Basic Information

Gene symbol FLT1 Synonyms FLT, FLT-1, VEGFR-1, VEGFR1 Type of gene protein-coding
Gene perturbation-related omics dataset PerturbAtlas | PertOrg
Description fms related receptor tyrosine kinase 1

GTO ID GTC0090
Trial ID NCT01024998
Disease Age-Related Macular Degeneration
Altered gene FLT1
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment AAV2-sFLT01
PhasePhase1
Recruitment statusCompleted
TitleA Phase 1, Open-Label, Multi-Center, Dose-Escalating, Safety and Tolerability Study of a Single Intravitreal Injection of AAV2-sFLT01 in Patients With Neovascular Age-Related Macular Degeneration
Year2009
CountryUnited States
Company sponsorGenzyme, a Sanofi Company
Other ID(s)sFLT01-AMD-00106|0810-948|MSC12870
Vector information
Vectoradeno-associated virus
ConstructAAV2-sFLT01
Vector typeadeno-associated virus serotype 2 (AAV2) vector
Transgene/Inserted genechimeric VEGF-neutralising protein, consisting of domain 2 of Flt-1 (VEGF receptor 1) linked by a polyglycine 9-mer to human IgG1-Fc (sFLT01)
Regulatory elementa ubiquitous promoter

Clinical Result

Cohort1: dose level 1
Administration route intravitreal injection
Dosage 2E8 vg
Pts 3
Age Adult, Older_Adult
References PMID: 28526489
Cohort2: dose level 2
Administration route intravitreal injection
Dosage 2E9 vg
Pts 3
Age Adult, Older_Adult
References PMID: 28526489
Cohort3: dose level 3
Administration route intravitreal injection
Dosage 6E9 vg
Pts 3
Age Adult, Older_Adult
Adverse reactions 3/3(Conjunctival hyperaemia; Eye pain; Eyelids pruritus)
References PMID: 28526489
Cohort4: dose level 4
Administration route intravitreal injection
Dosage 2E10 vg
Pts 10
Age Adult, Older_Adult
Outcome Five patients had aqueous humour concentrations of sFLT01 that peaked at 32.7-112.0 ng/mL (mean 73.7 ng/mL, SD 30.5) by week 26 with a slight decrease to a mean of 53.2 ng/mL at week 52 (SD 17.1).
Adverse reactions 8/10(Conjunctival haemorrhage; Conjunctival oedema; Eye pain; Eyelids pruritus)
References PMID: 28526489

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