Ziclumeran, Single Guide Rna Targeting The Human Ttr Gene

3 trials 1 recruiting Orphan designation

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3 Registered trials

1 Recruiting

1 Completed trials

1 Sponsors

14 Countries

Phase III

Phase II/III

Phase I

Phase III: 1 Phase II/III: 1 Phase I: 1

Oversight events:

14 temporary halts 1 corrective measure across 1 trial

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Indications tested

Cardiac amyloidosis 1 Hereditary neuropathic amyloidosis 1

Sponsors using this IMP

Intellia Therapeutics Inc. 3

All trials

3 total

Long-Term Follow-Up of Subjects Treated With NTLA-2001 Recruiting

2023-507385-11-00 Phase II/III 2 countries Intellia Therapeutics Inc.
A Phase 3 Study of NTLA 2001 in participants with amyloidosis with cardiac muscle disease. Halted

2023-507220-23-00 Phase III 14 countries Intellia Therapeutics Inc.
NTLA-2001 in Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy (ATTRv-PN) Ended

2024-511170-69-00 Phase I 2 countries Intellia Therapeutics Inc.

Showing 1–3 of 3 trials

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