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 GTC3280
Trial ID NCT05657301
Disease Age-Related Macular Degeneration
Altered gene VEGFA
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment KH631
PhasePhase1
Recruitment statusRecruiting
TitleA Phase I, Open-label, Multicenter, Dose-Escalating Study to Evaluate the Safety and Tolerability of KH631 Gene Therapy in Participants With Neovascular Age-related Macular Degeneration
Year2022
CountryUnited States
Company sponsorChengdu Origen Biotechnology Co., Ltd.
Other ID(s)VAN-2201
Vector information
Vectoradeno-associated virus
ConstructrAAV8-anti-VEGF protein
Vector typerecombinant adeno-associated virus serotype 8 (rAAV8) vector
Transgene/Inserted geneencoding a human VEGF receptor fusion protein
Regulatory elementthe cytomegalovirus (CMV) early enhancer/chicken b-actin (CBA) ubiquitous promoter; a chicken b-actin intron; a rabbit b-globin poly (A) signal
Vector production methodHEK293 cells grown in 293Pro chemically defined 293M medium (BasalMedia) in suspension were triple transfected with pHelper, pAAV transgene (encoding pAAV-CBAanti-VEGF protein49), and pAAV Rep-Cap plasmids using FectoVIR-AAV (Polyplus) in a BIOSTAT STR200 bioreactor (Sartorius).

Clinical Result

Cohort 1
Administration route intraocular injection
Age Adult, Older_Adult
References PMID: 37752703

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