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. Everyone at Synageva shares a passion and commitment to improving the lives of patients with rare diseases. Our team is drawn from leading biotech companies and have 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 enzyme replacement therapies for other lysosomal storage disorders as well as programs for rare, life-threatening conditions beyond the lysosomal storage disorders. These protein therapeutic programs were selected based on scientific rationale, unmet medical need, potential to impact disease course and strategic alignment with Synageva’s corporate focus. These pipeline programs (SBC-103, SBC-104, SBC-105, and SBC-106) have the potential to offer patients and health care providers 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 have a meaningful impact on disease progression.


History

Synageva’s business focus on products for rare diseases was established in 2008 with the appointment of Sanj K. Patel, President and CEO of Synageva. The formation of Synageva and its change to a product-based company focused on ultra rare diseases with unmet medical need 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.

Patients and Families: Click here for more information on LAL Deficiency, or contact Synageva here.

Physicians: Click here to contact Synageva if you have a patient with LAL Deficiency or if you are interested in participating in research and clinical trials related to LAL Deficiency.