TTR

Synonyms

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

Description

TTR (Transthyretin) is a thyroid hormone-binding protein. Probably transports thyroxine from the bloodstream to the brain.

KO Status

F0

Drug Information

Launched drugs: 3
Drugs in clinical trials: 5
Latest Research Phase: Approved

Drug Name

Code

Phase

Company

Indications

Clinical Trials

Patisiran

SAR-438037, GENZ-438027, ALN-TTR02, ALN-18328

Approved

Alnylam Pharmaceuticals Inc

Amyloid Neuropathies, Familial, Amyloidosis, Familial, Cardiomyopathies, Amyloidosis, Transthyretin amyloidosis, Amyloid Neuropathies

Inotersen sodium

GSK-2998728, ISIS-420915

Approved

Ionis Pharmaceuticals Inc

Amyloid Neuropathies, Familial, Adenomatous Polyposis Coli, Amyloidosis, Transthyretin amyloidosis

Tafamidis Meglumine

PF-6291826, Fx-1006A, PF-06291826, FX-1006A

Approved

Pfizer Inc

Amyloid Neuropathies, Familial, Immunoglobulin Light-chain Amyloidosis, Heart Diseases, Cardiomyopathies, Amyloidosis, Transthyretin amyloidosis, Amyloid Neuropathies

Vutrisiran

ALN-TTRSC02

Phase 3 Clinical

Alnylam Pharmaceuticals Inc

Amyloidosis, Familial, Cardiomyopathies, Amyloidosis, Transthyretin amyloidosis

ION-682884

ION-682884, ION-TTR-LRx, AKCEA-TTR-LRx, IONIS-TTR-LRx

Phase 3 Clinical

Ionis Pharmaceuticals Inc

Amyloid Neuropathies, Familial, Transthyretin amyloidosis

Acoramidis

BBP-265, AG-10, ALXN-2060

Phase 3 Clinical

Stanford University

Heart Diseases, Cardiomyopathies, Amyloidosis, Transthyretin amyloidosis

PRX-004

PRX-004

Phase 1 Clinical

Prothena

Immunoglobulin Light-chain Amyloidosis, Transthyretin amyloidosis

NI-006

NI-006

Phase 1 Clinical

Neurimmune

Cardiomyopathies

ALN-TTR01

ALN-TTR01

Discontinued

Alnylam Pharmaceuticals Inc

Amyloidosis, Transthyretin amyloidosis

Revusiran

SAR-438714

Discontinued

Alnylam Pharmaceuticals Inc

Amyloid Neuropathies, Familial, Amyloidosis, Familial, Polyneuropathies, Amyloidosis, Amyloid Neuropathies, Transthyretin amyloidosis

References


Title

Authors

Source

Transthyretin amyloidosis: a new mutation associated with dementia

Petersen R.B., Goren H., Cohen M., Richardson S.L., Tresser N., Lynn A., Gali M., Estes M., Gambetti P.,

Ann. Neurol. 41:307-313(1997)

Transthyretin ILE20, a new variant associated with late-onset cardiac amyloidosis

Jacobson D.R., Pan T., Kyle R.A., Buxbaum J.N.,

Hum. Mutat. 9:83-85(1997)

Novel transthyretin missense mutation (Thr34) in an Italian family with hereditary amyloidosis

Patrosso M.C., Salvi F., De Grandis D., Vezzoni P., Jacobson D.R., Ferlini A.,

Am. J. Med. Genet. 77:135-138(1998)

A novel variant of transthyretin (Glu42Asp) associated with sporadic late-onset cardiac amyloidosis

Dupuy O., Bletry O., Blanc A.S., Droz D., Viemont M., Delpech M., Grateau G.,

Amyloid 5:285-287(1998)

New transthyretin variants Ser 91 and Ser 116 associated with familial amyloidotic polyneuropathy

Misrahi A.M., Plante V., Lalu T., Serre I., Adams D., Lacroix D.C., Said G.,

Hum. Mutat. 12:71-71(1998)

Transthyretin Ile73Val is associated with familial amyloidotic polyneuropathy in a Bangladeshi family

Booth D.R., Gillmore J.D., Persey M.R., Booth S.E., Cafferty K.D., Tennent G.A., Madhoo S., Cochrane S.W., Whitehead T.C., Pasvol G., Hawkins P.N.,

Hum. Mutat. 12:135-135(1998)

A new nonamyloid transthyretin variant, G101S, detected by electrospray ionization/mass spectrometry

Kishikawa M., Nakanishi T., Miyazaki A., Hatanaka M., Shimizu A., Tamoto S., Ohsawa N., Hayashi H., Kanai M.,

Hum. Mutat. 12:363-363(1998)

Transthyretin amyloidosis (serine 44) with headache, hearing loss, and peripheral neuropathy

Klein C.J., Nakumura M., Jacobson D.R., Lacy M.Q., Benson M.D., Petersen R.C.,

Neurology 51:1462-1464(1998)

A new amyloidogenic transthyretin variant (Val122Ala) found in a compound heterozygous patient

Theberge R., Connors L., Skare J., Skinner M., Falk R.H., Costello C.E.,

Amyloid 6:54-58(1999)

A new transthyretin variant (Ser23Asn) associated with familial amyloidosis in a Portuguese patient

Connors L.H., Theberge R., Skare J., Costello C.E., Falk R.H., Skinner M.,

Amyloid 6:114-118(1999)