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Research programme: mRNA therapeutics - Translate Bio

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Drug Profile

Research programme: mRNA therapeutics - Translate Bio

Latest Information Update: 28 Apr 2025

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At a glance

  • Originator RaNA Therapeutics
  • Developer Translate Bio
  • Class Anti-inflammatories; Antifibrotics; Eye disorder therapies; Hepatoprotectants; RNA
  • Mechanism of Action Protein synthesis stimulants; RNA 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

  • No development reported Cystic fibrosis; Eye disorders; Fabry's disease; Idiopathic pulmonary fibrosis; Liver disorders; Primary ciliary dyskinesia; Pulmonary arterial hypertension
  • Discontinued Friedreich's ataxia; Inflammation; Musculoskeletal disorders; Neurodegenerative disorders; Spinal muscular atrophy

Most Recent Events

  • 28 Apr 2025 No recent reports of development identified for preclinical development in Cystic-fibrosis in USA (Inhalation, Aerosol)
  • 28 Oct 2024 No recent reports of development identified for research development in Idiopathic-pulmonary-fibrosis in USA (Inhalation)
  • 28 Oct 2024 No recent reports of development identified for research development in Liver-disorders in USA (IV)

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