Lead ERT against lysosomal acid lipase deficiency is progressing into clinical development.

Synageva BioPharma has moved into larger office and laboratory premises in Lexington, MA. The firm says the bigger headquarters will allow it to broaden its discovery and translational research capabilities. “Over the last two years we have developed a pipeline of novel therapeutics for a variety of rare diseases, including lysosomal storage disorders and other genetic conditions for which there are currently no approved treatments,” remarks Sanj K. Patel, president and CEO. “The move is an important step toward ensuring the successful development of our lead program as well as advancing other pipeline programs.”

Synageva is dedicated to the discovery and development of treatments for rare diseases. The firm’s lead candidate, SBC-102, is a recombinant enzyme-replacement therapy for lysosomal acid lipase deficiency; it is moving into clinical development. Orphan drug designation for the treatment was granted by FDA in July. Synageva has four earlier-stage orphan products in development, targeting two lysosomal storage disorders, a metabolic disorder, and a genetic disease.

The firm claims its Synageva Expression Platform (SEP™)  demonstrates advantages over current expression methods for therapeutic proteins. SEP hinges on a proprietary vector technology that Synegeva maintains has achieved protein-expression levels of  about 14 g/L. The platform can be used to generate monoclonal antibodies, fusion proteins, cytokines, and therapeutic enzymes. It has also formed the production platform for products under evaluation in global Phase II trials, the company points out.

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