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 GTC3227
Trial ID NCT05607810
Disease Diabetic Macular Edema
Altered gene VEGFA
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment ADVM-022
Recruitment statusRecruiting
TitleA Long-Term Follow-Up Study of ADVM-022 in Diabetic Macular Edema- INFINITY Extension
Year2022
CountryPuerto Rico|United States
Company sponsorAdverum Biotechnologies, Inc.
Other ID(s)ADVM-022-10
Vector information
Vectoradeno-associated virus
ConstructAAV2.7m8-C11.CO.aflibercept
Vector typerecombinant, replication-deficient adeno-associated virus (AAV.7m8) 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

Cohort 1
Administration route intravitreal injection
Age Adult, Older_Adult
References PMID: 30528929

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