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Basic Information Clinical Results Relationship Graph

Basic Information

Gene symbol	TTR	Synonyms	ATTR, CTS, CTS1, HEL111, HsT2651, PALB, TBPA, TTN	Type of gene	protein-coding
Chromosome	18	Map location	18q12.1	dbXrefs
Gene perturbation-related omics dataset	PerturbAtlas
Description	transthyretin

GTO ID	GTC0471
Trial ID	NCT03862807
Disease	Amyloidosis
Altered gene	TTR
Therapeutic/Target gene	Target gene
Therapy	siRNA
Treatment	ALN-TTR02\|ONPATTRO\|patisiran
Location approved	US, EU, UK, Japan, Canada, Switzerland, Brazil, China, Israel, Turkey, Australia
Phase	Phase3
Recruitment status	Completed
Title	An Open-label Study to Evaluate Safety, Efficacy and Pharmacokinetics (PK) of Patisiran-LNP in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) With Disease Progression Post-Orthotopic Liver Transplant
Year	2019
Country	France\|Germany\|Italy\|Portugal\|Spain\|Sweden\|United Kingdom
Company sponsor	Alnylam Pharmaceuticals
Other ID(s)	ALN-TTR02-008\|2018-003519-24

Vector information

Vector	lipid nanoparticle

Clinical Result

Cohort 1

Administration route	intravenous infusion
Dosage	patisiran, 0.3 mg/kg, once every 3 weeks (q3w) for 12 months; participants weighing 100 kg or more: patisiran, total dose of 30 mg q3w
Pts	24
Age	Adult, Older_Adult
Outcome	Average of Month 6 and Month 12 Percentage Reduction From Baseline in Serum Transthyretin (TTR):Median (95% Confidence Interval):91.0(86.1 to 92.3)
Adverse reactions	0/23(All-cause mortality); 5/23(Cardiac disorders; Hepatobiliary disorders; Immune system disorders; Infections and infestations; Injury, poisoning and procedural complications; Nervous system disorders; Renal and urinary disorders; Respiratory, thoracic and mediastinal disorders)
References	PMID: 35213769

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