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Lysosomal Storage Disorders: Azafaros Takes Aim At Small Molecules

 
• By John Davis

BioGeneration Ventures has given birth to a new European biotech, Azafaros, supplying seed funding for the development of a new generation of compounds to tackle rare metabolic disorders.  

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Seed funding can be found to progress good ideas • Source: Shutterstock

Biotech and pharma companies have had some success in the past developing enzyme replacement therapies for lysosomal storage disorders such as Fabry disease and Gaucher disease, and a new European biotech company, Azafaros, spun out of Leiden University in the Netherlands, is aiming to build on that achievement by developing a new generation of agents for rare metabolic disorders.

The potential new class of therapeutic agents are small-molecule orally active, aza-sugar compounds which interfere with the metabolism of glycolipids and are expected to tackle some of the underlying causes of LSDs, conditions often linked

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