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Basic Information Clinical Results Relationship Graph

Basic Information

Gene symbol	TMPRSS6	Synonyms	IRIDA, MT2	Type of gene	protein-coding
Chromosome	22	Map location	22q12.3	dbXrefs
Gene perturbation-related omics dataset	PertOrg
Description	transmembrane serine protease 6

GTO ID	GTC1734
Trial ID	NCT03165864
Disease	Thalassemia
Altered gene	TMPRSS6
Therapeutic/Target gene	Target gene
Therapy	ASO
Treatment	Sapablursen\|ISIS 702843\|IONIS-TMPRSS6-LRx
Phase	Phase1
Recruitment status	Completed
Title	A Double-Blind, Placebo-Controlled, Dose-Escalation, Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ISIS 702843 Administered Subcutaneously to Healthy Volunteers
Year	2017
Country	Australia
Company sponsor	Ionis Pharmaceuticals, Inc.
Other ID(s)	ISIS 702843-CS1

Vector information

Vector	GalNAc3

Clinical Result

Cohort1: Sapablursen_dose level1

Administration route	subcutaneous injection
Dosage	Sapablursen, single dose, 20 mg, on day 1, followed by three additional doses of the same strength on days 22, 36, and 50
Pts	9
Age	Adult, Older_Adult
References	PMID: 36690839

Cohort2: Sapablursen_dose level2

Administration route	subcutaneous injection
Dosage	Sapablursen, single dose, 40 mg, on day 1, followed by three additional doses of the same strength on days 22, 36, and 50
Pts	9
Age	Adult, Older_Adult
References	PMID: 36690839

Cohort3: Sapablursen_dose level3

Administration route	subcutaneous injection
Dosage	Sapablursen, single dose, 60 mg, on day 1, followed by three additional doses of the same strength on days 22, 36, and 50
Pts	9
Age	Adult, Older_Adult
References	PMID: 36690839

Cohort4: Placebo

Administration route	subcutaneous injection
Pts	9
Age	Adult, Older_Adult
References	PMID: 36690839

Relationship Graph

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