Neuronal Ceroid Lipofuscinosis · Batten Disease

#BattenDisease#CGT#Phase1#cell-gene-therapy#CAR-T+3
Trials 1Status Active

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