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ABO 201

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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: 28 Oct 2023

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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

  • No development reported Neuronal ceroid lipofuscinosis

Most Recent Events

  • 28 Oct 2023 No recent reports of development identified for preclinical development in Neuronal ceroid lipofuscinosis in USA (Parenteral)
  • 29 Dec 2020 Abeona Therapeutics has patent protection for ABO 201 in USA
  • 23 Sep 2019 ABO 201 is still in preclinical development in Neuronal ceroid lipofuscinosis in USA (Parenteral)

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