Research programme: antisense oligonucleotides - Baylor College of Medicine/Ionis PharmaceuticalsAlternative Names: UBE3A gene expression stimulant; UBE3A-ATS targeting oligoneucleotides
Latest Information Update: 24 Dec 2015
At a glance
- Originator Baylor College of Medicine; Isis Pharmaceuticals
- Developer Baylor College of Medicine; Ionis Pharmaceuticals
- Class Oligonucleotides
- Mechanism of Action Antisense RNA inhibitors
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 Angelman syndrome