New effort will search for potential therapies targeting the most
common mutation of cystic fibrosis
CAMBRIDGE, Mass. & BETHESDA, Md.--(BUSINESS WIRE)--Genzyme,
a Sanofi company (EURONEXT: SAN and NYSE: SNY), and Cystic Fibrosis
Foundation Therapeutics Inc., the nonprofit affiliate of the Cystic
Fibrosis Foundation, today announced a research agreement to support the
discovery of new drugs to treat people with the most common mutation
found in patients with CF, Delta F508.
“Together with the CF Foundation, we look forward
to working to accelerate the pace of discovery on behalf of CF patients
around the world.”
People with cystic fibrosis, a genetic disease, experience a cascade of
symptoms that can lead to life-threatening lung infections and premature
death.
The program’s focus is to identify compounds known as “correctors,”
which may aid in the ability of the malfunctioning CFTR protein found in
CF patients to operate correctly. In the Delta F508 mutation, the CFTR
protein does not move to its proper place at the cell surface, impeding
the flow of fluids into the airways. Nearly 90 percent of people with CF
have at least one copy of the Delta F508 mutation.
In this collaboration, researchers will evaluate different compound
libraries for correctors for Delta F508, and will take advantage of the
vast compound libraries of both Genzyme and Sanofi. The research will
take place throughout several Genzyme and Sanofi R&D facilities
globally. Genzyme brings to the collaboration more than 20 years’
experience exploring treatments for people living with CF. The company’s
efforts have ranged from improved molecular diagnostics to clinical
trials with a gene therapy, and have included past collaboration with
the CF Foundation in the area of drug discovery.
“We are delighted to enter into a research collaboration with Genzyme, a
company that has long dedicated itself to improving the lives of people
with rare diseases,” said Robert J. Beall, Ph.D., president and CEO of
the CF Foundation. “Genzyme’s capabilities and resources will help the
CF Foundation accelerate its effort to find drugs to treat the most
common mutation in CF and have the greatest impact on those with this
disease.”
“While there has been great momentum recently in cystic fibrosis
research, there is still great unmet need,” said Genzyme’s president and
CEO David Meeker, MD. “Together with the CF Foundation, we look forward
to working to accelerate the pace of discovery on behalf of CF patients
around the world.”
About the Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the world’s leader in the search for a
cure for cystic fibrosis. The Foundation funds more CF research than any
other organization, and nearly every CF drug available today was made
possible because of Foundation support. Based in Bethesda, Md., the
Foundation also supports and accredits a national care center network
that has been recognized by the National Institutes of Health as a model
of care for a chronic disease. The CF Foundation is a donor-supported
nonprofit organization. For more information, go to www.cff.org.
About Genzyme, a Sanofi Company
Genzyme has pioneered the development and delivery of transformative
therapies for patients affected by rare and debilitating diseases for
over 30 years. We accomplish our goals through world-class research and
with the compassion and commitment of our employees. With a focus on
rare diseases and multiple sclerosis, we are dedicated to making a
positive impact on the lives of the patients and families we serve. That
goal guides and inspires us every day. Genzyme’s portfolio of
transformative therapies, which are marketed in countries around the
world, represents groundbreaking and life-saving advances in medicine.
As a Sanofi company, Genzyme benefits from the reach and resources of
one of the world’s largest pharmaceutical companies, with a shared
commitment to improving the lives of patients. Learn more at www.genzyme.com.