Basic Information

Gene symbol VEGFA Synonyms L-VEGF, MVCD1, VEGF, VPF Type of gene protein-coding
Gene perturbation-related omics dataset PerturbAtlas | PertOrg
Description vascular endothelial growth factor A

GTO ID GTC0492
Trial ID NCT03999801
Disease Age-Related Macular Degeneration
Altered gene VEGFA
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment RGX-314
PhasePhase2
Recruitment statusRecruiting
TitleA Long-term Follow-Up Study to Evaluate the Safety and Efficacy of RGX-314 Following Subretinal Administration in Participants With Neovascular Age-related Macular Degeneration and Fellow Eye Treatment Substudy
Year2019
CountryUnited States
Company sponsorAbbVie
Other ID(s)RGX-314-5101
Vector information
Vectoradeno-associated virus
ConstructrAAV8-anti-VEGF protein
Vector typerecombinant adeno-associated virus serotype 8 (rAAV8) vector
Transgene/Inserted genea coding sequence for a soluble anti-VEGF protein

Clinical Result

Cohort1: dose level 1
Administration route subretinal injection
Dosage 1.6E11 GC/eye
Pts 12
Age Adult, Older_Adult
Outcome improved visual acuity and decreased retinal thickness, with a mean BCVA change of +4 letters and a mean change in CRT of -68μm after one-time administration of RGX-314. The 9 subjects who were anti-VEGF injection-free after administration of RGX-314 showed a mean BCVA improvement of +5 letters, and a mean improvement in CRT of -80μm.
References PMID: 35017696
Cohort2: dose level 2
Administration route subretinal injection
Dosage 2.5E11 GC/eye
Pts 12
Age Adult, Older_Adult
Outcome maintained visual acuity and decreased retinal thickness, with a mean BCVA change of +2 letters, and a mean change in CRT of -42 μm. The 5 subjects who did not receive anti-VEGF injections after administration of RGX-314 showed a mean BCVA change of +2 letters, and a mean improvement in CRT of -61μm.
References PMID: 35017696

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