Basic Information

Gene symbol RPE65 Synonyms BCO3, LCA2, RP20, mRPE65, p63, rd12, sRPE65 Type of gene protein-coding
Description retinoid isomerohydrolase RPE65

GTO ID GTC1930
Trial ID NCT03597399
Disease Retinal Dystrophy
Altered gene RPE65
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment voretigene neparvovec|Luxturna
Recruitment statusActive, Not Recruiting
TitleA Post-Authorization, Multicenter, Longitudinal, Observational Safety Registry Study for Patients Treated With Voretigene Neparvovec in US
Year2018
CountryUnited States
Company sponsorSpark Therapeutics, Inc.
Other ID(s)SPKRPE-PASS
Vector information
Vectoradeno-associated virus
ConstructAAV2-hRPE65v2
Vector typeadeno-associated virus serotype 2 (AAV2) vector
Transgene/Inserted genehuman RPE65 cDNA with a modified Kozak sequence engineered at the translational start site
Regulatory elementa hybrid chicken β-actin promoter; a cytomegalovirus enhancer

Clinical Result

Cohort 1
Administration route subretinal injection
Pts 87
Age Child, Adult, Older_Adult
References PMID: 38254722

Relationship Graph

Overview of Knowledge Graph