ABO 101

Drug Profile

ABO 101

Alternative Names: AA9 NAGLU; AAV-NAGLU; ABO 101; ABX A; ABX-101; rAAV9.CMV.hNAGLU; Sanfilippo syndrome type-A gene therapy; SC AAV9 NAGLU

Latest Information Update: 21 Mar 2018

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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 Alpha N acetyl D glucosaminidase replacements; 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
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Mucopolysaccharidosis III

Most Recent Events

  • 16 Mar 2018 Abeona Therapeutics receives patent allowance in USA and has patents pending in the European Union for AAV capsid proteins
  • 16 Mar 2018 Abeona Therapeutics has patent protection for ABO 101 in USA
  • 08 Feb 2018 Positive preliminary safety and pharmacodynamic data from a phase I/II trial in Mucopolysaccharidosis III released by Abeona Therapeutics
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