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Synageva BioPharma to Present at the Cowen and Company 32nd Annual Healthcare Conference
LEXINGTON, Mass.--(BUSINESS WIRE)--Feb. 28, 2012-- Synageva BioPharma Corp. (“
About Synageva’s Lead Program
SBC-102 is being developed as an enzyme replacement for Lysosomal Acid Lipase (LAL) Deficiency, a lysosomal storage disorder (LSD), and is a recombinant form of the human LAL enzyme. SBC-102 is currently being evaluated in global clinical trials and has been granted orphan designations by the
Interim results from a 4-week Phase I/II trial in adults with LAL Deficiency indicated that SBC-102 was well-tolerated with no serious adverse events or infusion-related reactions, and all subjects completed their scheduled infusions. The majority of adverse events were mild and unrelated to SBC-102. SBC-102 resulted in rapid and significant decreases in serum transaminases with evidence of mobilization of lipids out of the liver and other tissues and into the blood, consistent with its mechanism of action.
About LAL Deficiency
Lysosomal Acid Lipase Deficiency is a rare, autosomal recessive lysosomal storage disorder (LSD) that is caused by a marked decrease in lysosomal acid lipase activity. Late onset LAL Deficiency, sometimes called Cholesteryl Ester Storage Disease (CESD), affects both children and adults. In these patients, the buildup of fatty material in the liver, spleen and blood vessel walls leads to complications resulting in significant morbidity and mortality. Early onset LAL Deficiency, sometimes called Wolman Disease, affects infants in the first year of life and is characterized by growth failure, malabsorption, steatorrhea and hepatomegaly and is rapidly fatal, usually within the first year of life.
Synageva is a clinical stage biopharmaceutical company focused on the discovery, development, and commercialization of therapeutic products for patients with life-threatening rare diseases and unmet medical need. Synageva has several protein therapeutics in its pipeline. The company has assembled a team with a proven record of bringing orphan therapies to patients.
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