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Transthyretin

Brief Information

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
Number of Launched Drugs:6
Number of Drugs in Clinical Trials:5
Lastest Research Phase:Approved

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TTR-H5223 Human Human Transthyretin / Prealbumin Protein, His Tag
TTR-H5223-structure
TTR-H5223-sds
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Synonym Name

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

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.

Clinical and Translational Updates

Public Drug Information

Name Research Code Research Phase Company First Brand Name First Approved Country First Indication First Approved Company First Approved Date Indications Clinical Trials
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, Familial; Immunoglobulin Light-chain Amyloidosis; Cardiomyopathies; Amyloidosis; Transthyretin amyloidosis; Amyloid Neuropathies Details
Eplontersen IONIS-TTR-LRx; AKCEA-TTR-LRx; ION-682884; ION-TTR-LRx Approved Ionis Pharmaceuticals Inc WAINUA United States Transthyretin amyloidosis Ionis Pharmaceuticals Inc 2023-12-21 Amyloid Neuropathies, Familial; Cardiomyopathies; 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
Inotersen sodium ISIS-420915; GSK-2998728 Approved Ionis Pharmaceuticals Inc Tegsedi EU Amyloidosis Akcea Therapeutics Ireland Ltd 2018-07-05 Amyloid Neuropathies, Familial; Adenomatous Polyposis Coli; Amyloidosis; Transthyretin amyloidosis Details
Vutrisiran ALN-TTRSC02 Approved Alnylam Pharmaceuticals Inc AMVUTTRA United States Amyloid Neuropathies, Familial; Transthyretin amyloidosis Alnylam Pharmaceuticals Inc 2022-06-13 Amyloidosis, Familial; Amyloid Neuropathies, Familial; Cardiomyopathies; Amyloidosis; Transthyretin amyloidosis Details
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 Amyloidosis, Familial; Amyloid Neuropathies, Familial; Polyneuropathies; Cardiomyopathies; Amyloidosis; Transthyretin amyloidosis; Amyloid Neuropathies Details

Clinical Drug Information

Name Research Code Research Phase Company Indications Clinical Trials
NI-006 NI-006 Phase 1 Clinical Neurimmune Ag Cardiomyopathies; Transthyretin amyloidosis Details
NPT-189 NPT-189 Phase 1 Clinical Proclara Biosciences Inc Amyloidosis Details
NTLA-2001 NTLA-2001 Phase 3 Clinical Intellia Therapeutics Inc Amyloidosis, Familial; Cardiomyopathies; Amyloid Neuropathies; Transthyretin amyloidosis Details
NNC6019-0001 NNC6019-0001; NNC-6019; PRX-004 Phase 2 Clinical Novo Nordisk A/S Cardiomyopathies; Transthyretin amyloidosis Details

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