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A registrational trial assessing AXO AAV GM2 for GM2 gangliosidosis

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Trial Profile

A registrational trial assessing AXO AAV GM2 for GM2 gangliosidosis

Status: Planning
Phase of Trial: Phase III

Latest Information Update: 19 Nov 2020

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At a glance

  • Drugs Cobnabexagene anvuparvovec (Primary)
  • Indications GM2 gangliosidoses
  • Focus Registrational; Therapeutic Use
  • Sponsors Sio Gene Therapies [CEASED]

Most Recent Events

  • 09 Nov 2020 Status changed from suspended to planning, according to an Axovant Gene Therapies media release.
  • 09 Nov 2020 According to an Axovant Gene Therapies media release, Terence R. Flotte, M.D., Professor of Pediatrics and Dean at the University of Massachusetts Medical School, will serve as principal investigator on the clinical trial.
  • 09 Nov 2020 According to an Axovant Gene Therapies media release, this study will form the basis of the registrational program.

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