Research programme: RNA-interference therapeutics - BioMarinAlternative Names: PRO 052; PRO 055; PRO 105; PRO 135; PRO 289
Latest Information Update: 10 Oct 2015
At a glance
- Originator Leiden University
- Developer BioMarin Nederland
- Class Antisense oligonucleotides
- Mechanism of Action Antisense RNA modulators; Dystrophin stimulants
Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
On Fast track
Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.
- New Molecular Entity Yes
Highest Development Phases
- Preclinical Duchenne muscular dystrophy; Huntington's disease; Muscular dystrophies
- No development reported Spinal muscular atrophy
- Discontinued Neuroblastoma