ABO 202

Drug Profile

ABO 202

Alternative Names: AAV-CLN1 - Abeona Therapeutics; ABO-202; scAAV-CLN1

Latest Information Update: 24 Apr 2018

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At a glance

  • Originator University of North Carolina at Chapel Hill
  • Developer Abeona Therapeutics; University of North Carolina at Chapel Hill
  • 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 - Neuronal ceroid lipofuscinosis
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Neuronal ceroid lipofuscinosis

Most Recent Events

  • 20 Apr 2018 ABO 202 receives Orphan Drug status for Neuronal ceroid lipofuscinosis in European Union
  • 16 Mar 2018 Abeona Therapeutics files for patent protection with WIPO for ABO 202 for the treatment of Neuronal ceroid lipofuscinosis before March 2018, and has patents pending in Australia, Canada, China, the European Union, Japan, New Zealand and USA
  • 15 Mar 2018 The US FDA grants Rare Pediatric Disease Designation for Neuronal ceroid lipofuscinosis
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