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Transthyretin

Transthyretin Overview

English Name	Transthyretin	Approved Drugs	7
Drugs in Clinical Trials	8	Target Synonym	TTR，Transthyretin，Prealbumin， Amyloidosis Type I，PALB，ATTR，TBPA，Epididymis Luminal Protein 111，Thyroxine-Binding Prealbumin，Carpal Tunnel Syndrome 1，Prealbumin，HsT2651，HEL111，CTS1，CTS
Highest Development Stage	Approved

English Name	Transthyretin
Target Synonym	TTR，Transthyretin，Prealbumin， Amyloidosis Type I，PALB，ATTR，TBPA，Epididymis Luminal Protein 111，Thyroxine-Binding Prealbumin，Carpal Tunnel Syndrome 1，Prealbumin，HsT2651，HEL111，CTS1，CTS
Approved Drugs	7
Drugs in Clinical Trials	8
Highest Development Stage	Approved

Transthyretin Product List

Product Category
Protein (1)
Product Status
Launched (1)
Species
Human (1)
Tag
His Tag (1)
Conjugate
Unconjugated (1)

Cat. No.	Species	Host	Product Description	Structure	Purity	Feature
TTR-H5223	Human	HEK293	Human Transthyretin / Prealbumin Protein, His Tag

Total: 1

Transthyretin Background

Transthyretin (TTR) is also known as Prealbumin, ATTR, TBPA, PALB, which belongs to the transthyretin family. Transthyretin / TTR is a serum and cerebrospinal fluid carrier of the thyroid hormone thyroxine (T4) and retinol-binding protein bound to retinol. In cerebrospinal fluid TTR is the primary carrier of T4. TTR also acts as a carrier of retinol (vitamin A) through its association with retinol-binding protein (RBP) in the blood and the CSF. Less than 1% of TTR's T4 binding sites are occupied in blood. TTR misfolding and aggregation is known to be associated with the amyloid diseases.

Transthyretin Synonyms

Transthyretin,TTR,Prealbumin,TBPA,ATTR,PALB,CTS,CTS1,HsT2651

Clinical Drug Information

English Name	Research Code	Highest Development Stage	Company	Indication	Clinical Trial
Nucresiran	ALNTTRsc-04	Phase 3 Clinical	Alnylam Pharmaceuticals Inc	Cardiomyopathies; Transthyretin amyloidosis	Details
Florbetaben	LNTH-2515	Phase 3 Clinical	Arcus Biosciences Inc	Immunoglobulin Light-chain Amyloidosis; Transthyretin amyloidosis	Details
Cliramitug	ALXN2220; ALXN-2220; NI006; NI-006	Phase 3 Clinical	Neurimmune Ag	Cardiomyopathies; Transthyretin amyloidosis	Details
Coramitug	NNC6019-0001; NNC-6019; PRX-004; NN6019	Phase 3 Clinical	Novo Nordisk A/S	Cardiomyopathies; Transthyretin amyloidosis	Details
Nexiguran ziclumeran	NTLA-2001	Phase 3 Clinical	Intellia Therapeutics Inc	Amyloid Neuropathies; Amyloid Neuropathies, Familial; Amyloidosis; Amyloidosis, Familial; Cardiomyopathies; Genetic Diseases, Inborn; Heredodegenerative Disorders, Nervous System; Metabolic Diseases; Metabolism, Inborn Errors; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Diseases; Peripheral Nervous System Diseases; Polyneuropathies; Transthyretin amyloidosis	Details
ART-001	ART-001; ART001	Phase 2 Clinical	Ruizheng Gene (Suzhou) Co Ltd	Cardiomyopathies; Transthyretin amyloidosis	Details
YOLT-201	YOLT-201	Phase 2 Clinical	Shanghai Yaotang Biotechnology Co Ltd	Cardiomyopathies; Genetic Diseases, Inborn; Polyneuropathies; Transthyretin amyloidosis	Details
BPR-30221616	BPR-30221616; BPR30221616	Phase 1 Clinical	Chengdu Brilliant Pharmaceutical Co Ltd	Cardiomyopathies; Transthyretin amyloidosis	Details

Marketed Drug Information

English Name	Research Code	Highest Development Stage	Company	Earliest Brand Name	Earliest Approval Country	Earliest Indication	Earliest Approval Company	Earliest Approval Date	Indication	Clinical Trial
Patisiran	SAR-438037; GENZ-438027; ALN-TTR02; ALN-18328	Approved	Alnylam Pharmaceuticals Inc	Onpattro	United States	Amyloid Neuropathies, Familial	Alnylam Pharmaceuticals Inc	2018-08-10	Amyloid Neuropathies; Amyloid Neuropathies, Familial; Amyloidosis; Amyloidosis, Familial; Cardiomyopathies; Polyneuropathies; Transthyretin amyloidosis	Details
Vutrisiran sodium	ALN-TTRSC02; ALN‑TTRsc02; ALN‑65492; AD-65492	Approved	Alnylam Pharmaceuticals Inc	AMVUTTRA	United States	Amyloid Neuropathies, Familial; Transthyretin amyloidosis	Alnylam Pharmaceuticals Inc	2022-06-13	Amyloid Neuropathies, Familial; Amyloidosis, Familial; Cardiomyopathies; Transthyretin amyloidosis	Details
Acoramidis Hydrochloride	BBP-265; AG-10; ALXN-2060; ALXN 2060	Approved	Stanford University	Attruby, Beyonttra	United States	Transthyretin amyloidosis	Bridgebio Pharma Inc	2024-11-22	Amyloid Neuropathies; Amyloid Neuropathies, Familial; Amyloidosis; Amyloidosis, Familial; Cardiomyopathies; Heart Diseases; Polyneuropathies; Transthyretin amyloidosis	Details
Inotersen sodium	ISIS-420915; GSK-2998728	Approved	Ionis Pharmaceuticals Inc	Tegsedi	EU	Amyloidosis	Akcea Therapeutics Ireland Ltd	2018-07-05	Adenomatous Polyposis Coli; Amyloid Neuropathies, Familial; Amyloidosis; Transthyretin amyloidosis	Details
Tafamidis	PF-6291826; Fx-1006A; PF-06291826; FX-1006A	Approved	Pfizer Inc	维万心, VYNDAMAX	United States	Heart Diseases	Foldrx Pharmaceuticals Inc	2019-05-03	Heart Diseases; Transthyretin amyloidosis	Details
Eplontersen	IONIS-TTR-LRx; AKCEA-TTR-LRx; ION-682884; ION-TTR-LRx	Approved	Ionis Pharmaceuticals Inc	WAINUA, 万诺维	United States	Transthyretin amyloidosis	Astrazeneca Ab	2023-12-21	Amyloid Neuropathies, Familial; Cardiomyopathies; Transthyretin amyloidosis	Details
Tafamidis Meglumine	FX-1006A; PF-6291826; Fx-1006A; PF-06291826	Approved	Pfizer Inc	维达全, Vyndaqel	EU	Amyloidosis	Pfizer Europe Ma Eeig	2011-11-16	Amyloid Neuropathies; Amyloid Neuropathies, Familial; Amyloidosis; Cardiomyopathies; Immunoglobulin Light-chain Amyloidosis; Transthyretin amyloidosis	Details