Orphazyme lands $20M A round to back new drugs for rare genetic disorder

Fresh from gaining a big injection of new drug technology, Danish biotech startup Orphazyme is banking a $20 million venture round to back its work on experimental treatments for rare lysosomal storage disorders. New investor Aescap Venture stepped in on the A round along with existing investors Novo A/S and Sunstone Capital.

The money arrives just a few months after the biotech acquired CytRx's molecular chaperone technology, along with the experimental drug arimoclomol, which has been tested in a few mid-stage studies. Orphazyme is planning to develop oral drugs.

"Lysosomal storage disorders, a group of debilitating diseases with high unmet medical need, still lack new and effective treatments," says Hakan Goker, Principal at Aescap Venture. "Our efforts in this space have identified Orphazyme as one of the best biotechs globally with potentially the most broadly applicable technology that can revolutionize the treatment of these diseases. We are proud to be able to support the strong leadership and science of Orphazyme, and be a part of this exciting development with an excellent syndicate of investors."

Orphazyme is hoping to make its mark with a molecular "chaperone," heat-shock protein 70, which reverts the pathology in cell extracts from patients suffering from various lysosomal storage diseases. Currently, only a few treatments exist for these genetic disorders, which are often fatal. Rare disorders have become a hot field in biotechnology as start-ups set out to mimic the success of companies like Genzyme.

- here's the Orphazyme release

Special Report: Top 5 biotech venture capital deals, H1 2011

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