Basic Information

Gene symbol NR2E3 Synonyms ESCS, PNR, RNR, RP37, rd7 Type of gene protein-coding
Gene perturbation-related omics dataset PerturbAtlas | PertOrg
Description nuclear receptor subfamily 2 group E member 3

GTO ID GTC3867
Trial ID NCT06388200
Disease Retinitis Pigmentosa
Altered gene NR2E3
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment OCU400-301
PhasePhase3
Recruitment statusRecruiting
TitleA Phase 3, Multi-Center, Randomized Study to Assess The Efficacy, Safety and Tolerability of Subretinal OCU400 Gene Therapy for the Treatment of Retinitis Pigmentosa
Year2024
CountryUnited States
Company sponsorOcugen
Other ID(s)OCU400-301
Vector information
Vectoradeno-associated virus
ConstructAAV-NR2E3
Vector typeadeno-associated virus (AAV) vector
Transgene/Inserted genehuman NR2E3 gene

Clinical Result

Cohort1: RHO mutations arm
Administration route subretinal injection
Dosage 2.5E10 vg/eye
Pts 75
Age Child, Adult, Older_Adult
Cohort2: Gene agnostic arm
Administration route subretinal injection
Dosage 2.5E10 vg/eye
Pts 75
Age Child, Adult, Older_Adult

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