ABO 201

Drug Profile

ABO 201

Alternative Names: AAV-based juvenile Batten disease gene therapy - Abeona/University of Nebraska Medical Center; AAV-based neuronal ceroid lipofuscinosis gene therapy - Abeona/University of Nebraska Medical Center; AAV-CLN3; ABO-201; scAAV-CLN3; scAAV/MeCP2-hCLN3; scAAV/ß-actin-hCLN3; scAAV9 CLN3

Latest Information Update: 21 Mar 2018

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At a glance

  • Originator University of Nebraska Medical Center
  • Developer Abeona Therapeutics; University of Nebraska Medical Center
  • 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

  • 16 Mar 2018 Abeona Therapeutics files for patent protection with WIPO for ABO 201 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
  • 29 Jun 2017 ABO 201 receives Orphan Drug status for Neuronal ceroid lipofuscinosis (In children) in USA
  • 03 Jan 2017 ABO 201 receives Orphan Drug status for Neuronal ceroid lipofuscinosis (In children) in European Union
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