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Prosensa

Leiden, South Holland, Netherlandsorpha.net ↗

Ownership: Private
Development stage: Phase 3

Overview

Prosensa developed a portfolio of RNA-modulating therapeutics for rare neuromuscular diseases. Its lead compound, drisapersen for DMD, failed a pivotal Phase III trial but showed promise in extension studies. The company regained rights from GSK before being acquired by BioMarin for up to $840 million. At acquisition, its pipeline included several exon-skipping compounds at various stages targeting different mutations within the dystrophin gene and other neuromuscular disorders.

Therapeutic areas: Rare DiseasesNeurology
Modalities: RNA-modulating therapeuticsAntisense oligonucleotides (exon-skipping technology)
Industry: Biotech

Drug pipeline

6 assets

drisapersen

Phase 3

Indications: Duchenne muscular dystrophy

5 more assets in the full pipeline.

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Partnerships

GlaxoSmithKline – strategic alliance/licensing agreement on part of the exon skipping program including drisapersen until rights reverted back to Prosensa in January 2014.BioMarin – acquirer; continued development post-acquisition.Academic collaborations with patient groups and experts worldwide are noted but not individually named.

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