CGT

1,163개 · ADC 890 · Antibody 4071 · CGT 2714 · 다음 갱신 D-4 · 마지막 9월 2일

PopularRecently updatedCuratedMost trialsNeeds refreshORRLatestPhase 3FDA approved1,163 programs
CGTLimited DatastaleUniversity of OxfordPhase 2

rAAV2.REP1 (rAAV2.REP1)

Choroideremia · Choroideremia

#Choroideremia#CGT#Phase2#cell-gene-therapy#CAR-T+2
Trials 1Status Active
CGTLimited DatastaleUniversity of AlbertaPhase 2

rAAV2.REP1 vector (rAAV2.REP1 vector)

Choroideremia · Choroideremia

#Choroideremia#CGT#Phase2#cell-gene-therapy#CAR-T+2
Trials 1Status Active
CGTLimited DatastaleNantes University HospitalPhase 2

rAAV2/4.hRPE65 (rAAV2/4.hRPE65)

Leber Congenital Amaurosis · Leber Congenital Amaurosis

#LeberCongenitalAmaurosis#CGT#Phase2#cell-gene-therapy#CAR-T+2
Trials 1Status Active
CGTLimited DatastaleDigna Biotech S.L.Phase 1

rAAV2/5-PBGD vector dosage 1 (rAAV2/5-PBGD vector dosage 1)

Acute Intermittent Porphyria · Acute Intermittent Porphyria

#AcuteIntermittentPorphyria#CGT#Phase1#cell-gene-therapy#CAR-T+2
Trials 1Status Active
CGTLimited DatastaleDigna Biotech S.L.Phase 1

rAAV2/5-PBGD vector dosage 2 (rAAV2/5-PBGD vector dosage 2)

Acute Intermittent Porphyria · Acute Intermittent Porphyria

#AcuteIntermittentPorphyria#CGT#Phase1#cell-gene-therapy#CAR-T+2
Trials 1Status Active
CGTLimited DatastaleDigna Biotech S.L.Phase 1

rAAV2/5-PBGD vector dosage 3 (rAAV2/5-PBGD vector dosage 3)

Acute Intermittent Porphyria · Acute Intermittent Porphyria

#AcuteIntermittentPorphyria#CGT#Phase1#cell-gene-therapy#CAR-T+2
Trials 1Status Active
CGTLimited DatastaleDigna Biotech S.L.Phase 1

rAAV2/5-PBGD vector dosage 4 (rAAV2/5-PBGD vector dosage 4)

Acute Intermittent Porphyria · Acute Intermittent Porphyria

#AcuteIntermittentPorphyria#CGT#Phase1#cell-gene-therapy#CAR-T+2
Trials 1Status Active
CGTLimited DatastaleUniQure Biopharma B.V.Phase 2

rAAV2/5-hNAGLU (rAAV2/5-hNAGLU)

Sanfilippo Syndrome B · Sanfilippo Syndrome B

#SanfilippoSyndromeB#CGT#Phase2#cell-gene-therapy#CAR-T+2
Trials 1Status Active
CGTLimited DatastaleBeacon TherapeuticsPhase 2

rAAV2tYF-CB-hRS1 (rAAV2tYF-CB-hRS1)

X-linked Retinoschisis · X-linked Retinoschisis

#X-linkedRetinoschisis#CGT#Phase2#cell-gene-therapy#CAR-T+2
Trials 1Status Active
CGTLimited DatastaleBeacon TherapeuticsPhase 3

rAAV2tYF-GRK1-hRPGRco (rAAV2tYF-GRK1-hRPGRco)

X-Linked Retinitis Pigmentosa · X-Linked Retinitis Pigmentosa

#X-LinkedRetinitisPigmentosa#CGT#Phase3#cell-gene-therapy#CAR-T+2
Trials 1Status Recruiting
CGTLimited DatastaleBeacon TherapeuticsPhase 2

rAAV2tYF-PR1.7-hCNGB3 (rAAV2tYF-PR1.7-hCNGB3)

Achromatopsia · Achromatopsia

#Achromatopsia#CGT#Phase2#cell-gene-therapy#CAR-T+2
Trials 1Status Active
CGTLimited DatastaleEunice Kennedy Shriver National…Phase 1

recombinant adenovirus containing the ornithine transcarbamylase gene (recombinant adenovirus containing the ornithine transcarbamylase gene)

Ornithine Transcarbamylase Deficiency Disease · Ornithine Transcarbamylase Deficiency Di

#OrnithineTranscarbamylaseDeficie#CGT#Phase1#cell-gene-therapy#CAR-T+2
Trials 1Status Discontinued
CGTLimited DatastaleNationwide Children's HospitalPhase 2

scAAV1.tMCK.NTF3 (scAAV1.tMCK.NTF3)

Charcot-Marie-Tooth Neuropathy Type 1A · Charcot-Marie-Tooth Neuropathy Type 1A

#Charcot-Marie-ToothNeuropathyTyp#CGT#Phase2#cell-gene-therapy#CAR-T+2
Trials 1Status Recruiting
CGTLimited DatastaleUltragenyx Pharmaceutical IncPhase 2

scAAV8OTC (scAAV8OTC)

Ornithine Transcarbamylase (OTC) Deficiency · Ornithine Transcarbamylase (OTC) Deficie

#OrnithineTranscarbamylaseOTCDefi#CGT#Phase2#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
CGTLimited DatastaleRare Trait HopePhase 2

scAAV9/AGA (scAAV9/AGA)

Aspartylglucosaminuria; Aspartylglucosamidase (AGA) Deficiency · Aspartylglucosaminuria

#Aspartylglucosaminuria#CGT#Phase2#cell-gene-therapy#CAR-T+3
Trials 1Status Recruiting
CGTLimited DatastaleThe First Affiliated Hospital o…Phase 2

ssCART-19 cells combined with CD19+ feeding T cells (FTCs, ssCART-19 cells combined with CD19+ feeding T cells, ssCART-19 cells combined with CD19+ feeding T cells (FTCs) infusion)

Acute Lymphoblastic Leukemia, Adult B-Cell · CD19

#CD19#CGT#Phase2#cell-gene-therapy#CAR-T+4
Trials 1Status Recruiting
CGTLimited DatastaleUniversity College, LondonPhase 2

rAAV 2/2.hRPE65p.hRPE65 (tgAAG76, tgAAG76 (rAAV 2/2.hRPE65p.hRPE65))

Retinal Degeneration · Retinal Degeneration

#RetinalDegeneration#CGT#Phase2#cell-gene-therapy#CAR-T+2
Trials 1Status Active
CGTLimited DatastaleTargeted Genetics CorporationPhase 3

tgAAVCF (tgAAVCF)

Cystic Fibrosis · Cystic Fibrosis

#CysticFibrosis#CGT#Phase3#cell-gene-therapy#CAR-T+2
Trials 1Status Discontinued
CGTLimited DatastaleInstitute of Hematology & Blood…Phase 1

uCD7 CART (uCD7 CART)

Acute Myeloid Leukemia (AML); T Lymphoblastic Leukemia/Lymphoma · Acute Myeloid Leukemia (AML)

#AcuteMyeloidLeukemiaAML#CGT#Phase1#cell-gene-therapy#CAR-T+4
Trials 1Status Recruiting

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