About UsVisionSynageva BioPharma is a clinical stage biopharmaceutical company dedicated to discovering, developing and delivering medicines for patients with rare disease and unmet medical need. At Synageva, we share a passion and commitment to improving the lives of patients with rare diseases. Ou…
About Us Vision Synageva BioPharma is a clinical stage biopharmaceutical company dedicated to discovering, developing and delivering medicines for patients with rare disease and unmet medical need. At Synageva, we share a passion and commitment to improving the lives of patients with rare diseases. Our team is drawn from leading biotech companies and has helped bring several life-saving therapies to patients whose diseases were once considered too rare for developing treatments. We believe that every patient deserves treatment options, regardless of the rarity of their disease. Programs Our lead program, SBC-102, is a recombinant human lysosomal acid lipase currently under clinical investigation for the treatment of patients with early onset and late onset Lysosomal Acid Lipase (LAL) Deficiency. This progressive and often fatal lysosomal storage disorder is also known as Wolman Disease and Cholesteryl Ester Storage Disease (CESD), respectively. SBC-102 has been granted Orphan Designations by both FDA and EMA, and fast track designation by the FDA.
In addition to SBC-102, Synageva has several other protein therapeutic programs targeting rare diseases in various stages of preclinical development. These include additional enzyme replacement therapies for lysosomal storage disorders as well as other programs targeting rare, life-threatening conditions beyond the lysosomal storage disorders. We selected these programs based on scientific rationale, unmet medical need, potential to impact disease course and strategic alignment with our corporate focus. These pipeline programs (SBC-103, SBC-104, SBC-105, and SBC-106) have the potential to offer patients and health care providers with effective therapies to treat the rare and devastating diseases targeted by these programs. As with LAL Deficiency, these diseases are characterized by significant morbidity and mortality, currently have high unmet medical need and are conditions in which protein replacement treatment has the potential to make a meaningful impact on disease progression.
History Synageva's focus on developing, delivering and commercializing products for rare diseases was established in 2008 with the appointment of Sanj K. Patel as President and CEO. The formation of Synageva and its change to a product-based company represented a considerable shift in the business strategy of the original company, AviGenics, Inc., which was founded in 1996 and was focused on the development of a novel protein production technology. Today, many of Synageva's product candidates are produced using this proprietary expression system, which is based on over 15 years of research and clinical development. Synageva's proprietary technology is highly capital efficient, allowing the rapid initiation and simultaneous advancement of multiple programs, enabling Synageva to perform initial investigations of programs in multiple diseases.
