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 GTC0359
Trial ID NCT03066258
Disease Age-Related Macular Degeneration
Altered gene VEGFA
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment RGX-314
PhasePhase1|Phase2
Recruitment statusCompleted
TitleA Phase I/IIa (Phase 1/Phase 2a), Open-label, Multiple-cohort, Dose-escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With RGX-314 in Subjects With Neovascular AMD (nAMD)
Year2017
CountryUnited States
Company sponsorREGENXBIO Inc.
Other ID(s)RGX-314-001
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 3E9 GC/eye
Pts 5
Age Adult, Older_Adult
Outcome The change from baseline in Best Corrected Visual Acuity (BCVA) had a mean of -7.6 with a full range from -16 to 6. Additionally, the change from baseline in Central Retinal Thickness (CRT) had a mean of -88.6 with a full range from -248.0 to 11.0.
Adverse reactions 2/6(Infections and infestations; Metabolism and nutrition disorders; Musculoskeletal and connective tissue disorders; Respiratory, thoracic and mediastinal disorders)
References PMID: 35017696
Cohort2: dose level 2
Administration route subretinal injection
Dosage 1E10 GC/eye
Pts 6
Age Adult, Older_Adult
Outcome The change from baseline in Best Corrected Visual Acuity (BCVA) had a mean of 1.2 with a full range from -9 to 8. Additionally, the change from baseline in Central Retinal Thickness (CRT) had a mean of 25.3 with a full range from -10.0 to 110.0.
Adverse reactions 2/6(Cardiac disorders; Injury, poisoning and procedural complications; Musculoskeletal and connective tissue disorders)
References PMID: 35017696
Cohort3: dose level 3
Administration route subretinal injection
Dosage 6E10 GC/eye
Pts 6
Age Adult, Older_Adult
Outcome The change from baseline in Best Corrected Visual Acuity (BCVA) had a mean of 14.0 with a full range from -3 to 32. Additionally, the change from baseline in Central Retinal Thickness (CRT) had a mean of 2.0 with a full range from -46.0 to 142.0.
Adverse reactions 1/6(Eye disorders)
References PMID: 35017696
Cohort4: dose level 4
Administration route subretinal injection
Dosage 1.6E11 GC/eye
Pts 12
Age Adult, Older_Adult
Outcome The change from baseline in Best Corrected Visual Acuity (BCVA) had a mean of 0.9 with a full range from -45 to 17. Additionally, the change from baseline in Central Retinal Thickness (CRT) had a mean of -56.7 with a full range from -170.0 to 160.0.
Adverse reactions 3/12(Hepatobiliary disorders; Infections and infestations; Injury, poisoning and procedural complications)
References PMID: 35017696
Cohort5: dose level 5
Administration route subretinal injection
Dosage 2.5E11 GC/eye
Pts 11
Age Adult, Older_Adult
Outcome The change from baseline in Best Corrected Visual Acuity (BCVA) had a mean of -3.8 with a full range from -37 to 8. Additionally, the change from baseline in Central Retinal Thickness (CRT) had a mean of -108.2 with a full range from -360.0 to 85.0.
Adverse reactions 4/12(Eye disorders; Gastrointestinal disorders; General disorders; Infections and infestations; Nervous system disorders; Psychiatric disorders; Vascular disorders)
References PMID: 35017696

Relationship Graph

Overview of Knowledge Graph