Pipeline

Prosensa’s proprietary antisense oligonucleotide 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 preclinical programs for the development of 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) Drisapersen
13% of DMD patients
PRO044
6% of DMD patients
PRO045
8% of DMD patients
PRO053
8% of DMD patients
PRO052
4% of DMD patients
PRO055
2% of DMD patients
PROSPECT
Myotonic Dystrophy (DM1) PRO135
Huntington's Disease (HD) PRO289