ABO 201

Drug Profile

ABO 201

Alternative Names: AAV CLN3; 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; ABO-201; scAAV/MeCP2-hCLN3; scAAV/ß-actin-hCLN3; scAAV9 CLN3

Latest Information Update: 04 Jul 2017

$50 / €47 *
* $ price applies to USA, Canada and Latin America. € price applies to the rest of the world. Final gross prices may vary according to local VAT.

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

  • 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
  • 07 Oct 2016 Pharmacodynamics and pharmacokinetics data from preclinical studies in Neuronal ceroid lipofuscinosis released by Abeona Therapeutics
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