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 GTC2019
Trial ID NCT03748784
Disease Age-Related Macular Degeneration
Altered gene VEGFA
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment ADVM-022
PhasePhase1
Recruitment statusCompleted
TitleAn Open Label Phase 1 Study of ADVM-022 (AAV.7m8-aflibercept) in Neovascular (Wet) Age-Related Macular Degeneration
Year2018
CountryUnited States
Company sponsorAdverum Biotechnologies, Inc.
Other ID(s)ADVM-022-01
Vector information
Vectoradeno-associated virus
ConstructAAV2.7m8-C11.CO.aflibercept
Vector typeadeno-associated virus serotype 2 (AAV2) vector
Transgene/Inserted genethe codon-optimized aflibercept cDNA expression cassette, consisting of a recombinant chimeric protein comprising the VEGFA-binding portions of the extracellular domains of human VEGFA receptors 1 and 2 and the fragment crystallizable region (Fc) portion of human immunoglobulin (IgG1)
Regulatory elementthe human cytomegalovirus (CMV) immediate-early enhancer and promoter; an enhancer element from the major late promoter (eMLP); a synthetic intron; a Kozak sequence driving expression of aflibercept; a human scaffold attachment region (SAR); the human growth hormone (GH) polyadenylation site
Vector production methodADVM-022 was produced in the baculovirus expression system in Sf9 cells where two baculoviruses were used, one encoding the genes for AAV2 Rep and AAV2.7m8 Cap proteins, and the other encoding the vector genome carrying the codon-optimized aflibercept cDNA expression cassette.

Clinical Result

Cohort1: dose level 1
Administration route intravitreal injection
Dosage 6E11 vg
Age Adult, Older_Adult
References PMID: 30528929
Cohort2: dose level 2
Administration route intravitreal injection
Dosage 2E11 vg
Age Adult, Older_Adult
References PMID: 30528929

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