Voyager Therapeutics to Present Broad Set of Translational Data Supporting IV-Delivered, CNS Gene Therapy Programs Advancing Toward Clinical Trials at the ASGCT 27th Annual Meeting
22 Abril 2024 - 3:30PM
Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company
dedicated to advancing neurogenetic medicines, today announced that
it will present a broad set of data related to its TRACER™ capsid
discovery platform and TRACER-driven gene therapy programs across
12 oral and poster presentations at the upcoming American Society
of Gene & Cell Therapy’s (ASGCT) 27th annual meeting taking
place in Baltimore, May 7-11. The findings span the cross-species
and receptor-driven translatability, manufacturability, and overall
performance of Voyager’s novel IV-delivered CNS capsids, which are
enabling multiple gene therapies to advance towards clinical trials
next year.
“Voyager’s novel TRACER-derived capsids underlie 13 partnered
programs and three wholly-owned programs to enable IV-delivery of
gene therapies for diseases of the central nervous system. Three of
those programs now have development candidates selected, and we see
the potential for them to enter clinical trials next year,” said
Todd Carter, Ph.D., Chief Scientific Officer of Voyager
Therapeutics. “At ASGCT, we will highlight data on the
second-generation capsids that have enabled this progress, as well
as preclinical data from our wholly-owned Alzheimer’s disease and
ALS programs, and multiple studies of cross-species and
receptor-based data that increase our confidence in the potential
for these capsids to work in humans.”
Second-Generation Capsids
- Oral Presentation: Continued directed evolution of VCAP-101 and
VCAP-102 identifies second generation capsids with increased brain
tropism in non-human primates and mice (#119). Tyler Moyer, Ph.D.,
Senior Scientist I, Novel Capsid Discovery. Wednesday, May 8, 2024,
4:30 p.m. – 4:45 p.m. ET
Advancements in Wholly-Owned CNS Gene Therapy
Programs
- Intravenous administration of BBB-penetrant, MAPT-Silencing,
AAV gene therapy provides broad and robust CNS Tau lowering in
tauopathy mouse models (#1602). Hechen Bao, Ph.D., Scientist I, and
Shiron Lee, M.S., Senior Research Associate I, Neuroscience.
Friday, May 10, 2024, 12:00 p.m. ET
- Intravenous delivery of AAV gene therapy for the treatment of
SOD1-ALS provides broad SOD1 lowering in NHP (#1647). Michael
Grannan, Ph.D., Associate Director, Neuroscience. Friday, May 10,
2024, 12:00 p.m. ET
Mechanism of Action and Cross-Species
Translation
- Identification and characterization of a highly conserved cell
surface receptor utilized by engineered BBB-penetrant AAV capsids
with enhanced brain tropism in non-human primates and mice (#975).
Brett Hoffman, Ph.D., Senior Scientist II, Novel Capsid Discovery.
Thursday, May 9, 2024, 12:00 p.m. ET
- Establishment of a predictive transcytosis model to
recapitulate capsid-receptor interaction and phenotype of
BBB-penetrant AAV variants (#976). Ishan Shah, Ph.D., Senior
Scientist I, Vector Genome. Thursday, May 9, 2024, 12:00 p.m.
ET
- Evaluation of cross-species expression across four species and
cellular tropism of VCAP-102, an engineered blood-brain
barrier-penetrating AAV derived capsid from TRACER Platform screens
(#1452). Hamza Khalid, Research Associate II, Histology. Friday,
May 10, 2024, 12:00 p.m. ET
- High-resolution quantitative analysis of multiple AAV capsids
in rodent and primate models using multiplexed reporter protein
tagging platform (#511). Matthew Child, M.S., Principal Research
Associate, Novel Capsid Discovery. Wednesday, May 8, 2024, 12:00
p.m. ET
Reduced Immunogenicity, Developability, and
Manufacturing
- Discovery of TRACER AAV capsids escaping pre-existing
neutralizing antibodies (#973). Damien Maura, Ph.D., Senior
Scientist II, Novel Capsid Discovery. Thursday, May 9, 2024, 12:00
p.m. ET
- Oral Presentation: Developability assessment of novel AAV
capsids and payloads at early preclinical stage to enable
development of AAV gene therapies (#65). Matteo Placidi, Ph.D.,
Director, Analytical Sciences. Wednesday, May 8, 2024, 2:45 p.m. –
3:00 p.m. ET
- Machine learning for AAV production-fitness modeling (#974).
Daniel Cox, Ph.D., M.S., Senior Scientist, Data Science. Thursday,
May 9, 2024, 12:00 p.m. ET
- Comparing CsCl density gradient ultracentrifugation and anion
exchange chromatography for the enrichment of full adeno-associated
viral (AAV) vectors (#1037). Roberto Facendola, B.S., Scientist II,
Downstream Process Development. Thursday, May 9, 2024, 12:00 p.m.
ET
- Development of HEK293 cell line for optimal production of Novel
capsids with enhanced brain tropism (#1035). Varshini Venkatesan,
M.S., Senior Associate Engineer II, Process Development. Thursday,
May 9, 2024, 12:00 p.m. ET
About the TRACER™ Capsid Discovery
PlatformVoyager’s TRACER™ (Tropism Redirection of AAV by
Cell-type-specific Expression of RNA) capsid discovery platform is
a broadly applicable, RNA-based screening platform that enables
rapid discovery of AAV capsids with robust penetration of the
blood-brain barrier and enhanced central nervous system (CNS)
tropism in multiple species, including non-human primates (NHPs).
TRACER™ generated capsids have demonstrated superior and widespread
gene expression in the CNS compared to conventional AAV capsids as
well as cell- and tissue-specific transduction, including to areas
of the brain that have been traditionally difficult to reach, while
de-targeting the liver and dorsal root ganglia. As part of its
external partnership strategy, Voyager has established multiple
collaboration agreements providing access to its next-generation
TRACER™ capsids to potentially enable its partners’ gene therapy
programs to treat a variety of diseases.
About Voyager TherapeuticsVoyager Therapeutics,
Inc. (Nasdaq: VYGR) is a biotechnology company dedicated to
leveraging the power of human genetics to modify the course of –
and ultimately cure – neurological diseases. Our pipeline includes
programs for Alzheimer’s disease, amyotrophic lateral sclerosis
(ALS), Parkinson’s disease, and multiple other diseases of the
central nervous system. Many of our programs are derived from our
TRACER™ AAV capsid discovery platform, which we have used to
generate novel capsids and identify associated receptors to
potentially enable high brain penetration with genetic medicines
following intravenous dosing. Some of our programs are wholly
owned, and some are advancing with partners including Alexion,
AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine
Biosciences, Inc.; and Sangamo Therapeutics, Inc. For more
information, visit www.voyagertherapeutics.com.
Voyager Therapeutics® is a registered trademark, and TRACER™ is
a trademark, of Voyager Therapeutics, Inc.
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 “potential” and other similar expressions are intended to
identify forward-looking statements.
For example, all statements Voyager makes regarding Voyager’s
ability to advance its AAV-based gene therapy programs, including
expectations for Voyager’s achievement of preclinical and clinical
development milestones for its potential development candidates
such as the initiation of clinical trials; Voyager’s ability to
advance gene therapy product candidates under its partnered
programs; and the potential for Voyager’s novel TRACER capsids to
achieve desired results in humans 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 Voyager’s
technology platforms, including Voyager’s TRACER platform and its
antibody screening technology; the ability to initiate and conduct
preclinical studies in animal models; the development by third
parties of capsid identification platforms that may be competitive
to Voyager’s TRACER capsid discovery platform; Voyager’s ability to
create and protect intellectual property rights associated with the
TRACER capsid discovery platform, the capsids identified by the
platform, and development candidates for Voyager’s pipeline
programs; the initiation, timing, conduct and outcomes of Voyager’s
preclinical and clinical studies; the possibility or the timing of
Voyager’s receipt of program reimbursement, development or
commercialization milestones, option exercise, and other payments
under Voyager’s existing licensing or collaboration agreements; the
ability of Voyager to negotiate and complete licensing or
collaboration agreements with other parties on terms acceptable to
Voyager and the third parties; the ability to attract and retain
talented directors, employees, and contractors; and the sufficiency
of cash resources to fund its operations and pursue its corporate
objectives.
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. 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.
ContactsTrista Morrison, NACD.DC,
tmorrison@vygr.com Investors: Adam Bero, Ph.D., abero@kendallir.com
Media: Brooke Shenkin, brooke@scientpr.com
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