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Nucresiran - Alnylam Pharmaceuticals

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Drug Profile

Nucresiran - Alnylam Pharmaceuticals

Alternative Names: ALN-TTRsc04

Latest Information Update: 14 Nov 2025

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At a glance

  • Originator Alnylam Pharmaceuticals
  • Class Small interfering RNA
  • Mechanism of Action Prealbumin expression inhibitors; RNA interference
  • 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 Yes

Highest Development Phases

  • Phase I Transthyretin-related hereditary amyloidosis

Most Recent Events

  • 30 Sep 2025 Alnylam Pharmaceuticals completes a phase I trials in Transthyretin-related hereditary amyloidosis (In volunteers) in United Kingdom (SC) (NCT05661916)
  • 31 Jul 2025 Alnylam Pharmaceuticals plans the phase III TRITON-PN trial for Transthyretin-related hereditary amyloidosis with Polyneuropathy (NCT07223203)
  • 31 Jul 2025 Nucresiran receives Fast track status from the US FDA for Transthyretin-related hereditary amyloidosis

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