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Lysosomal storage disorder treatment moving into proof-of-concept phase

A company formed to develop protein therapeutics to treat lysosomal storage diseases — rare genetic disorders that affect the metabolism of certain natural biological substances in cells — is raising $5 million in a series A financing round, according to a filing with the U.S. Securities and Exchange Commission With the investment, the company is […]

A company formed to develop protein therapeutics to treat lysosomal storage diseases — rare genetic disorders that affect the metabolism of certain natural biological substances in cells — is raising $5 million in a series A financing round, according to a filing with the U.S. Securities and Exchange Commission

With the investment, the company is seeking to demonstrate proof of concept of its protein therapeutics in animals. It is currently in the preclinical stage.

Callidus Biopharma, based in Doylestown, Pennsylvania, was cofounded by Hung Do, the chief science officer of the company. He has 14 years of experience developing enzyme replacement therapies while he worked for Amicus Therapeutics, NovaZyme and at Genzyme, part of Sanofi (NYSE:SNY) when it acquired NovaZyme.

Do said although a handful of FDA-approved protein therapies, enzyme replacement therapies (ERTs) were developed to treat lysosomal storage diseases, the ERTs are not “optimal.” He explained that the current therapies each have deficiencies that cause only a small fraction of the therapeutic drug to be delivered to the intended tissues.

He said: “We are developing better versions of those enzymes with our proprietary technologies that address the deficiencies in current ERTs and deliver more of the therapy.”

 [Photo from flickr user GE Reports]