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 GTC2258
Trial ID NCT04176653
Disease Non-Transfusion-Dependent Thalassemia | Myelodysplastic Syndrome
Altered gene TMPRSS6
Therapeutic/Target gene Target gene
TherapysiRNA
Treatment SLN124
PhasePhase1
Recruitment statusWithdrawn
TitlePhase 1b Single Ascending and Multiple Dose First-in-man Study in Adult Patients With Non-transfusion-dependent Beta-thalassaemia or Low Risk Myelodysplastic Syndrome to Investigate the Safety, Tolerability, Pharmacokinetic and Pharmacodynamic Response of SLN124
Year2019
CountryBulgaria|United Kingdom
Company sponsorSilence Therapeutics plc
Other ID(s)SLN124-001
Vector information
VectorGalNAc

Clinical Result

Cohort1: Placebo
Administration route None
Pts 0
Age Child, Adult, Older_Adult
Cohort2: SLN124_dose level 1
Administration route None
Dosage SLN124, 0.3 mg/kg
Pts 0
Age Child, Adult, Older_Adult
Cohort3: SLN124_dose level 2
Administration route None
Dosage SLN124, 1.0 mg/kg
Pts 0
Age Child, Adult, Older_Adult
Cohort4: SLN124_dose level 3
Administration route None
Dosage SLN124, 3.0 mg/kg
Pts 0
Age Child, Adult, Older_Adult
Cohort5: SLN124_dose level 4
Administration route None
Dosage SLN124, 10.0 mg/kg
Pts 0
Age Child, Adult, Older_Adult

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