Basic Information

Gene symbol S100A1 Synonyms S100, S100-alpha, S100A Type of gene protein-coding
Gene perturbation-related omics dataset PerturbAtlas
Description S100 calcium binding protein A1

Gene symbol CXCL12 Synonyms IRH, PBSF, SCYB12, SDF1, TLSF, TPAR1 Type of gene protein-coding
Gene perturbation-related omics dataset PerturbAtlas | PertOrg
Description C-X-C motif chemokine ligand 12

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 GTC0415
Trial ID NCT03409627
Disease Heart Failure
Altered gene SDF1|VEGFA|S100A1
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment INXN-4001
PhasePhase1
Recruitment statusCompleted
TitleA Phase I, Open Label, Safety Study of INXN-4001 Delivered Via Retrograde Coronary Sinus Infusion in Patients With an Outpatient Left Ventricular Assist Device (LVAD)
Year2017
CountryUnited States
Company sponsorTriple-Gene, LLC
Other ID(s)INXN-4001-001
Vector information
Vectorplasmid

Clinical Result

Cohort1: dose level 1
Administration route retrograde coronary sinus infusion
Dosage 80 mg in 40mL volumes
Pts 6
Age Adult, Older_Adult
Outcome Four patients improvement by at least five points in total KCCQ score, a validated tool qualified by the FDA for Clinical Outcome Assessment.
References : Precigen Triple-Gene Provides Six-month Follow-up Data from Phase I Study of INXN-4001, a Multigenic Investigational Therapeutic Candidate for Heart Failure
Cohort2: dose level 2
Administration route retrograde coronary sinus infusion
Dosage 80 mg in 80mL volumes
Pts 6
Age Adult, Older_Adult
Outcome One patients improvement by at least five points in total KCCQ score, a validated tool qualified by the FDA for Clinical Outcome Assessment.
References : Precigen Triple-Gene Provides Six-month Follow-up Data from Phase I Study of INXN-4001, a Multigenic Investigational Therapeutic Candidate for Heart Failure

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