Voyager Therapeutics and CHDI Foundation Collaborate to Develop Novel Gene Therapy for Huntington’s Disease
September 12 2016 - 7:05AM
Voyager Therapeutics, Inc. (NASDAQ:VYGR), a clinical-stage gene
therapy company developing life-changing treatments for severe
diseases of the central nervous system (CNS) today announced a
research collaboration with CHDI Foundation, Inc. (CHDI) to advance
Voyager’s VY-HTT01 program, an adeno-associated virus
(AAV)-mediated gene-silencing therapy for Huntington’s
disease. The collaboration builds upon a previous
collaboration between CHDI and Sanofi Genzyme and includes funding
from CHDI to help support preparation for and filing of an
investigational new drug application, as well as completion of a
Phase 1 clinical trial. CHDI will be reimbursed for its
support of Voyager’s program upon VY-HTT01 achieving certain
commercial milestones.
“Voyager is pleased to collaborate with CHDI to
help advance our gene therapy program for Huntington’s disease,”
said Steven Paul, M.D., president and chief executive officer of
Voyager Therapeutics. “The effort and expertise that Voyager
and Sanofi Genzyme continue to commit to the VY-HTT01 program, now
further strengthened with CHDI’s extensive experience in
Huntington’s disease research, puts us in a strong position to
advance the clinical development of a potential disease-modifying
medicine for patients suffering from Huntington’s disease.”
“CHDI is committed to identifying and
facilitating the development of a diverse pipeline of therapeutic
strategies for Huntington’s disease,” said Robi Blumenstein,
president of CHDI Management, Inc. “Voyager is a leader in gene
therapy, particularly for central nervous system diseases, and we
are confident that their expertise will allow us to further extend
the important progress that Sanofi Genzyme has already made in our
collaboration.”
About Huntington’s Disease
Huntington’s disease is an inherited
neurodegenerative disorder caused by a mutation in the huntingtin
gene. The defect causes a DNA sequence called a CAG repeat to
occur many more times than normal. Each child of a parent with a
mutation in the huntingtin gene has a 50% chance of inheriting the
mutation. As a result of carrying the mutation, an
individual's brain cells degenerate leading to behavioral,
cognitive and motor impairments that, over the course of the
disease, significantly reduce the individual's quality of life and
ultimately cause death within 15 to 25 years of overt clinical
onset. It is estimated that around one person in 10,000
carries the mutated huntingtin gene. There are currently no
therapeutics approved that slow the progression of Huntington's
disease.
About CHDI
CHDI Foundation, Inc. is a privately-funded
nonprofit biomedical research organization that is exclusively
dedicated to rapidly developing therapies that slow the progression
of Huntington’s disease. As a collaborative enabler, CHDI
seeks to bring the right partners together to identify and address
critical scientific issues and move drug candidates to clinical
evaluation as quickly as possible. CHDI scientists work
closely with a network of more than 700 researchers in academic and
industrial laboratories around the world in the pursuit of these
novel therapies, providing strategic scientific direction to ensure
that our common goals remain in focus. More information about
CHDI can be found at www.chdifoundation.org.
About Voyager Therapeutics
Voyager Therapeutics is a clinical-stage gene
therapy company developing life-changing treatments for severe
diseases of the CNS. Voyager is committed to advancing the
field of AAV (adeno-associated virus) gene therapy through
innovation and investment in vector engineering and optimization,
manufacturing and dosing and delivery techniques. The
Company’s pipeline is focused on severe CNS diseases in need of
effective new therapies, including advanced Parkinson’s disease, a
monogenic form of amyotrophic lateral sclerosis (ALS), Friedreich’s
ataxia, Huntington’s disease, spinal muscular atrophy (SMA),
frontotemporal dementia, Alzheimer’s disease and severe, chronic
pain. Voyager has broad strategic collaborations with Sanofi
Genzyme, the specialty care global business unit of Sanofi, and the
University of Massachusetts Medical School. Founded by
scientific and clinical leaders in the fields of AAV gene therapy,
expressed RNA interference and neuroscience, Voyager Therapeutics
is headquartered in Cambridge, Massachusetts. For more
information, please visit www.voyagertherapeutics.com. Follow
Voyager on LinkedIn.
Forward-Looking Statements
This press release contains forward-looking
statements for the purposes of the safe harbor provisions under The
Private Securities Litigation Reform Act of 1995 and other federal
securities law. The use of words such as “may,” “might,” “will,”
“should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,”
“project,” “intend,” “future,” “potential,” or “continue,” and
other similar expressions are intended to identify forward-looking
statements. For example, all statements Voyager makes
regarding the initiation, timing, progress and reporting of results
of its preclinical programs and clinical trials and its research
and development programs, its ability to advance its AAV-based gene
therapies into, and successfully complete, clinical trials, its
ability to continue to develop its product engine, its ability to
add new programs to its pipeline, its expected cash, cash
equivalents and marketable securities at the end of the fiscal year
and anticipation for how long expected cash, cash equivalents and
marketable securities will last, and the timing or likelihood of
its regulatory filings and approvals, are forward looking.
All forward-looking statements are based on estimates and
assumptions by Voyager’s management that, although Voyager believes
to be reasonable, are inherently uncertain. All
forward-looking statements are subject to risks and uncertainties
that may cause actual results to differ materially from those that
Voyager expected. These statements are also subject to a
number of material risks and uncertainties that are described in
Voyager’s most recent Quarterly Report on Form 10-Q filed with the
Securities and Exchange Commission, as updated by its future
filings with the Securities and Exchange Commission. Any
forward-looking statement speaks only as of the date on which it
was made. Voyager undertakes no obligation to publicly update
or revise any forward-looking statement, whether as a result of new
information, future events or otherwise, except as required by
law.
Investor Relations:
Matt Osborne
Head of Investor Relations & Corporate Communications
857-259-5353
mosborne@vygr.com
Media:
Katie Engleman
Pure Communications, Inc.
910-509-3977
Katie@purecommunicationsinc.com
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