Renowned Rare-Disease Drug Developer Emil Kakkis, M.D., Ph.D., to Join Actio Biosciences Board of Directors
2024年7月17日 - 9:30PM
ビジネスワイヤ(英語)
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.
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Dan Budwick, 1AB dan@1abmedia.com