Basic Information

Gene symbol RPGR Synonyms COD1, CORDX1, CRD, PCDX, RP15, RP3, XLRP3, orf15 Type of gene protein-coding
Description retinitis pigmentosa GTPase regulator

GTO ID GTC2431
Trial ID NCT04517149
Disease Retinitis Pigmentosa
Altered gene RPGR
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment 4D-125
PhasePhase1|Phase2
Recruitment statusActive, Not Recruiting
TitleAn Open-label, Phase 1/2 Trial of Gene Therapy 4D-125 in Males With X-linked Retinitis Pigmentosa (XLRP) Caused by Mutations in the RPGR Gene
Year2020
CountryUnited States
Company sponsor4D Molecular Therapeutics
Other ID(s)4D-125-C001
Vector information
Vectoradeno-associated virus
ConstructAAV-coRPGR
Vector typeadeno-associated virus (AAV) vector
Transgene/Inserted genecodon-optimized human Retinitis Pigmentosa GTPase Regulator transgene

Clinical Result

Cohort 1
Administration route intravitreal injection
Age Child, Adult, Older_Adult
References DOI: 10.1101/2021.06.24.449775

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