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Nexiguran ziclumeran - Intellia Therapeutics

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Drug Profile

Nexiguran ziclumeran - Intellia Therapeutics

Alternative Names: nex-z; NTLA-2001

Latest Information Update: 04 Dec 2024

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At a glance

  • Originator Intellia Therapeutics
  • Developer Intellia Therapeutics; Regeneron Pharmaceuticals
  • Class Gene therapies
  • Mechanism of Action Gene expression inhibitors
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Transthyretin-related hereditary amyloidosis
  • New Molecular Entity No

Highest Development Phases

  • Phase III Transthyretin-related hereditary amyloidosis

Most Recent Events

  • 25 Nov 2024 Intellia Therapeutics initiates a phase III trial for Transthyretin-related hereditary amyloidosis with polyneuropathy in New Zealand (IV, Infusion) (NCT06672237)
  • 25 Nov 2024 Nexiguran ziclumeran receives Regenerative Medicine Advanced Therapy (RMAT) status for Transthyretin-related hereditary amyloidosis in USA
  • 16 Nov 2024 Efficacay and adverse events data from phase-I trial in Transthyretin-related hereditary amyloidosis released by Intellia Therapeutics

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