Other Novel Proteins in Pipeline

Synageva is developing several additional rare disease therapeutics:

• There are 2 additional ERT programs in development for LSDs without effective treatments
• There are 2 non LSD programs for life threatening genetic conditions in development.  

Our development strategy is not based on a certain type of protein or disease, and it is not restricted to expression system. We select rare disease targets based on high unmet medical need and the probability that a targeted therapy will have a substantial impact on disease course and improve patient health. If a disease target meets these criteria, we implement the best possible technology to express and develop the protein (the vast majority of the time, this is our SEPTM).

Importantly, the majority of our development programs, including SBC-102, have potential applications to help patients beyond the initial indication for which we are developing them.