Details of the Drug-Related molecule(s) Interaction Atlas

General Information of Drug (ID: DM1WDGE)

Drug Name

NTLA-2001 Drug Info

Indication

Disease Entry	ICD 11	Status	REF
Transthyretin amyloidosis	5D00	Phase 1	[1]

Cross-matching ID

Molecule-Related Drug Atlas

Molecule Type:

DTT

Drug Status:

Approved Drug(s)

Clinical Trial Drug(s)

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Drug(s) Targeting Transthyretin (TTR)

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
Inotersen	DMJ93CT	Hereditary amyloidosis	5D00.2	Approved	[3]
ALN-TTRsc	DMFUOM7	Cardiomyopathy	BC43	Phase 3	[4]
Acoramidis	DMRAP4S	Transthyretin amyloid cardiomyopathy	5D00.20	Phase 3	[5]
NI006	DM8U8KU	Amyloid transthyretin cardiomyopathy	5D00.20	Phase 1	[6]
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Molecular Interaction Atlas of This Drug

Molecular Interaction Atlas

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Drug Therapeutic Target (DTT)

References

1	ClinicalTrials.gov (NCT04601051) Phase 1 Two-Part (Open-label, Single Ascending Dose (Part 1) and Open-label, Single Dose Expansion (Part 2)) Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM). U.S.National Institutes of Health.
2	CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis. N Engl J Med. 2021 Aug 5;385(6):493-502.
3	2018 FDA drug approvals.Nat Rev Drug Discov. 2019 Feb;18(2):85-89.
4	Clinical pipeline report, company report or official report of Alnylam Pharmaceuticals, Inc.
5	Clinical pipeline report, company report or official report of AstraZeneca
6	Phase 1 Trial of Antibody NI006 for Depletion of Cardiac Transthyretin Amyloid. N Engl J Med. 2023 Jul 20;389(3):239-250.