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Brief Information

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:3
Number of Drugs in Clinical Trials:5
Lastest Research Phase:Approved

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

Synonym Name



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
Inotersen sodium GSK-2998728; ISIS-420915 Approved Ionis Pharmaceuticals Inc Tegsedi United States Transthyretin amyloidosis Akcea Therapeutics Inc 2018-07-05 Amyloid Neuropathies, Familial; Adenomatous Polyposis Coli; Amyloidosis; Transthyretin amyloidosis Details
Patisiran SAR-438037; GENZ-438027; ALN-TTR02; ALN-18328 Approved Alnylam Pharmaceuticals Inc Onpattro Japan Transthyretin amyloidosis Alnylam Pharmaceuticals Inc 2018-08-10 Amyloid Neuropathies, Familial; Amyloidosis, Familial; Cardiomyopathies; Amyloidosis; Transthyretin amyloidosis; Amyloid Neuropathies Details
Tafamidis Meglumine PF-6291826; Fx-1006A; PF-06291826; FX-1006A Approved Pfizer Inc 维达全, Vyndaqel, Vyndamax, Vyndaqel/Vyndamax Mainland China Transthyretin amyloidosis; Amyloid Neuropathies Pfizer Europe Ma Eeig 2011-11-16 Amyloid Neuropathies, Familial; Immunoglobulin Light-chain Amyloidosis; Heart Diseases; Cardiomyopathies; Amyloidosis; Transthyretin amyloidosis; Amyloid Neuropathies Details

Clinical Drug Information

Name Research Code Research Phase Company Indications Clinical Trials
PRX-004 PRX-004 Phase 1 Clinical Prothena Immunoglobulin Light-chain Amyloidosis; Transthyretin amyloidosis Details
ION-682884 ION-682884; ION-TTR-LRx; AKCEA-TTR-LRx; IONIS-TTR-LRx Phase 3 Clinical Ionis Pharmaceuticals Inc Amyloid Neuropathies, Familial; Transthyretin amyloidosis Details
NI-006 NI-006 Phase 1 Clinical Neurimmune Cardiomyopathies; Transthyretin amyloidosis Details
Acoramidis BBP-265; AG-10; ALXN-2060 Phase 3 Clinical Stanford University Amyloidosis, Familial; Heart Diseases; Cardiomyopathies; Amyloidosis; Transthyretin amyloidosis; Amyloid Neuropathies Details

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