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 Dystroph, and has now also initiated (pre)clinical programs for the development of antisense products for other indications including Myotonic Dystrophy and Huntington’s Disease.

Indication Compound Discovery Pre-clinical Phase I/II Phase III
Duchenne Muscular Dystrophy (DMD) PRO051
PRO044
PRO045
PRO053
PRO052
PRO055
Myotonic Dystrophy (DM1) PRO135
Huntington's Disease (HD) PRO289