ABO 101

Drug Profile

ABO 101

Alternative Names: AA9 NAGLU; AAV-NAGLU; ABO 101; ABX A; ABX-101; rAAV9.CMV.hNAGLU; SC AAV9 NAGLU

Latest Information Update: 31 Oct 2017

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At a glance

  • Originator Nationwide Children's Hospital
  • Developer Abeona Therapeutics; Nationwide Children's Hospital
  • Class Gene therapies
  • Mechanism of Action Gene transference; Nervous system modulators
  • Orphan Drug Status

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

    Yes - Mucopolysaccharidosis III
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Mucopolysaccharidosis III

Most Recent Events

  • 01 Oct 2017 Abeona Therapeutics initiates enrolment in a phase I/II trial in Mucopolysaccharidosis III in USA (IV) (NCT03315182)
  • 30 Mar 2017 Abeona Therapeutics has patent protection for AAV capsid proteins in USA
  • 30 Mar 2017 Abeona Therapeutics has patents pending for AAV capsid proteins in Europe
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