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AVR RD 02

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AVR RD 02

Alternative Names: AVR-RD-02

Latest Information Update: 25 Jun 2024

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At a glance

  • Originator Lund University
  • Developer Tectonic Therapeutic
  • Class Gene therapies; Haematopoietic stem cells therapies
  • Mechanism of Action Gene transference; Glucosylceramidase replacements
  • Orphan Drug Status

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

    Yes - Gaucher's disease
  • New Molecular Entity No

Highest Development Phases

  • Suspended Gaucher's disease type I

Most Recent Events

  • 20 Jun 2024 AVROBIO has been merged with Tectonic Therapeutic to form Tectonic Therapeutic
  • 21 Aug 2023 AVROBIO voluntarily terminates the phase I/II Guard1 trial in Gaucher's disease type I (In adults) in USA, Australia and Canada (IV, Infusion), due to a business decision not to proceed, and not due to any safety or efficacy issue (NCT04145037)
  • 09 Aug 2023 AVROBIO withdraws a phase II/III Guard3 trial in Gaucher's disease type III (In children, In adolescents, In adults) (IV) prior to enrollment, before August 2023 (NCT05815004)

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