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 GTC2574
Trial ID NCT04718844
Disease Non-Transfusion-Dependent Thalassemia | Myelodysplastic Syndrome
Altered gene TMPRSS6
Therapeutic/Target gene Target gene
TherapysiRNA
Treatment SLN124
PhasePhase1
Recruitment statusCompleted
TitleA Randomised, Single-blind, Placebo-controlled, Phase 1, Single-ascending and Multiple-dose Study in Adult Subjects With Alpha/Beta-thalassaemia and Very Low- and Low-risk Myelodysplastic Syndrome to Investigate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Response of SLN124
Year2021
CountryGermany|Israel|Italy|Jordan|Malaysia|Thailand|UnitedKingdom
Company sponsorSilence Therapeutics plc
Other ID(s)SLN124-002
Vector information
VectorGalNAc

Clinical Result

Cohort1: SLN124_dose level 1_Thalassaemia
Administration route subcutaneous injection
Dosage SLN124, 1.0 mg/kg
Age Adult, Older_Adult
Cohort2: SLN124_dose level 2_Thalassaemia
Administration route subcutaneous injection
Dosage SLN124, 3.0 mg/kg
Age Adult, Older_Adult
Cohort3: SLN124_dose level 3_Thalassaemia
Administration route subcutaneous injection
Dosage SLN124, 6.0 mg/kg
Age Adult, Older_Adult
Cohort4: Placebo
Administration route subcutaneous injection
Age Adult, Older_Adult
Cohort5: SLN124_dose level 4_Thalassaemia
Administration route subcutaneous injection
Dosage SLN124, Xmg/kg
Age Adult, Older_Adult
Cohort6: SLN124_dose level 1_Myelodysplastic Syndrome
Administration route subcutaneous injection
Dosage SLN124, 1.0 mg/kg
Age Adult, Older_Adult
Cohort7: SLN124_dose level 2_Myelodysplastic Syndrome
Administration route subcutaneous injection
Dosage SLN124, 3.0 mg/kg
Age Adult, Older_Adult
Cohort8: SLN124_dose level 3_Myelodysplastic Syndrome
Administration route subcutaneous injection
Dosage SLN124, 10.0 mg/kg
Age Adult, Older_Adult
Cohort9: SLN124_dose level 4_Myelodysplastic Syndrome
Administration route subcutaneous injection
Dosage SLN124, Xmg/kg
Age Adult, Older_Adult
Cohort10: SLN124_dose level 2_Thalassaemia_multi dose
Administration route subcutaneous injection
Dosage SLN124, 3.0 mg/kg, multi dose
Age Adult, Older_Adult
Cohort11: SLN124_dose level 3_Thalassaemia_multi dose
Administration route subcutaneous injection
Dosage SLN124, 10.0 mg/kg, multi dose
Age Adult, Older_Adult
Cohort12: SLN124_dose level 4_Thalassaemia_multi dose
Administration route subcutaneous injection
Dosage SLN124, Xmg/kg, multi dose
Age Adult, Older_Adult
Cohort13: SLN124_dose level 2_Myelodysplastic Syndrome_multi dose
Administration route subcutaneous injection
Dosage SLN124, 3.0 mg/kg, multi dose
Age Adult, Older_Adult
Cohort14: SLN124_dose level 3_Myelodysplastic Syndrome_multi dose
Administration route subcutaneous injection
Dosage SLN124, 10.0 mg/kg, multi dose
Age Adult, Older_Adult
Cohort15: SLN124_dose level 4_Myelodysplastic Syndrome_multi dose
Administration route subcutaneous injection
Dosage SLN124, Xmg/kg, multi dose
Age Adult, Older_Adult
Cohort16: Placebo_Thalassaemia_multi dose
Administration route subcutaneous injection
Age Adult, Older_Adult
Cohort17: Placebo_Myelodysplastic Syndrome
Administration route subcutaneous injection
Age Adult, Older_Adult
Cohort18: Placebo_Myelodysplastic Syndrome_multi dose
Administration route subcutaneous injection
Age Adult, Older_Adult

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