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ABO 101

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Drug Profile

ABO 101

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

Latest Information Update: 13 Jun 2022

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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

  • Discontinued Mucopolysaccharidosis III

Most Recent Events

  • 31 May 2022 Abeona Therapeutics terminates a phase I/II trial in Mucopolysaccharidosis III (In children, In infants, In adolescents, In adults, In the elderly) in USA, Germany and France (IV), as the company discontinued development activities due to a lack of drug supply and for business reasons unrelated to the product safety profile and signs of efficacy (NCT04655911)
  • 07 Apr 2022 Abeona Therapeutics terminates the phase I/II Transpher B trial in Mucopolysaccharidosis III (In children, In neonates, In infants, In adolescents, In adults, In the elderly) in USA, France, Germany, Spain and the UK (IV) (NCT03315182)
  • 01 Mar 2022 Discontinued - Phase-I/II for Mucopolysaccharidosis III (In adolescents, In children, In the elderly, In infants, In adults) in USA, Spain, France, Germany, United Kingdom (IV)

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