ABO 102

Drug Profile

ABO 102

Alternative Names: AAV SGSH - Abeona; AAV-SGSH; 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: 25 Apr 2018

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At a glance

  • Originator Nationwide Children's Hospital
  • Developer Abeona Therapeutics
  • Class Gene therapies
  • Mechanism of Action Gene transference; N-sulfoglucosamine-sulfohydrolase-expression-stimulants
  • 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

  • 23 Apr 2018 ABO 102 receives Regenerative Medicine Advanced Therapy (RMAT) designation from the US FDA for Mucopolysaccharidosis
  • 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 102 in USA
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