Basic Information

Gene symbol PDE6B Synonyms CSNB3, CSNBAD2, GMP-PDEbeta, PDEB, RP40, rd1 Type of gene protein-coding
Description phosphodiesterase 6B

GTO ID GTC1812
Trial ID NCT03328130
Disease Retinitis Pigmentosa
Altered gene PDE6B
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment AAV2/5-hPDE6B
PhasePhase1|Phase2
Recruitment statusRecruiting
TitleSafety and Efficacy of a Unilateral Subretinal Administration of HORA-PDE6B in Patients With Retinitis Pigmentosa Harbouring Mutations in the PDE6B Gene Leading to a Defect in PDE6β Expression
Year2017
CountryUnited States
Company sponsoreyeDNA Therapeutics
Other ID(s)HORA-PDE6B-001|2016-001429-16
Vector information
Vectoradeno-associated virus
ConstructAAV2/5-hPDE6B
Vector typeadeno-associated virus serotype 2/5 (AAV2/5) vector
Transgene/Inserted genethe normal human PDE6B gene

Clinical Result

Cohort 1
Administration route subretinal injection
Pts 23
Age Child, Adult, Older_Adult

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