|[June 23, 2014]
Dimension Therapeutics and Bayer HealthCare Enter Collaboration to Develop Novel Gene Therapy for Hemophilia A
CAMBRIDGE, Mass. --(Business Wire)--
Therapeutics, a company focused on developing novel adeno-associated
virus (AAV) gene therapy treatments for rare diseases, today announced
it has entered into a collaboration with Bayer HealthCare (Bayer) for
the development and commercialization of a novel gene therapy for the
treatment of hemophilia A.
Under the terms of the agreement, Dimension will receive an upfront
payment of $20 million and will be eligible for potential development
and commercialization milestone payments of up to $232 million.
Dimension will be responsible for all pre-clinical development
activities and the Phase 1/2a clinical trial, with funding from Bayer.
Depending on the results of the Phase 1/2a clinical trial, Bayer will
conduct the confirmatory Phase 3 trial, make all regulatory submissions,
and will have worldwide rights to commercialize the potential future
product for the treatment of hemophilia A. Dimension is eligible to
receive tiered royalties based on product sales.
"Currently available replacement therapies for hemophilia A are often
administered intravenously multiple times a week and may be required for
life, depending on the severity o a patient's disease," said Thomas R.
Beck, M.D., chief executive officer of Dimension Therapeutics. "Gene
therapy offers the potential to transform the treatment of hemophilia by
inserting a correct version of the faulty gene responsible for the
disease. We are proud to partner with Bayer, a leader in the treatment
of hemophilia A, to develop a therapy with the potential to
significantly change the treatment landscape."
"Bayer is a worldwide leader in the treatment of hemophilia A and we are
highly committed to advancing innovative treatment options for patients
with hemophilia A," said Prof. Dr. Andreas Busch, member of the Bayer
HealthCare executive committee and head of global drug discovery. "We
are excited to partner with Dimension Therapeutics to jointly harness
the power of gene therapy to drive the development of new long-term
options in treating this disease."
Hemophilia is a rare bleeding disorder in which patients produce little
or no clotting factor, a protein needed for normal blood clotting, which
can cause life threatening bleeding. Hemophilia A is the most common
type of hemophilia in the United States, with approximately one in 5,000
males born with the condition1.
Dimension's AAV vector technology allows for systemic intravenous
administration of the clotting factor gene in vivo, which has
been shown in preclinical studies to target the liver, resulting in long
lasting expression of FVIII protein at therapeutic levels. Dimension's
vectors are enabled by REGENX Biosciences' proprietary NAV® technology.
About Dimension Therapeutics
Dimension Therapeutics is a gene therapy company focused on developing
novel therapies to treat rare diseases. Formed in 2013, the Dimension
team comprises biotech industry veterans and renowned thought leaders in
gene therapy and rare diseases. The company is focused on building its
adeno-associated virus (AAV) therapeutic capabilities and advancing
multiple gene therapy programs in rare diseases, and is advancing a
wholly-owned hemophilia B program towards clinical development.
Dimension's partnerships with REGENX Biosciences and the University of
Pennsylvania provide Dimension with exclusive gene therapy intellectual
property and preferred access to multiple best-in-class AAV vectors
based on REGENX's NAV® technology. Dimension has been funded by
Fidelity Biosciences and OrbiMed Advisors. For more information, please
1 National Hemophilia Foundation
[ InfoTech Spotlight's Homepage ]