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Nucresiran - Alnylam Pharmaceuticals

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Drug Profile

Nucresiran - Alnylam Pharmaceuticals

Alternative Names: ALN-TTRsc04

Latest Information Update: 08 Aug 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

  • 31 Jul 2025 Nucresiran receives Fast track status from the US FDA for Transthyretin-related hereditary amyloidosis
  • 31 Jul 2025 Alnylam Pharmaceuticals plans the phase III TRITON-PM trial for Transthyretin-related hereditary amyloidosis with Polyneuropathy in second half of 2025
  • 25 Feb 2025 Alnylam Pharmaceuticals plans phase-III TRITON-CM/TRITON-PM trial for Transthyretin-related hereditary amyloidosis (SC)

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