Company Description (as filed with the SEC)
We are a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of therapeutic products for patients with life-threatening rare diseases and unmet medical needs. We have several protein therapeutics in our pipeline, including two enzyme replacement therapies for lysosomal storage disorders (LSDs) and additional programs for other life-threatening genetic conditions for which there are currently no approved treatments. Our business focus on products for rare diseases was established in 2008 with the appointment of Sanj K. Patel as President and Chief Executive Officer and his redirection of the company. This change represented a substantial shift in the business strategy of the company, formerly known as AviGenics, Inc., which was focused on the development of a novel protein production technology for biosimilars. ... More ...