Basic Information

Gene symbol CNGA1 Synonyms CNCG, CNCG1, CNG-1, CNG1, RCNC1, RCNCa, RCNCalpha, RP49 Type of gene protein-coding
Gene perturbation-related omics dataset PerturbAtlas
Description cyclic nucleotide gated channel subunit alpha 1

GTO ID GTC3784
Trial ID NCT06291935
Disease Retinitis Pigmentosa
Altered gene CNGA1
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment VG901
PhasePhase1
Recruitment statusRecruiting
TitleA Prospective, Open-label, Phase 1b, Single-arm, Safety Study of an Intravitreal Application of a Recombinant Adeno-associated Virus Vector Expressing CNGA1 (AAV2.NN-CNGA1) in Patients With Retinitis Pigmentosa Due to CNGA1 Mutations
Year2024
CountryGermany
Company sponsorViGeneron GmbH
Other ID(s)VG901-2021 A|EU CT: 2023-504383-42-00
Vector information
Vectoradeno-associated virus
ConstructAAV2.NN-CNGA1
Vector typeadeno-associated virus serotype 2 (AAV2.NN) vector
Transgene/Inserted geneCNGA1 gene

Clinical Result

Cohort 1
Administration route intravitreal injection
Pts 6
Age Adult, Older_Adult

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