Synageva BioPharma is a publicly traded biopharmaceutical company with headquarters in Lexington, MA. The company focuses on the discovery, development, and commercialization of therapeutic products for people suffering from rare diseases for which there is high unmet medical need.
Synageva’s most advanced pipeline programs consist of enzyme replacement therapies for lysosomal storage diseases (LSDs), which are rare, genetic diseases with devastating complications. The company’s lead program, sebelipase alfa, is in global Phase 3 clinical trials for lysosomal acid lipase deficiency (or LAL Deficiency). Sebelipase alfa has been granted orphan designation by the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the Japanese Ministry of Health, Labour and Welfare. Additionally, sebelipase alfa received fast track designation by the FDA, and Breakthrough Therapy designation by the FDA for LAL Deficiency presenting in infants.
Synageva’s next program, SBC-103, is being developed by the company as an enzyme replacement therapy for mucopolysaccharidosis IIIB (or MPS IIIB, also known as Sanfilippo B syndrome). Synageva’s additional pipeline programs include other proteins targeting rare diseases at various stages of preclinical development. These diseases are characterized by significant morbidity and mortality and are selected based on scientific rationale, high unmet medical need, potential to impact disease course and strategic alignment with our corporate focus.
To ensure that these therapeutic candidates reach patients in need, Synageva has recruited a team with a proven track record of manufacturing, discovery, development, and commercial success within rare diseases.
Our mission is to deliver breakthrough medicines globally for patients suffering from rare, devastating diseases. Achieving this will result in a sustainable business that can continue to positively impact the lives of patients and their families, healthcare providers and our employees. Our success depends upon our ability to seek and retain individuals who share our integrity, drive, energy and passion.