Research programme: gene modulation therapeutics - Fulcrum Therapeutics

Drug Profile

Research programme: gene modulation therapeutics - Fulcrum Therapeutics

Latest Information Update: 14 Sep 2016

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At a glance

  • Originator Fulcrum Therapeutics
  • Class Gene therapies; Small molecules
  • Mechanism of Action Gene expression modulators
  • Orphan Drug Status

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

    No
  • New Molecular Entity Yes

Highest Development Phases

  • Research Facioscapulohumeral muscular dystrophy; Fragile X syndrome

Most Recent Events

  • 06 Sep 2016 Fulcrum Therapeutics and Horizon Discovery enter into a collaboration for CRISPR-based target discovery for genetic diseases
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