Voyager Therapeutics’ Novel Capsids Demonstrate Robust Delivery Across the Blood Brain Barrier, Widespread CNS Transduction...
Voyager Therapeutics, Inc. (NASDAQ: VYGR), a clinical-stage
gene therapy company focused on developing life-changing treatments
for serious neurological diseases, today will present new
preclinical data demonstrating high transduction efficiency of the
company’s novel adeno-associated virus (AAV) capsids in the central
nervous system (CNS) after intravenous dosing in non-human
primates. Data will be presented at the 24th Annual Meeting of the
American Society of Gene and Cell Therapy (ASGCT)
taking place virtually May 11-14, 2021.
“Efficient targeting of the CNS, including the ability to
penetrate the blood brain barrier and transduce target cells, have
represented a significant challenge in gene therapy hindering the
field’s ability to effectively address many serious neurological
diseases,” Omar Khwaja, M.D., Ph.D., Chief Medical Officer and Head
of Research & Development at Voyager. “We’ve now been able to
show that our novel capsid variants not only cross the blood brain
barrier but achieve widespread transduction of multiple brain
regions including the cortex, thalamus, striatum, cerebellum,
brainstem and spinal cord. We believe these capsids can provide a
new way forward to treat a broad range of CNS indications,
potentially at significantly lower doses than currently available
Data will be shared in an oral presentation titled, “RNA-driven
Evolution of AAV Capsid Libraries Identifies Variants with
High Transduction Efficiency in Non-Human Primate
Central Nervous System,” (Abstract #51) by Mathieu
Nonnenmacher, Ph.D., Director and Head of Capsid Discovery, showing
that several capsid variants derived from the company’s directed
evolution RNA-based screening platform, TRACERTM (Tropism
Redirection of AAV by Cell-type-specific Expression of RNA),
demonstrated significantly enhanced activity relative to AAV9, the
most commonly used vector for CNS gene therapy.
- A subset of capsids showed a 10-fold
or higher improvement in transduction of the brain and spinal cord,
compared to AAV9.
- The most efficient capsid identified
in the work to be presented, TRACER 9P801, displayed more than
1,000-fold higher transgene expression in the brain and 100-fold
higher transgene expression in the spinal cord.
- The overall tolerability of 9P801
was favorable and no toxicity was observed in the liver, spinal
cord or dorsal root ganglia (DRG).
- Immunohistochemical analysis
indicated that 9P801 displayed predominant neuronal tropism and
achieved widespread transduction of multiple brain regions
including the cortex, thalamus, putamen and brainstem.
- The TRACER platform generates large
data sets on engineered capsid performance in a relevant primate
species suitable for in silico approaches to optimizing capsid
selection for specific cellular targeting and tropism.
Voyager’s TRACER system is a broadly-applicable, RNA-based
functional screening platform that allows for rapid in vivo
evolution of AAV capsids with cell-specific transduction properties
in wild-type animals. TRACER candidates were tested individually by
low dose intravenous injection and their tropism for the CNS
analyzed by measuring transgene RNA expression, viral DNA
biodistribution and immunohistochemistry.
Further information about the Company’s TRACER platform and
pipeline, including novel capsid-enabled new programs, will be
shared at an upcoming investor and analyst event in July 2021.
About Voyager Therapeutics
Voyager Therapeutics is a clinical-stage gene therapy
company focused on developing life-changing treatments for serious
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. For more information on Voyager
Therapeutics, please visit the company’s website
at www.voyagertherapeutics.com or
follow @VoyagerTx on Twitter and LinkedIn.
Voyager Therapeutics® is a registered trademark, and TRACER™ is
a trademark, of Voyager Therapeutics, Inc.
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 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 initiation, timing, progress, activities, goals
and reporting of results of its research and development programs;
Voyager’s ability to present preclinical data on its early pipeline
progress and novel capsid discovery efforts in non-human primates
at scientific meetings and other presentations; Voyager’s ability
to continue to develop its novel AAV capsids; the ability of
Voyager’s novel capsids to treat CNS diseases and to treat with
lower doses than currently available AAV serotypes, in each
instance 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 severity and length of the COVID-19
health crisis; the initiation and conduct of preclinical studies
involving our novel capsid program; the continued development of
the Voyager gene therapy platform and Voyager’s TRACER system; the
applicability of novel capsids identified by the TRACER system to a
broad spectrum of CNS and non-CNS disease indications; Voyager’s
scientific approach and general development progress; the ability
to attract and retain highly skilled and experienced
scientists and technicians; the ability to create and protect
intellectual property related to Voyager’s development of novel
capsids; and the availability of resources necessary to conduct the
novel capsid development program, including access to sufficient
number and types of primates suitable for participation in ongoing
and planned preclinical research activities.
These statements are also subject to a number of material risks
and uncertainties that are described in Voyager’s Annual Report on
Form 10-K for the year ended December 31, 2020 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.
SteeleVerge Scientific Communications202.930.4762 x
Voyager Therapeutics (NASDAQ:VYGR)
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