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Research programme: antisense RNA therapeutics - Ionis Pharmaceuticals

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Drug Profile

Research programme: antisense RNA therapeutics - Ionis Pharmaceuticals

Latest Information Update: 01 Apr 2025

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At a glance

  • Originator Ionis Pharmaceuticals
  • Developer Ionis Pharmaceuticals; Metagenomi
  • Class Antihypertensives; Antisense RNA; Cardiovascular therapies
  • Mechanism of Action Angiotensinogen expression inhibitors; RNA interference
  • Orphan Drug Status

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

    No
  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Cardiovascular disorders; Resistant hypertension; Transthyretin-related hereditary amyloidosis

Most Recent Events

  • 17 Mar 2025 Metagenomi plans to initiate IND-enabling activities for development candidates in USA (Parenteral), in 2026
  • 17 Mar 2025 Pharmacodynamics data from preclinical trials in Resistant hypertension released by Metagenomi
  • 14 Aug 2024 Preclinical trials in Cardiovascular disorders in USA (Parenteral)

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