Research programme: RNA targeted therapeutics - Translate Bio

Drug Profile

Research programme: RNA targeted therapeutics - Translate Bio

Latest Information Update: 05 Jul 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 Inflammation; Musculoskeletal disorders

Most Recent Events

  • 27 Jun 2017 RaNA Therapeutics is now called Translate Bio
  • 27 Jun 2017 Translate Bio announces intention to file IND to the US FDA for Cystic fibrosis and Inborn urea cycle disorders by the end of 2017
  • 27 Jun 2017 Translate Bio plans a phase I trial in Cystic fibrosis in early 2018
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