ABO 102

Drug Profile

ABO 102

Alternative Names: AAV SGSH; AAV SGSH - Abeona; AAV9 SGSH - Abeona; ABO-102; ABO-102 - Abeona; ABX 102 - Abeona; ABX B - Abeona; SC AAV9 SGSH; scAAV9 SGHG; scAAV9.U1a.hSGSH

Latest Information Update: 14 Oct 2017

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At a glance

  • Originator Nationwide Children's Hospital
  • Developer Abeona Therapeutics
  • 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
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Mucopolysaccharidosis III

Most Recent Events

  • 12 Oct 2017 Updated efficacy data from a phase I/II trial in Mucopolysaccharidosis III released by Abeona Therapeutics
  • 06 Oct 2017 Top-line one year efficacy and safety data from a phase I/II trial in Mucopolysaccharidosis III released by Abeona Therapeutics
  • 16 Aug 2017 Regulatory and ethical committee approvals granted for ABO 102 for phase I/II trial in Mucopolusaccharidosis III in Europe and Australia
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