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Research programme: gene therapies - Novo Nordisk

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Drug Profile

Research programme: gene therapies - Novo Nordisk

Latest Information Update: 03 Oct 2025

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At a glance

  • Originator bluebird bio; Novo Nordisk
  • Developer Genetix Biotherapeutics; Novo Nordisk
  • Class Blood coagulation factors; Gene therapies
  • Mechanism of Action Factor VIII 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

  • Preclinical Haemophilia A

Most Recent Events

  • 18 Sep 2025 bluebird bio is now called Genetix Biotherapeutics
  • 26 Jun 2024 Novo Nordisk acquires the Hemophilia A program and rights to MegaTAL in vivo gene editing technology from 2seventy bio
  • 01 May 2023 Preclinical trials in Haemophilia A in USA, Denmark (Parenteral) before May 2023

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