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Belcesiran - Dicerna Pharmaceuticals

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Drug Profile

Belcesiran - Dicerna Pharmaceuticals

Alternative Names: DCR A1AT; DCR-S1459; NN 6021

Latest Information Update: 24 Jul 2024

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At a glance

  • Originator Dicerna Pharmaceuticals
  • Class Amino sugars; Drug conjugates; Gene therapies; Small interfering RNA
  • Mechanism of Action Alpha 1-antitrypsin expression modulators; RNA interference
  • Orphan Drug Status

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

    Yes - Alpha 1-antitrypsin deficiency
  • New Molecular Entity Yes

Highest Development Phases

  • Discontinued Alpha 1-antitrypsin deficiency

Most Recent Events

  • 29 May 2024 Dicerna Pharmaceuticals terminates the phase II ESTRELLA trial in Alpha 1-antitrypsin deficiency in Australia, Austria, Belgium, Canada, France, Germany, Ireland, Netherlands, New Zealand, Portugal, Spain, Sweden, United Kingdom and USA as the development project was discontinued (NCT04764448)
  • 10 Nov 2023 Safety and efficacy data from a phase I trial in Alpha 1-antitrypsin deficiency presented at the 74th Annual Meeting of the American Association for the Study of Liver Diseases (AASLD-2023)
  • 28 Nov 2022 No recent reports of development identified for phase-I development in Alpha-1-antitrypsin-deficiency(In volunteers) in Sweden (SC, Injection)

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