Synageva BioPharma is a biopharmaceutical company dedicated to discovering, developing, and delivering medicines for patients with rare diseases and high unmet medical need.
Everyone at Synageva shares a passion and unwavering commitment to improving the lives of patients with rare diseases. We have assembled a team from leading biotech companies that collectively have helped to bring several life saving therapies to patients whose diseases were once considered too rare to effectively develop treatments. Our guiding philosophy is that every patient fundamentally deserves to have effective treatment options for their disease, regardless of how rare their condition may be.
Synageva BioPharma’s lead program SBC-102 is an enzyme replacement therapy for lysosomal acid lipase (LAL) deficiency, also known as Wolman disease, cholesteryl ester storage disease (CESD), or Acid Lipase deficiency. LAL deficiency is a rare lysosomal storage disease (LSD) that is a progressive and often fatal condition. SBC-102 is in preclinical development and is designed to address the specific etiology of LAL deficiency by replacing the deficient or absent lysosomal acid lipase enzyme.
In addition to SBC-102, Synageva Biopharma has several other programs in development to help patients with lysosomal storage diseases and other life-threatening genetic conditions for which there are currently no effective treatments.
