Rare disease therapeutics specialist Synageva BioPharma raised $25 million in a round of private equity financing with all its existing lead investors. The new capital takes the total funding raised by the firm to $70 million over the last two and a half years.
Synageva’s lead clinical-stage candidate is SBC-102, a recombinant enzyme replacement therapy for treating lysosomal acid lipase (LAL) deficiency. The product has been granted orphan drug designation by FDA and the European authorities. The firm is in addition exploiting its expression platform and protein engineering expertise to build a pipeline of preclinical candidates. This comprises two enzyme replacement therapies for lysosomal storage disorders, an enzyme therapy for a metabolic disorder, and an extracellular protein for a genetic condition.
Synageva’s vector technology and expression platform allows the production of a broad range of recombinant therapeutic proteins in egg white. The firm says the platform has already undergone regulatory scrutiny through three Phase I and one global Phase II trials. The potential range of proteins that can be produced using the approach includes monoclonal antibodies, fusion proteins, cytokines, and therapeutic enzymes.
In January Synageva teamed up with Dutch firm to-BBB technologies to evaluate the latter’s technology for transporting therapeutic proteins across the blood-brain barrier into the central nervous system. The collaboration will form the basis for developing therapies against rare diseases including lysosomal storage diseases, which have a CNS component and require the therapies to be transported across the blood-brain barrier.