Today is Rare Disease Day 2016. Because it is an important field for Biotech, we went back to review some of our best articles on the topic from the Labiotech archives.

Here are some of our best – in addition to an overview of 7 Rare diseases and who in Biotech is working on them.

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Nº 1: Rare Diseases: Still a ‘Blue Ocean’ for Biotechs?

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We wanted to get a deeper understanding of what is today considered a Rare Disease, who is acting the field and the reasons for so many Biotech companies to dive into the field.

We 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.


Nº 2: The French Woman who started a Biotech to Save her Daughter

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We interviewed the Founder and CEO of Lysogene from Paris (France).

Karen Aiach had no background in science or the biotech industry, until her daughter (Ornella) was diagnosed with the super-rare Sanfilippo A syndrome (also known as mucopolysaccharidosis or MPS-III).

This inspired her to co-found the clinical stage biotech which raised €16.5M from major investors in November 2014.


Nº 3: Why is Genzyme still Crazy Successful 5 Years after Sanofi’s Acquisition?

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Born in the Rare Disease field, Genzyme (US) was the Biotech that was too big and successful. Sanofi (France) revenues grew by up to 30% in 2015 after Genzyme’s buyout – a true Acquisition success story.


Nº4: World’s Best Journal in Medicine Publishes on Phase II Llama Antibodies

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Ablynx, the llama antibody biotech in Belgium, has proven to be on solid ground regarding their orphan drug for a rare blood disease. The New England Journal of Medicine has published their phase II results.


Nº 5: Leading-cause of blindness has new US & EU Orphan Drug Immunotherapy

TxCell, from Nice (France), has been granted EU and US Orphan Drug Designation for its T-cell based therapy for autoimmune uveitis – an eye inflammatory disease responsible of 10-15% legal blindness in developed countries.


We hope you found this Tops List useful. What do you think will be the future of Biotech & Rare Diseases?

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