Synageva BioPharma Corp. is recruiting patients to participate in global clinical trials that evaluate sebelipase alfa (SBC-102) as an enzyme replacement therapy for Lysosomal Acid Lipase (LAL) Deficiency. For more information, please visit, or click here if you would like to contact Synageva regarding LAL Deficiency. 

Overview / Rare Disease Pipeline

Our strategy is to discover, develop and deliver medicines that make a meaningful impact on the lives of patients. We select innovative therapeutic approaches for rare diseases based on high unmet medical need and ability to have a substantial impact on disease course and patient health. We have several therapeutics in development including enzyme replacement therapies (ERTs) for lysosomal storage disorders (LSDs) and other programs for life threatening conditions.

Key elements of our strategy include:

  • Advance sebelipase alfa toward regulatory approval and commercialization for the treatment of LAL Deficiency

  • Advance SBC-103 toward human clinical trials for the treatment of MPS IIIB or Sanfilippo B syndrome. 

  • Integrate new candidates into our pipeline

Our research laboratory in Lexington, MA, has protein engineering capabilities and expertise in the rapid production of recombinant proteins for biological characterization. These capabilities allow us to engineer and manufacture proteins that are either identical to the defective protein or incorporate unique modifications to enhance the biological activity and/or therapeutic usefulness of the defective protein.