Last update 21 Nov 2024

Nexiguran ziclumeran

Overview

Basic Info

Drug Type
CRISPR/Cas9
Synonyms
Nex-z
Target
Mechanism
TTR inhibitors(Transthyretin inhibitors)
Inactive Indication-
Originator Organization
Inactive Organization-
Drug Highest PhasePhase 3
First Approval Date-
RegulationOrphan Drug (US)
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Transthyretin Amyloid CardiomyopathyPhase 3
SE
13 Dec 2023
Transthyretin Amyloid CardiomyopathyPhase 3
DE
13 Dec 2023
Transthyretin Amyloid CardiomyopathyPhase 3
DK
13 Dec 2023
Transthyretin Amyloid CardiomyopathyPhase 3
ES
13 Dec 2023
Transthyretin Amyloid CardiomyopathyPhase 3
CA
13 Dec 2023
Transthyretin Amyloid CardiomyopathyPhase 3
TW
13 Dec 2023
Transthyretin Amyloid CardiomyopathyPhase 3
AU
13 Dec 2023
Transthyretin Amyloid CardiomyopathyPhase 3
AU
13 Dec 2023
Transthyretin Amyloid CardiomyopathyPhase 3
TW
13 Dec 2023
Transthyretin Amyloid CardiomyopathyPhase 3
SG
13 Dec 2023
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 1
36
(felzpxcopy) = bcgqpmsbxy bzmfgdaoyp (gheprtcikc, -33 to 49)
Positive
16 Nov 2024
Phase 1
62
(gcjlingapv) = The most commonly reported adverse events were infusion-related reactions, which occurred in 38% of patients. The majority of adverse events, including infusion-related reactions, were Grade 1 or 2 in severity, transient and resolved spontaneously. Other adverse events that were reported in greater than 10% of patients included headache, diarrhea and back pain, and were all Grade 1 or 2. pgfnjfeqbf (jmpuyfqqnx )
Positive
02 Nov 2023
Phase 1
14
wobpbfzadp(xpwsnninko) = Mild and transient infusion reactions were the most common adverse event with NTLA-2001 mtndtdbeqh (glnvwdnkil )
Positive
24 Jun 2022
Phase 1
6
(0.1 mg / kg)
(jvsfbjuvmq) = uklkfcaupt ptoxbputum (klcdugmusg )
Positive
05 Aug 2021
(0.3 mg / kg)
(jvsfbjuvmq) = wjotidplix ptoxbputum (klcdugmusg )
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Core Patent

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Approval

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Regulation

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