Research programme: genetic disorder therapeutics - BioBlast PharmaAlternative Names: BB-FA; BB-OTC; BBrm 01; BBrm02; BBrm1; Intrathecal azithromycin - BioBlast
Latest Information Update: 14 Jul 2015
At a glance
- Originator Bioblast Pharma
- Class Macrolides; Proteins; Small molecules
- Mechanism of Action Frataxin protein replacements; Gene expression modulators; Ornithine carbamoyltransferase replacements; Survival of motor neuron 2 protein expression 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 No
Highest Development Phases
- Preclinical Friedreich's ataxia; Inborn urea cycle disorders; Spinal muscular atrophy