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Research programme: rare haematological disorder gene therapies - Abeona Therapeutics

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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: 23 Aug 2023

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At a glance

  • Originator University of Minnesota
  • Developer Abeona Therapeutics; University of Minnesota
  • Class Antianaemics; 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.

    No
  • New Molecular Entity No

Highest Development Phases

  • Discontinued Fanconi's anaemia; Haematological disorders

Most Recent Events

  • 23 Aug 2023 Discontinued - Preclinical for Fanconi's anaemia in USA (Parenteral)
  • 23 Aug 2023 Discontinued - Preclinical for Haematological disorders in USA (Parenteral)
  • 28 Jul 2019 No recent reports of development identified for preclinical development in Fanconi's-anaemia in USA (Parenteral)

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