Research programme: RNA targeted therapeutics - Translate Bio

Drug Profile

Research programme: RNA targeted therapeutics - Translate Bio

Alternative Names: MRT 5005

Latest Information Update: 04 Nov 2017

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At a glance

  • Originator RaNA Therapeutics
  • Developer Shire; Translate Bio
  • Class Oligonucleotides
  • Mechanism of Action RNA 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

Highest Development Phases

  • Preclinical Cystic fibrosis; Friedreich's ataxia; Inborn urea cycle disorders; Spinal muscular atrophy
  • Research Musculoskeletal disorders
  • No development reported Inflammation

Most Recent Events

  • 15 Dec 2017 Translate Bio plans the phase I/II RESTORE-CF trial of MRT 5005 in Cystic fibrosis in USA (Inhalation,aerosol) (NCT03375047)
  • 04 Nov 2017 No recent reports of development identified for research development in Inflammation in USA
  • 27 Jun 2017 RaNA Therapeutics is now called Translate Bio
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