Research programme: rare genetic disease therapeutics - Blueprint Medicines

Drug Profile

Research programme: rare genetic disease therapeutics - Blueprint Medicines

Alternative Names: ALK-2 targeting therapeutics - Blueprint Medicines; BLU-782

Latest Information Update: 31 Jul 2017

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At a glance

  • Originator Blueprint Medicines
  • Class Small molecules
  • Mechanism of Action Activin receptor modulators
  • Orphan Drug Status

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

  • New Molecular Entity Yes
  • Available For Licensing Yes

Highest Development Phases

  • Research Fibrodysplasia ossificans progressiva

Most Recent Events

  • 21 Feb 2018 Blueprint Medicines plans to initiate investigational new drug (IND) application-enabling studies for BLU 782 for Fibrodysplasia ossificans progressiva, in the first half of 2018
  • 27 Jul 2017 Alexion terminates research and development agreement with Blueprint Medicines for therapeutics targeting ALK-2 while still retaining their commercialisation rights (Blueprint Medicines pipeline, July 2017)
  • 27 Jul 2017 Research programme: rare genetic disease therapeutics - Blueprint Medicines is available for licensing as of 27 Jul 2017.
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