Research programme: rare haematological disorder gene therapies - Abeona Therapeutics

Drug Profile

Research programme: rare haematological disorder gene therapies - Abeona Therapeutics

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

Latest Information Update: 14 Oct 2015

Price : $50

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.

    No
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

    No
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Fanconi's anaemia; Haematological disorders

Most Recent Events

  • 07 Oct 2015 Abeona Therapeutics in-licenses an adeno-associated virus delivery vector from Stanford University
  • 15 Jun 2015 Preclinical trials in Haematological disorders in USA (Parenteral)
  • 15 Jun 2015 Rare haematological disorder gene therapies licensed to PlasmaTech Biopharmaceuticals worldwide (9183206; 9180639)
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at AsktheExpert.AdisInsight@springer.com so we can help.

Back to top