Research programme: rare haematological disorder gene therapies - Abeona Therapeutics

Drug Profile

Research programme: rare haematological disorder gene therapies - Abeona Therapeutics

Alternative Names: AAV LK19 FANCC - Abeona Therapeutics/University of Minnesota; AAV-based Fanconi's anaemia gene therapy - Abeona Therapeutics/University of Minnesota; AAV-FANCC; ABO 301; ABO 302; CRISPR-CAS9 AAV - Abeona Therapeutics/University of Minnesota

Latest Information Update: 21 Mar 2018

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At a glance

  • Originator University of Minnesota
  • Developer Abeona Therapeutics; University of Minnesota
  • Class Gene therapies
  • Mechanism of Action Gene modulators; Gene transference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

  • New Molecular Entity No

Highest Development Phases

  • Preclinical Fanconi's anaemia; Haematological disorders

Most Recent Events

  • 16 Mar 2018 Abeona has patents pending for an AAV gene therapy for the treatment of Fanconi's anaemia and other Haematological disorders in Canada, Hong Kong and Japan
  • 17 May 2017 Abeome has patents pending for AAV gene therapy in USA and Europe
  • 07 Oct 2015 Abeona Therapeutics in-licenses an adeno-associated virus delivery vector from Stanford University
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