Synageva BioPharma Corp. is recruiting patients to participate in a clinical trial that evaluates SBC-102 as an enzyme replacement therapy for Lysosomal Acid Lipase (LAL) Deficiency. For more information, please visit clinicaltrials.gov, 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 life saving therapies. We select innovative therapeutic approaches for rare diseases based on unmet medical need and ability to have a substantial impact on disease course and patient health. We have several orphan therapeutics in development including two enzyme replacement therapies (ERTs) for lysosomal storage disorders (LSDs) and two other programs for life threatening conditions.

Key elements for our strategy include:

• Advance SBC-102 toward regulatory approval and successful commercialization for the treatment of LAL Deficiency

• Continue to move pipeline programs forward 

• 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.