We aim to develop innovative, RNA-based therapeutics to address unmet medical needs for patients with genetic disorders.

All neuromuscular disorders

Duchenne Muscular Dystrophy Patient Stories

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Pipeline

Compound Discovery Pre-clinical Phase I / II Phase III
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

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Understanding DMD

About Prosensa

Prosensa is a biotechnology company engaged in the discovery, development and commercialization of ribonucleic acid (RNA) modulating therapeutics. The company targets the treatment of genetic disorders with a large unmet medical need, with a primary focus on neuromuscular and neurodegenerative disorders, such as Duchenne Muscular Dystrophy (DMD), Myotonic Dystrophy and Huntington’s disease. Our clinical portfolio of RNA-based product candidates is focused on the treatment of DMD. Each of our DMD compounds has been granted orphan drug status in the United States and the European Union.

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RNA Modulation

RNA-modulating therapeutics provide a powerful tool for targeted modulation of gene expression. Our unique proprietary technology platform employs single-stranded RNA-based antisense oligonucleotides (AONs) to correct mutated mRNA causing life threatening disorders.

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Exon Skipping

Our exon skipping technology is based on interference with exon inclusion signals during the splicing of the pre-mRNA, in order to induce the skipping of the targeted exon from the mature mRNA (messenger RNA) and to restore the level of functional transcripts.

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