Voyager Therapeutics Announces Publication from the California Institute of Technology of a Second-Generation Gene Therapy Ca...
July 17 2017 - 7:04AM
A single, intravenous administration of a novel
AAV capsid provided up to 100-fold increase in gene transfer to the
central nervous system in a preclinical model compared to the
historical standard AAV9
Voyager Therapeutics, Inc. (NASDAQ:VYGR), a clinical-stage gene
therapy company developing life-changing treatments for severe
neurological diseases, today announced the publication in Nature
Neuroscience (link to publication) of new preclinical data from
ongoing efforts of Dr. Benjamin Deverman, Professor Viviana
Gradinaru and the Gradinaru Laboratory at the California Institute
of Technology (Caltech) to develop novel adeno-associated viral
(AAV) capsids that efficiently cross the blood-brain barrier and
widely transduce, or transfer, genes into the central nervous
system (CNS) after intravenous administration. From these
efforts, a new, second-generation AAV capsid provided up to a
100-fold increase in the transduction of the CNS in an adult
preclinical model over the historical standard, AAV9, as compared
with the first-generation capsid reported last year1 by the
Gradinaru group that provided a more than 40-fold improvement over
AAV9.
“The ongoing work by Dr. Deverman, Professor
Gradinaru and the Gradinaru Laboratory at Caltech continues to
generate exciting AAV variant capsids for application to the
treatment of CNS diseases,” said Dinah Sah, Ph.D., chief scientific
officer at Voyager Therapeutics. “Key translational studies
in non-human primates are underway by Voyager to evaluate these AAV
variant capsids that have the potential to transform our ability to
deliver gene therapies to the CNS. We are pleased to be
partnered with the Gradinaru group at Caltech to further advance
this technology.”
Voyager obtained a co-exclusive license to the
Caltech novel AAV capsids, intellectual property and related
technology in September 2016. The license agreement covers
all fields of use and includes novel AAV capsids that have
demonstrated enhanced crossing of the blood-brain barrier for the
potential treatment of CNS diseases following systemic
administration of an AAV gene therapy vector.
About Voyager Therapeutics
Voyager Therapeutics is a clinical-stage gene
therapy company developing life-changing treatments for severe
neurological diseases. Voyager is committed to advancing the
field of AAV gene therapy through innovation and investment in
vector engineering and optimization, manufacturing and dosing and
delivery techniques. The company’s pipeline focuses on severe
neurological diseases in need of effective new therapies, including
advanced Parkinson’s disease, a monogenic form of ALS, Huntington’s
disease, Friedreich’s ataxia, 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,”
“undoubtedly,” “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
deliver gene therapy to the CNS, its ability to advance its
AAV-based gene therapies into, and successfully complete, clinical
trials, and its ability to continue to develop its product engine,
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 Annual Report on Form 10-K 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.
_____________________
1 Deverman, BE, et al. (February 2016) Nature Biotechnology
34 (2): 204-211
Investor Relations:
Matt Osborne
Vice President 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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