Details of the Drug Therapeutic Target (DTT)

General Information of Drug Therapeutic Target (DTT) (ID: TTC1I86)

• DTT Name: Transthyretin (TTR)
• Synonyms: TBPA; Prealbumin; PALB; ATTR
• Gene Name: TTR
• DTT Type: Successful target; [1]
• UniProt ID: TTHY_HUMAN
• TTD ID: T86462
• Sequence:
  MASHRLLLLCLAGLVFVSEAGPTGTGESKCPLMVKVLDAVRGSPAINVAVHVFRKAADDT
  WEPFASGKTSESGELHGLTTEEEFVEGIYKVEIDTKSYWKALGISPFHEHAEVVFTANDS
  GPRRYTIAALLSPYSYSTTAVVTNPKE
• Function: Probably transports thyroxine from the bloodstream to the brain. Thyroid hormone-binding protein.
• KEGG Pathway: Thyroid hormone synthesis (hsa04918)
• Reactome Pathway:
  The canonical retinoid cycle in rods (twilight vision) (R-HSA-2453902)
  Non-integrin membrane-ECM interactions (R-HSA-3000171)
  Retinoid metabolism and transport (R-HSA-975634)
  Amyloid formation (R-HSA-977225)
  Retinoid cycle disease events (R-HSA-2453864)

Molecular Interaction Atlas (MIA) of This DTT

1 Approved Drug(s) Targeting This DTT

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
Inotersen	DMJ93CT	Hereditary amyloidosis	5D00.2	Approved	[1]

4 Clinical Trial Drug(s) Targeting This DTT

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
Acoramidis	DMRAP4S	Transthyretin amyloid cardiomyopathy	5D00.20	Phase 3	[2]
ALN-TTRsc	DMFUOM7	Cardiomyopathy	BC43	Phase 3	[3]
NI006	DM8U8KU	Amyloid transthyretin cardiomyopathy	5D00.20	Phase 1	[4]
NTLA-2001	DM1WDGE	Transthyretin amyloidosis	5D00	Phase 1	[5]

References

• [1] 2018 FDA drug approvals.Nat Rev Drug Discov. 2019 Feb;18(2):85-89.
• [2] Clinical pipeline report, company report or official report of AstraZeneca
• [3] Clinical pipeline report, company report or official report of Alnylam Pharmaceuticals, Inc.
• [4] Phase 1 Trial of Antibody NI006 for Depletion of Cardiac Transthyretin Amyloid. N Engl J Med. 2023 Jul 20;389(3):239-250.
• [5] CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis. N Engl J Med. 2021 Aug 5;385(6):493-502.