Renowned Rare-Disease Drug Developer Emil Kakkis, M.D., Ph.D., to Join Actio Biosciences Board of Directors

SAN DIEGO--(BUSINESS WIRE)--Actio Biosciences, a biotechnology company leveraging a novel platform approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced that Emil Kakkis, M.D., Ph.D., CEO, president and director of Ultragenyx, has joined the company’s board of directors. Dr. Kakkis, a visionary in rare disease drug development, founded Ultragenyx in 2010 to create a company that is uniquely built around a deep and meaningful engagement with patients and their caregivers to fully understand their needs.

“Emil’s passionate dedication to developing therapies for rare diseases has long been an inspiration to me, and was a driving factor and motivation for us to start Actio,” said David Goldstein, Ph.D., co-founder and CEO of Actio. “From his pioneering work in enzyme replacement therapy to the remarkable portfolio of rare-disease programs he has built at Ultragenyx, Emil has shown us just how much is possible in the development of life-changing medicines for many of the rarest and most severe diseases. This work has inspired a new age of discovery for rare disease medicines, and I am thrilled to welcome him to Actio’s board of directors.”

“Having seen the impact that the development of innovative medicines can have on patients living with rare diseases and their families, I am motivated by Actio’s focus on developing small molecule treatments that effectively target the precise underlying biology and cause of rare diseases,” said Dr. Kakkis. “I am pleased to join the company’s board of directors as we work toward a collective goal focused not only on addressing rare conditions with no treatments today, but potentially addressing more common diseases that lack effective treatment options.”

Actio’s chairperson, George Golumbeski, Ph.D., added, “Few individuals in our industry possess a combination of personal commitment, scientific integrity and exceptional business acumen that Emil has fostered throughout his career, and we are honored to welcome him to the Actio board. His insights, expertise and passion will be invaluable to the company’s long-term commitment to developing first in class life-changing medicines.”

Dr. Kakkis began his career at the Harbor-UCLA Medical Center, where he led work to develop an enzyme replacement therapy for the rare disorder MPS I. He joined BioMarin in 1998, where he guided the development and approval of three treatments for rare diseases and contributed to the development of approved or development-stage products for four other rare indications. He has worked to advance the cause of rare disease treatment through advocacy on policy issues by founding and supporting the EveryLife Foundation for Rare Diseases, a nonprofit foundation dedicated to the acceleration of biotech innovation for rare diseases through practical and scientifically sound improvements to development strategies, regulatory policy and law. Dr. Kakkis received combined M.D. and Ph.D. degrees from the UCLA Medical Scientist Program and received the Bogen Prize for his research upon graduation. He completed a pediatrics residency and a Medical Genetics Training Fellowship at Harbor-UCLA Medical Center, where he was an assistant professor of pediatrics and initiated the enzyme therapy program for MPS I. Dr. Kakkis has received numerous awards including the Life Science Leadership Pantheon award from California Life Sciences, a Lifetime Achievement Award from the National MPS Society, the Roscoe O Brady Award for innovation from the WORLD Symposium and the Henri Termeer Visionary Leader Award from BIO, the biotechnology innovation organization.

About Actio Biosciences

Actio Biosciences is leveraging advances in precision medicine to develop new therapeutics that target shared genetics in rare and common diseases—bringing meaningful medicines from one to many. Applying its expertise in genetics, drug discovery and data sciences, Actio seeks to identify programs where both biological and technical risk can be minimized to streamline the drug development process and bring forward exceptionally potent and precisely targeted therapeutics. Founded in October 2021, the San Diego-based company is led by leaders in genetics and drug development and backed by top healthcare investors. For more information, please visit ActioBiosciences.com.