CGT · 황반변성·망막

11개 · ADC 890 · Antibody 4071 · CGT 2714 · 다음 갱신 D-5 · 마지막 9월 2일

PopularRecently updatedCuratedMost trialsNeeds refreshORRLatestPhase 3FDA approved11 programs
CGTAAVCurated CoreFDASpark TherapeuticsApproved

voretigene neparvovec (Luxturna, AAV2-hRPE65v2, voretigene neparvovec-rzyl)

Inherited retinal dystrophy due to confirmed biallelic RPE65 mutations · RPE65 (AAV2)

#RPE65AAV2#CGT#Approved#FDA-approved#BioJungboMoa-pick+5
Trials 7Status Approved
CGTCurated CorestaleREGENXBIO Inc.Phase 2

RGX-314 (RGX-314)

Neovascular Age-related Macular Degeneration; Wet Age-related Macular Degeneration · Neovascular Age-related Macular Degenera

#NeovascularAge-relatedMacularDeg#CGT#Phase2#BioJungboMoa-pick#cell-gene-therapy+4
Trials 12Status Active
CGTLimited DatastaleFrontera TherapeuticsPhase 2

FT-003 (FT-003)

Diabetic Macular Edema · Neovascular Age-related Macular Degenera

#NeovascularAge-relatedMacularDeg#CGT#Phase2#cell-gene-therapy#CAR-T+4
Trials 4Status Recruiting
CGTLimited DatastaleNanoscope Therapeutics Inc.Phase 2

Gene Therapy product:vMCO-I (Gene Therapy product:vMCO-I)

Retinitis Pigmentosa; Retinal Diseases · Retinitis Pigmentosa

#RetinitisPigmentosa#CGT#Phase2#cell-gene-therapy#CAR-T+3
Trials 2Status Active
CGTAAVLimited DatastaleGenentech, Inc.Preclinical

AAV2-hRPE65v2 (AAV2-hRPE65v2)

Inherited Retinal Dystrophy Due to RPE65 Mutations · Multi-target

#Multi-target#CGT#Preclinical#cell-gene-therapy#CAR-T+3
Trials 1Status Recruiting
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
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 DatastaleElisigen, Inc.Phase 2

NG101 AAV gene therapy (NG101 AAV gene therapy)

Age-Related Macular Degeneration · Age-Related Macular Degeneration

#Age-RelatedMacularDegeneration#CGT#Phase2#cell-gene-therapy#CAR-T+2
Trials 1Status Recruiting
CGTLimited DatastaleLions Eye Institute, Perth, Wes…Phase 2

rAAV.sFlt-1 (rAAV.sFlt-1)

Macular Degeneration; Age-related Maculopathies · Macular Degeneration

#MacularDegeneration#CGT#Phase2#cell-gene-therapy#CAR-T+3
Trials 1Status Active
CGTLimited DatastaleUniversity of PennsylvaniaPhase 1

rAAV2-CBSB-hRPE65 (rAAV2-CBSB-hRPE65)

Amaurosis of Leber; Retinal Diseases · Amaurosis of Leber

#AmaurosisofLeber#CGT#Phase1#cell-gene-therapy#CAR-T+3
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