Transthyretin Overview
Molecule Name
Transthyretin
Synonyms
Transthyretin,TTR,Prealbumin,TBPA,ATTR,PALB,CTS,CTS1,HsT2651
Drug Development Progress
Drugs in Clinical Trials8
Approved Drugs7
Highest Development StageApproved
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
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 Product List
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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 | First Brand Name | First Approved Country | First Indication | First Approved Company | First Approved 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 |