Basic Information

Gene symbol PDE6A Synonyms CGPR-A, PDEA, RP43 Type of gene protein-coding
Description phosphodiesterase 6A

GTO ID GTC2495
Trial ID NCT04611503
Disease Retinitis Pigmentosa
Altered gene PDE6A
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment rAAV-hPDE6A
PhasePhase1|Phase2
Recruitment statusActive, Not Recruiting
TitlePIGMENT - PDE6A Gene Therapy for Retinitis Pigmentosa
Year2020
CountryGermany
Company sponsorSTZ eyetrial
Other ID(s)RDC-PDE6A-01
Vector information
Vectoradeno-associated virus
ConstructrAAV8.hPDE6A
Vector typerecombinant adeno-associated virus serotype 8 (rAAV8) vector
Transgene/Inserted genehuman PDE6A gene
Regulatory elementthe GRK1 promoter

Clinical Result

Cohort1: dose level 1
Administration route subretinal injection
Dosage 1E9 vg
Age Adult, Older_Adult
References PMID: 38955952
Cohort2: dose level 2
Administration route subretinal injection
Dosage 1E10 vg
Age Adult, Older_Adult
References PMID: 38955952
Cohort3: dose level 3
Administration route subretinal injection
Dosage 5E10 vg
Age Adult, Older_Adult
References PMID: 38955952

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