Commitment and Overview
Synageva BioPharma's ultimate goal is to improve the lives of patients with rare diseases by providing access to breakthrough protein therapeutics. Patients with rare diseases are faced with substantial challenges beyond the medical problems associated with their condition, including establishing the correct diagnosis, finding medical care with expertise in their condition, and gaining access to the right therapy. As a result, developing therapies for these diseases requires a fundamental commitment from biopharmaceutical companies to help establish and be a partner in a community of care that includes patients, physicians and other health care practitioners, advocacy organizations, insurers, and the government authorities and policy makers.
The Synageva team has a deep appreciation of the commitment that is necessary to build these communities. Our team has played pivotal roles in developing and/or commercializing the majority of the enzyme replacement therapies that have been approved. We are combining this expertise with the best new approaches in translational medicine to develop our pipeline of rare disease therapies for serious and life threatening conditions.
Our development process involves identifying rare disease areas with high unmet medical need, then assessing therapeutic approaches to the disease using a variety of protein technologies (such as our proprietary Synageva Expression Platform SEP™), and ultimately matching a selected development target to a protein technology that is most suited to that target.
Nearly all of our development programs utilize SEP™ which provide a number of clear competitive advantages for developing and commercializing protein therapeutics including: high expression levels, more efficient purification processes, rapid scalability, consistency of end product across production scales, and human glycosylation patterns that provide potential therapeutic advantages.
Strategy
Synageva’s business strategy is centered on developing a broad portfolio of rare disease therapies and bringing them to market. We have several compounds in development, each for a different rare disease that currently has no effective therapies. Given our substantial internal expertise, Synageva will independently develop and commercialize several of these programs.
Sustainability of our rare disease portfolio will be supported by two efforts that partner our key technology and product assets for large market opportunities:
- Product and Technology Partnerships with other biopharmaceutcial companies
a. We are actively discussing partnerships opportunities
for several later stage proteins targeting larger
specialty markets
b. We are actively discussing deals to express and
develop other companies’ proteins and leverage the
fundamental advantages of the SEP™
- Government Biodefense Contracts
a. The SEP’s robust scalability, flexibility, and ability to
disaggregate upstream and downstream production,
provide significant benefits in medical counter measure
preparation and readiness
b. Synageva is applying to select BAARDA and DOD
proposals where the SEP™ would add significant value