Basic Information

Gene symbol LDLR Synonyms FH, FHC, FHCL1, LDLCQ2 Type of gene protein-coding
Gene perturbation-related omics dataset PerturbAtlas | PertOrg
Description low density lipoprotein receptor

GTO ID GTC2790
Trial ID NCT05043181
Disease Familial Hypercholesterolemia
Altered gene LDLR
Therapeutic/Target gene Therapeutic gene
TherapymRNA
Treatment Low Density Lipoprotein Receptor mRNA Exosomes
PhasePhase1
Recruitment statusNot Recruiting
TitleExosome-based Nanoplatform for Ldlr mRNA Delivery in Familial Hypercholesterolemia
Year2021
CountryChina
Company sponsorTang-Du Hospital
Other ID(s)20210817V2.0
Vector information
VectorExosomes

Clinical Result

Cohort1: Dose escalation phase_dose level 1
Administration route intravenous|peritoneal infusion
Dosage Low Density Lipoprotein Receptor mRNA Exosomes, 0.044 mg/kg
Pts 3
Age Adult
Cohort2: Dose escalation phase_dose level 2
Administration route intravenous|peritoneal infusion
Dosage Low Density Lipoprotein Receptor mRNA Exosomes, 0.088 mg/kg
Pts 3
Age Adult
Cohort3: Dose escalation phase_dose level 3
Administration route intravenous|peritoneal infusion
Dosage Low Density Lipoprotein Receptor mRNA Exosomes, 0.145 mg/kg
Pts 3
Age Adult
Cohort4: Dose escalation phase_dose level 4
Administration route intravenous|peritoneal infusion
Dosage Low Density Lipoprotein Receptor mRNA Exosomes, 0.220 mg/kg
Pts 3
Age Adult
Cohort5: Dose escalation phase_dose level 5
Administration route intravenous|peritoneal infusion
Dosage Low Density Lipoprotein Receptor mRNA Exosomes, 0.295 mg/kg
Pts 3
Age Adult
Cohort6: Dose escalation phase_dose level 6
Administration route intravenous|peritoneal infusion
Dosage Low Density Lipoprotein Receptor mRNA Exosomes, 0.394 mg/kg
Pts 3
Age Adult
Cohort7: Extension phase
Administration route intravenous|peritoneal infusion
Pts 12
Age Adult

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