Pipeline
Prosensa’s proprietary technology platform, correcting mutated mRNA in life threatening disorders, may be applicable to a wide variety of diseases. Prosensa has achieved first clinical proof of concept in Duchenne Muscular Dystrophy, and has now also initiated (pre)clinical programs for the development of antisense products for other (neuromuscular) indications including Myotonic Dystrophy (DM1), Huntington’s Disease (HD), and Spinal Muscular Atrophy (SMA).
| Indication | Compound | Discovery | Pre-clinical | Phase I / II | Phase III |
|---|---|---|---|---|---|
| Duchenne Muscular Dystrophy (DMD) | PRO051/GSK2402968 | 100% finished | 100% finished | 100% finished | 10% finished |
| PRO044 | 100% finished | 100% finished | 70% finished | ||
| PRO045 | 100% finished | 70% finished | |||
| PRO053 | 100% finished | 70% finished | |||
| PRO052 | 100% finished | 20% finished | |||
| PRO055 | 100% finished | 20% finished | |||
| Myotonic Dystrophy (DM1) | PRO135 | 100% finished | 5% finished | ||
| Huntington’s Disease (HD) | PRO289 | 100% finished | 5% finished | ||
| Spinal Muscular Atrophy (SMA) | PRO105 | 100% finished |