Basic Information

Gene symbol RLBP1 Synonyms CRALBP Type of gene protein-coding
Description retinaldehyde binding protein 1

GTO ID GTC1835
Trial ID NCT03374657
Disease Retinitis Pigmentosa
Altered gene RLBP1
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment CPK850
PhasePhase1|Phase2
Recruitment statusActive, Not Recruiting
TitleAn Open-label First-in-human Single Ascending Dose Study to Explore Safety, Tolerability and Efficacy of Subretinal Administration of CPK850 Gene Therapy in Patients With Retinitis Pigmentosa Due to Mutations in the Retinaldehyde Binding Protein 1 (RLBP1) Gene
Year2017
CountrySweden
Company sponsorNovartis Pharmaceuticals
Other ID(s)CCPK850X2202|2016-002696-10
Vector information
Vectoradeno-associated virus
ConstructscAAV8-RLBP1
Vector typeself-complementary adeno-associated virus serotype 8 (scAAV8) vector
Transgene/Inserted genehuman RLBP1 gene
Regulatory elementa short RLBP1 promoter

Clinical Result

Cohort 1
Administration route subretinal injection
Age Adult, Older_Adult
References PMID: 29359172

Relationship Graph

Overview of Knowledge Graph