Rare Diseases: Still a ‘Blue Ocean’ for Biotechs?

01/12/2015 - 6 minutes

We all know the success stories linked to Rare Disease such as Genzyme. But I wanted to get a deeper understanding on what is today considered as Rare disease, who is acting the field and the reasons for so many Biotech companies to dive into the field.

Last week was the RARE 2015 congress in Montpellier, France. A good opportunity to cover the field of rare disease. I had a chat with three key opinion leaders from Europe to get an expert view on the subject: Andrew Weiss, SVP Communication from Actelion, Karen Aiach, the Founder & CEO of Lysogene and Stephane Boissel, the CEO of TxCell.

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A Rare or an Orphan Disease is defined as a disease affecting a low percentage of a certain population, 1 out of 1,500 people in the US, 1 out of 2,500 in Japan and 1 out of 2,000 in Europe.

According to the European Organisation for Rare Disease,

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