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Delpacibart etedesiran

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Drug Profile

Delpacibart etedesiran

Alternative Names: AOC-1001

Latest Information Update: 09 Oct 2024

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At a glance

  • Originator Avidity Biosciences
  • Class Drug conjugates; Immunoconjugates; Monoclonal antibodies; Small interfering RNA
  • Mechanism of Action Myotonin protein kinase 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 - Myotonic dystrophy
  • New Molecular Entity Yes

Highest Development Phases

  • Phase III Myotonic dystrophy

Most Recent Events

  • 03 Oct 2024 US FDA removes partial clinical hold for delpacibart etedesiran in Myotonic dystrophy type I
  • 30 May 2024 Phase-III clinical trials in Myotonic dystrophy in USA (IV) (NCT06411288)
  • 09 May 2024 Avidity Biosciences plans a phase III HARBOR™ trial of AOC 1001 for adults living with myotonic dystrophy type 1 (DM1) in the mid of 2024.

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