Basic Information

Gene symbol TMPRSS6 Synonyms IRIDA, MT2 Type of gene protein-coding
Gene perturbation-related omics dataset PertOrg
Description transmembrane serine protease 6

GTO ID GTC2464
Trial ID NCT04559971
Disease Myeloproliferative Neoplasm | Iron-Loading Anemias
Altered gene TMPRSS6
Therapeutic/Target gene Target gene
TherapysiRNA
Treatment SLN124
PhaseEarly_Phase1
Recruitment statusCompleted
TitleA Randomised, Double-blind, Placebo-controlled Phase 1 Single Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered SLN124 in Healthy Volunteers
Year2020
CountryUnited Kingdom
Company sponsorSilence Therapeutics plc
Other ID(s)SLN124-003
Vector information
VectorGalNAc

Clinical Result

Cohort1: SLN124_dose level 1
Administration route subcutaneous injection
Dosage SLN124, 1.0 mg/kg
Pts 6
Age Adult
References PMID: 37497888
Cohort2: Placebo
Administration route subcutaneous injection
Pts 6
Age Adult
References PMID: 37497888
Cohort3: SLN124_dose level 2
Administration route subcutaneous injection
Dosage SLN124, 3.0 mg/kg
Pts 6
Age Adult
References PMID: 37497888
Cohort4: SLN124_dose level 3
Administration route subcutaneous injection
Dosage SLN124, 4.5 mg/kg
Pts 6
Age Adult
References PMID: 37497888

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