The annual California Life Sciences Pantheon Awards celebrate companies and individuals making transformative contributions to our life sciences ecosystem. This year we were honored to celebrate our own CEO, Emil Kakkis, M.D., Ph.D., a co-recipient of the 2021 California Life Sciences Pantheon Leadership award. Emil was recognized alongside Frances H. Arnold, Ph.D. of CalTech, who was awarded the 2018 Nobel Prize for Chemistry for her work on directed evolution of enzymes. Emil and Dr. Arnold were selected from a group of esteemed peers, who each have an established track record of devoting their career to meaningfully improving people’s lives through science.
A medical geneticist by training, Emil has been a pioneer in developing new treatments for rare disease. He founded both the EveryLife Foundation for Rare Diseases in 2009 and Ultragenyx in 2010, to support his personal mission of getting effective therapies to children and adults living with untreated rare disease.
In 2020 alone, Ultragenyx received two FDA approvals – one for the treatment of tumor-induced osteomalacia (TIO), and another for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD). In fact, the company has a diversified pipeline that now includes three approved therapies in four indications and more than 20 programs in research and development for diseases with no sufficient treatments.
Throughout his career, Emil has been recognized for his work in developing novel treatments and working on policy issues affecting rare disease drug development. In 2019, he received the Biotechnology Innovation Organization (BIO) Henri A. Termeer Biotechnology Visionary Award for his efforts to focus the biotech industry on new treatments for rare and ultra-rare diseases. He’s led significant work on establishing new approaches to clinical analysis and study design in rare disease that accommodate small n-counts and heterogenous disease manifestations. Before founding Ultragenyx, Emil was the chief medical officer and development consultant at BioMarin where he guided the development and approval of multiple rare disease therapies.
Ultragenyx was also recognized as a finalist for the 2021 Company of the Year Award, which ultimately went to Gilead. The acknowledgement of our company and our leadership, particularly during a pandemic impacting all parts of our society and our industry, feels particularly meaningful right now.
This was the first year the Pantheon awards were decided in part by public vote – and we’re proud that this achievement is also a reflection of the passion and commitment of our team at Ultragenyx and the support from the broader life sciences community.
Congratulations to Emil, Dr. Arnold, and all of the Pantheon award recipients! Thank you to California Life Sciences for this accolade for our entire organization. And thank you to the patients and communities who inspire us and work with us every day as we lead the future of rare disease medicine.
Carolyn Wang is senior vice president of global corporate communications at Ultragenyx.