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: 24 Jan 2017

Price : $50

At a glance

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

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

    Yes - Mucopolysaccharidosis III
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

    No
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Mucopolysaccharidosis III

Most Recent Events

  • 19 Jan 2017 ABO 101 receives Orphan Drug status for Mucopolysaccharidosis III in European Union
  • 06 Sep 2016 Phase-I/II clinical trials in Mucopolysaccharidosis III in USA (Parenteral) (Abeona Therapeutics pipeline, September 2016)
  • 24 May 2016 US FDA approves IND application for ABO 101 in Mucopolysaccharidosis III
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