CGT

5개 · ADC 890 · Antibody 4097 · CGT 2985 · 다음 갱신 D-6 · 마지막 9월 2일

PopularRecently updatedCuratedMost trialsNeeds refreshORRLatestPhase 3FDA approved5 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
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
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