The Role of Biotechnology in Europe's Responsible Innovation - Prosensa


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Short listed SMEs for the SME Award


Prosensa is a biopharmaceutical company focused on the discovery, development and commercialization of RNA (ribonucleic acid) modulating therapeutics.

Prosensa was founded in 2002 and is located in Leiden, The Netherlands

The company targets genetic disorders with a large unmet medical need, with a primary focus on neuromuscular and neurodegenerative life threatening disorders such as Duchenne Muscular Dystrophy, Myotonic Dystrophy, Huntington's disease and Spinal Muscular Atrophy.

Prosensa has excelled in its creativity, innovativeness and effectiveness by creating a business model where a close collaboration with academic partners, patient organizations and the pharmaceutical industry plays a central role in achieving its objective of bringing innovative treatments to patients as quickly as possible. Prosensa aims to discover, develop and commercialise its products and to create a specialised orphan drug franchise. The company focuses on addressing diseases with a large unmet medical need, for which no curative treatments exist today.

Comment from the EuropaBio judging panel for the Most Innovative European SME on why Prosensa has been shortlisted for the award:

“Duchenne Muscular Dystrophy is a severely debilitating disease for which there is no effective treatment affecting 1 in 3500 boys. It is caused by an error in gene transcription and leads to respiratory failure and death at an early age. Prosensa has developed a technology to correct the mis-reading of the genetic information and is progressing a product in Phase III clinical development to reverse the symptoms of this disease. This technology is based upon an understanding of "exon skipping" which led to the development of a practical therapeutic strategy to overcome the safety and delivery constraints of gene therapy. In a very challenging environment for raising venture finance, Prosensa was able to get early stage financial support from leading patient advocacy groups. The success of this strategy has allowed the company to establish a partnership with a major pharmaceutical company and put it on the pathway to sustainability as a neuromuscular specialty biopharma company.”