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Cobnabexagene anvuparvovec - Sio Gene Therapies

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Drug Profile

Cobnabexagene anvuparvovec - Sio Gene Therapies

Alternative Names: AXO-AAV-GM2; AXO-AAV-GM2 Program; rAAVrh8-HexA/HexB

Latest Information Update: 24 Jan 2025

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At a glance

  • Originator University of Massachusetts Medical School
  • Developer Sio Gene Therapies; University of Massachusetts Medical School
  • Class Gene therapies
  • Mechanism of Action Gene transference; Hexosaminidase A replacements; Hexosaminidase B replacements
  • Orphan Drug Status

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

    Yes - Tay-Sachs disease
  • New Molecular Entity No
  • Available For Licensing Yes

Highest Development Phases

  • Discontinued Sandhoff disease; Tay-Sachs disease

Most Recent Events

  • 16 Dec 2024 Sio Gene completed phase-I/II clinical trials in Tay-Sachs or Sandhoff Diseases (In infants, In children) in USA (Intrathecal) (intracisternal) (NCT04669535)
  • 11 Aug 2022 Sio Gene Therapies terminates licensing agreement with The University of Massachusetts to develop and commercialise Cobnabexagene anvuparvovec for GM2 gangliosidosis including Tay-Sachs disease and Sandhoff disease
  • 11 Aug 2022 Discontinued - Phase-I/II for Sandhoff disease (In children, In infants) in USA (Intracisternal)

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