Voyager Therapeutics Presents Positive Preclinical Data Supporting CNS-Directed GBA1 Gene Therapy at Virtual Congress of the European Society of Gene and Cell Therapy
October 20 2021 - 8:54AM
Voyager Therapeutics, Inc. (Nasdaq: VYGR), a gene therapy company
developing life-changing treatments and next-generation
adeno-associated virus (AAV) platform technologies, today announced
the presentation of preclinical data demonstrating a single
intravenous (IV) dose of its novel GBA1 gene replacement therapy
achieved widespread distribution in the central nervous system
(CNS) and peripheral tissues of mice, and substantially raised
levels of the GCase enzyme in both in vivo and in vitro models. The
results are available in an on-demand poster session throughout the
virtual Congress of the European Society of Gene and Cell Therapy
(ESGCT) Oct. 19-22.
“These encouraging preclinical results demonstrate that a single
IV dose of GBA1 transgenes transported by a blood-brain barrier
penetrant AAV capsid can effectively deliver therapeutically
relevant levels of GBA1 protein to multiple brain regions of mice.
Notably, the increase over endogenous GCase levels in the range of
300% - 660% is promising given increased GCase levels of 30-50
percent is anticipated to be clinically impactful,” said Glenn
Pierce, M.D., Ph.D., interim chief scientific officer of Voyager.
“These data show the potential that blood-brain barrier penetrant
capsids may have to address severe neurological diseases as
compared to loco-regional injection of AAV into the CNS and offer a
solid foundation for continued investigation of this novel
approach.”
Data presented at ESGCT demonstrates that AAV gene transfer of
GBA1 transgenes in the cells of mouse models significantly reduced
GCase enzyme activity and the accumulation of glycosphingolipids
compared to non-AAV delivery. The treatment was well tolerated at
all doses and resulted in the safe delivery of therapeutically
relevant levels of GBA1 protein to multiple brain regions in mice.
Key findings included:
- AAV vector optimized GBA1 achieved widespread distribution and
sustained correction of GCase activity after IV administration in
WT mice.
- Consistent voltage gated (VG) distribution and GCase activity
was observed across the CNS following IV delivery.
- IV administration of BBB-penetrant capsid optimized GBA1
transgenes at a high dose resulted in approximately a 300% – 660%
increase over endogenous GCase levels, suggesting the potential for
dose reductions given that increased GCase activity of 30% - 50% is
anticipated to be clinically impactful.
- Results validate GBA1 transgene-level DRG tissue-detargeting
approach in vitro.
About GBA1 MutationsThe GBA1 gene encodes the
lysosomal enzyme β-glucocerebrosidase (GCase) and GBA1 loss of
function results in GCase deficit and cellular build-up of
glycosphingolipid-substrate. In patients with GBA1 mutations,
decreased levels of GCase can result in accumulations of lipids in
the body, driving a number of diseases include Gaucher disease,
Parkinson’s disease, and Lewy body dementia. While enzyme
replacement therapies can have clinical impact, they fail to
adequately cross the blood-brain barrier to address disease in the
CNS.
About Voyager TherapeuticsVoyager Therapeutics
(Nasdaq: VYGR) is leading the next generation of AAV gene therapy
to unlock the potential of the technology to treat devastating
diseases. Proprietary capsids born from the Company’s TRACER
screening platform are powering a rich early-stage pipeline of new
and second-generation programs and may elevate the field to
overcome the limitations of conventional gene therapy vectors
across neurologic disorders and other therapeutic areas.
voyagertherapeutics.com
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Forward-Looking StatementsThis 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 laws. The use of words
such as “may,” “might,” “will,” “would,” “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 positive
nature of the preclinical data presented at the Annual Congress of
the European Society of Gene & Cell Therapy (ESGCT); the
ability of a single intravenous (IV) dose of novel GBA1- gene
replacement therapy to achieve widespread distribution in the
central nervous system (CNS) and peripheral tissues of wild type
(WT) mice; the potential that blood-brain barrier penetrant capsids
may have to address severe neurological diseases; the ability of
intravenously-administered gene replacement therapy to
substantially raise levels of the GCase enzyme in both in vivo and
in vitro mice models, the validation of GBA1 transgene-level DRG
tissue-detargeting approach in vitro; and the potential for such
preclinical data to support the potential of a novel therapeutic
approach for Gaucher disease, Parkinson’s disease and Lewy body
dementia are forward looking.
All forward-looking statements are based on estimates and
assumptions by Voyager’s management that, although Voyager believes
such forward-looking statements 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. Such risks and uncertainties
include, among others,; the continued development of various
technology platforms, including Voyager’s TRACER platform;
Voyager’s scientific approach and general development progress,
including in particular with respect to the GBA1 program; the
ability to develop, manufacture and optimize capsids that
effectively deliver relevant levels of GBA1 protein to multiple
brain regions the ability to attract and retain talented
contractors and employees; the ability to create and protect
intellectual property; the sufficiency of cash resources; 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 subsequent filings with the
Securities and Exchange Commission. All information in the press
release is as of the date of this press release, and any
forward-looking statement speaks only as of the date on which it
was made. Voyager undertakes no obligation to publicly update or
revise this information or any forward-looking statement, whether
as a result of new information, future events or otherwise, except
as required by law.
Voyager
ContactsInvestorsInvestors@voyagertherapeutics.comMediaScott
Santiamo ssantiamo@vygr.com
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