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Drug Profile


Alternative Names: AVR-RD-03

Latest Information Update: 25 Aug 2023

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At a glance

  • Originator AVROBIO
  • Class Cell therapies; Gene therapies; Haematopoietic stem cells therapies; Stem cell therapies
  • Mechanism of Action Alpha-glucosidase stimulants; Gene transference
  • Orphan Drug Status

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

  • New Molecular Entity No

Highest Development Phases

  • Suspended Glycogen storage disease type II

Most Recent Events

  • 12 Jul 2023 Suspended - Preclinical for Glycogen storage disease type II in USA (Parenteral) as AVROBIO is exploring strategic alternatives
  • 31 Dec 2022 AVROBIO has a patent pending for AVR RD 03 pertaining to claims directed to CD34+ stem cells expressing acid alpha-glucosidase and methods of using the same for the treatment of Glycogen storage disease type II in USA
  • 18 May 2022 AVROBIO announces plans to engage with regulatory agencies regarding the clinical development strategy for AVR RD 03 in 2022
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