Basic Information

Gene symbol ABCA4 Synonyms ABC10, ABCR, ARMD2, CORD3, FFM, RMP, RP19, STGD, STGD1 Type of gene protein-coding
Gene perturbation-related omics dataset PerturbAtlas | PertOrg
Description ATP binding cassette subfamily A member 4

GTO ID GTC3794
Trial ID NCT06300476
Disease Stargardt Disease 1
Altered gene ABCA4
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment JWK006
PhasePhase1|Phase2
Recruitment statusActive, Not Recruiting
TitleSafety and Efficacy of a Single Subretinal Injection of JWK006 Gene Therapy in Subjects With Stargardt Disease(STGD1)
Year2024
CountryChina
Company sponsorWest China Hospital
Other ID(s)2023-1511
Vector information
Vectoradeno-associated virus
ConstructAAV-ABCA4
Vector typeadeno-associated virus (AAV) vector
Transgene/Inserted genehuman ABCA4 gene

Clinical Result

Cohort 1
Administration route subretinal injection
Pts 9
Age Child, Adult

Relationship Graph

Overview of Knowledge Graph