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Bomtabegagene bavoparvovec - Sio Gene Therapies

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Drug Profile

Bomtabegagene bavoparvovec - Sio Gene Therapies

Alternative Names: AAV9-GLB1; AXO-AAV-GM1; AXO-AAV-GM1 Program

Latest Information Update: 30 Aug 2022

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At a glance

  • Originator University of Massachusetts Medical School
  • Developer National Institutes of Health (USA); Sio Gene Therapies; University of Massachusetts Medical School
  • Class Gene therapies
  • Mechanism of Action Gene transference; GLB1 protein replacements
  • Orphan Drug Status

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

    Yes - Gangliosidoses
  • New Molecular Entity No

Highest Development Phases

  • Discontinued Gangliosidoses

Most Recent Events

  • 11 Aug 2022 Sio Gene Therapies terminates licensing agreement with The University of Massachusetts to develop and commercialise Bomtabegagene bavoparvovec for GM1 gangliosidosis
  • 11 Aug 2022 Discontinued - Phase-I/II for Gangliosidoses (In children, In infants) in USA (IV)
  • 11 Feb 2022 Updated safety data from a phase I/II trial in Gangliosidoses presented at the 18th Annual WORLDSymposium™ 2022

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