Drug Profile
Research programme: mRNA therapeutics - Translate Bio
Latest Information Update: 28 Apr 2023
Price :
$50
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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
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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
- Research Idiopathic pulmonary fibrosis; Liver disorders; Pulmonary arterial hypertension
- No development reported Eye disorders; Fabry's disease; Primary ciliary dyskinesia
- Discontinued Friedreich's ataxia; Inflammation; Musculoskeletal disorders; Neurodegenerative disorders; Spinal muscular atrophy
Most Recent Events
- 28 Apr 2023 No recent reports of development identified for preclinical development in Fabry's-disease in USA
- 28 Apr 2023 No recent reports of development identified for research development in Eye-disorders in USA (Intravitreous)
- 28 Apr 2023 No recent reports of development identified for research development in Primary-ciliary-dyskinesia in USA (Inhalation)