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Volrubigene ralaparvovec - Astellas Gene Therapies

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Drug Profile

Volrubigene ralaparvovec - Astellas Gene Therapies

Alternative Names: AAV-gene-therapy-Astellas Gene Therapies; AAV-UGT1A1; AT 342; AT003 - Astellas Gene Therapies; Crigler-Najjar-Syndrome-gene-therapy-Astellas Gene Therapies; Volrubigene ralaparvovec

Latest Information Update: 08 Apr 2021

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At a glance

  • Originator Audentes Therapeutics
  • Developer Astellas Gene Therapies
  • Class Gene therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

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

    Yes - Crigler-Najjar syndrome
  • New Molecular Entity No
  • Available For Licensing Yes

Highest Development Phases

  • Discontinued Crigler-Najjar syndrome

Most Recent Events

  • 01 Apr 2021 Audentes Therapeutics is now called Astellas Gene Therapies
  • 25 Feb 2021 Audentes Therapeutics terminates a phase I/II VALENS trial in Crigler-Najjar Syndrome in the USA, UK and Israeldue to sponsor decision (NCT03223194) (EudraCT 2017-001631-39)
  • 15 Jan 2020 Audentes Therapeutics was acquired by Astellas Pharma

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