Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine
company, and Voyager Therapeutics, Inc. (Nasdaq: VYGR), today
announced the parties have entered into a definitive license
agreement for a potential treatment of prion disease. Using its
proprietary epigenetic regulation platform, Sangamo has developed
zinc finger transcriptional regulators (ZF-TRs) which it believes
can specifically and potently block expression of the prion
protein, the pathogenic driver of prion disease. Sangamo’s ZF-TRs
have been shown in animal models to significantly reduce expression
of the prion protein in the brain, extend life span and limit
formation of toxic prion aggregates. Under the agreement, Voyager
has provided Sangamo with access to a capsid derived from Voyager’s
proprietary TRACER™ platform. This capsid has been shown to
effectively cross the blood-brain barrier when delivered
intravenously in animal models.
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“Prion is a devastating, rapidly progressing and deadly disease,
for which no treatment options currently exist,” said Sandy Macrae,
Chief Executive Officer at Sangamo. “While evaluating delivery
mechanisms for our zinc finger transcriptional regulators, we were
pleased to identify Voyager’s capsid as being well suited to
potentially achieve the specific central nervous system coverage
required for this indication today. Our agreement with Voyager
highlights the importance of collaboration to address difficult
indications with significant unmet medical need. I’m excited to
combine Sangamo’s cutting-edge epigenetic regulation capabilities
with the delivery abilities of Voyager’s capsid to potentially
create the first ever meaningful therapy for prion disease.”
Under the terms of the agreement, Sangamo has received a
non-exclusive license to combine a Voyager TRACER capsid with
Sangamo’s ZF-TRs designed to treat prion disease. Sangamo is solely
responsible for the research, development, manufacture and
commercialization of any product candidates using the Voyager
capsid. Voyager is eligible to earn certain license fees, royalties
on potential commercial sales of any products using Voyager’s
capsid, and, in the event the prion program is out licensed by
Sangamo, a portion of all licensing revenues received with respect
to this program. Sangamo expects to submit a potential IND for a
product candidate treating prion disease in 2025.
“We believe the novel, brain-penetrant capsids emerging from our
TRACER capsid discovery platform have the potential to enable gene
therapies for a wide variety of diseases of the central nervous
system – far more than we could prosecute internally,” said Alfred
W. Sandrock, Jr., M.D., Ph.D., Chief Executive Officer of Voyager.
“We continue to explore a variety of capsid license structures,
such as in this transaction with Sangamo, to leverage our
technology for more programs, and ultimately, more patients.”
About Prion Disease
Prion disease is a rapidly progressing, fatal neurodegenerative
disease caused by the misfolding of the cellular prion protein,
PrPC, encoded by the PRNP gene. Misfolded prion protein is acutely
toxic to neurons, which can lead to the rapid development of
dementia, difficulty walking and changes in gait, hallucinations,
muscle stiffness, confusion, fatigue, difficulty speaking, and
ultimately death. Most cases are sporadic or caused by inherited
dominant mutations in PRNP, with an estimated 500 patients
diagnosed per year in the US. The most common form of prion disease
that affects humans is Creutzfeldt-Jakob disease. There are
currently no approved or clinical-stage disease-modifying therapies
for the prevention or treatment of prion disease.
About Sangamo Zinc Finger Transcriptional Regulators
Sangamo’s zinc finger transcriptional regulators, or ZF-TRs,
recognize and bind to a specific DNA sequence within or near a
particular gene, allowing expression of that target gene to be
potentially regulated. ZF activators, or ZF-As, are created by
attaching a zinc finger array to an activation domain with the aim
of increasing the expression of a target gene relative to an
untreated cell. ZF repressors, or ZF-Rs, are created by attaching a
zinc finger array to a repression domain in order to down regulate
or completely turn off a gene. ZF-Rs can also be designed to
selectively repress expression of a mutant allele while allowing
for the expression of the healthy allele. Sangamo is currently
evaluating ZF-TRs in a variety of pre-clinical programs, including
Nav1.7 for the potential treatment of chronic neuropathic pain and
for prion disease.
About Sangamo Therapeutics
Sangamo Therapeutics is a clinical-stage biopharmaceutical
company with a robust genomic medicines pipeline. Using
ground-breaking science, including our proprietary zinc finger
genome engineering technology and manufacturing expertise, Sangamo
aims to create new genomic medicines for patients suffering from
diseases for which existing treatment options are inadequate or
currently don’t exist. To learn more, visit www.sangamo.com and
connect with us on LinkedIn and Twitter.
About the TRACER™ AAV Capsid Discovery Platform
Voyager’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.
Separate results have demonstrated the enhanced ability of certain
capsids to target cardiac muscle and to de-target the dorsal root
ganglia. Voyager is expanding its library of AAV capsids optimized
to deliver diverse therapeutic payloads to address a broad range of
CNS and other diseases. 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 Therapeutics
Voyager Therapeutics (Nasdaq: VYGR) is a biotechnology company
dedicated to breaking through barriers in gene therapy and
neurology. The potential of both disciplines has been constrained
by delivery challenges; Voyager is leveraging cutting-edge
expertise in capsid discovery and deep neuropharmacology
capabilities to address these constraints. Voyager’s TRACER™ AAV
capsid discovery platform has generated novel capsids with high
target delivery and blood-brain barrier penetration at low doses,
potentially addressing the narrow therapeutic window associated
with conventional gene therapy delivery vectors. This platform is
fueling alliances with Pfizer Inc., Novartis and Neurocrine
Biosciences as well as multiple programs in Voyager’s own pipeline.
Voyager’s pipeline includes wholly owned and collaborative
preclinical programs in Alzheimer’s disease, amyotrophic lateral
sclerosis (ALS), Parkinson’s disease, and Friedreich’s Ataxia, with
a focus on validated targets and biomarkers to enable a path to
rapid potential proof-of-biology. For more information, visit
www.voyagertherapeutics.com.
Voyager Therapeutics® is a registered trademark, and TRACER™ is
a trademark, of Voyager Therapeutics, Inc.
Sangamo Forward Looking Statements
This press release contains forward-looking statements based on
Sangamo's current expectations. These forward-looking statements
include, without limitation, statements relating to zinc finger
transcriptional regulators’ potential to specifically and potently
block expression of the prion protein, the development of a
potential treatment for prion disease through epigenetic regulation
of the prion protein and the occurrence and timing of a potential
IND submission in 2025. These statements are not guarantees of
future performance and are subject to certain risks and
uncertainties that are difficult to predict. Factors that could
cause actual results to differ include, but are not limited to, the
research and development process, including the results of clinical
trials; the regulatory approval process for product candidates; and
the potential for technological developments that obviate
technologies used by Sangamo and Voyager; the COVID-19 pandemic;
the potential for Voyager to breach or terminate its agreement with
Sangamo; and the potential for Sangamo to fail to realize its
expected benefits of the Voyager agreement. Actual results may
differ from those projected in forward-looking statements due to
risks and uncertainties that exist in Sangamo's operations and
business. These risks and uncertainties are described more fully in
our Securities and Exchange Commission filings and reports,
including in our Annual Report on Form 10-K for the year ended
December 31, 2022, and our Quarterly Report on Form 10-Q for the
quarter ended March 31, 2023. Forward-looking statements contained
in this announcement are made as of this date, and Sangamo
undertakes no duty to update such information except as required
under applicable law.
Voyager 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 laws.
The use of words such as "may," "might," "will," "would," "should,"
"expect," "plan," "anticipate," "believe," "estimate,"
"undoubtedly," “target,” "project," "intend," "future,"
"potential," or "continue," and other similar expressions are
intended to identify forward-looking statements.
For example, all statements Voyager makes regarding Voyager’s
ability to continue to develop its TRACER capsid discovery platform
and identify and develop proprietary, brain-penetrant capsids from
its TRACER capsid discovery platform with the potential to enable
gene therapies for a wide variety of diseases of the central
nervous system; Voyager’s ability to utilize its proprietary
capsids in its own product development programs; Voyager’s ability
to attract parties to license its novel proprietary capsids or to
participate with Voyager in research and development collaborations
utilizing its novel proprietary capsids; Voyager’s ability to
advance its AAV-based gene therapy programs; Voyager’s entitlement
to receive license fees and royalties from Sangamo under the
license agreement, Voyager’s ability to add new programs to its
pipeline and enter into new partnerships or collaborations, and the
sufficiency of Voyager’s cash resources to enable it to continue to
identify and develop proprietary capsids from its TRACER capsid
discovery platform 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. Such risks and uncertainties include, among others, the
continued development of Voyager’s capsid and gene therapy
platforms; Voyager’s ability to attract and retain talented
contractors and employees to continue the development of the TRACER
capsid discovery platform and the identification of proprietary
capsids; Voyager’s ability to create and protect intellectual
property rights associated with the TRACER capsid discovery
platform and the capsids identified by the platform; the
possibility or the timing of any development or commercialization
under the Sangamo license agreement and other license and
collaboration agreements; the ability of Voyager to negotiate and
complete licensing or collaboration agreements on terms acceptable
to Voyager and third parties; Voyager’s ability to perform its
obligations under its license option agreement with Sangamo’s
ability to perform its obligations under such agreement; and the
sufficiency of Voyager’s cash resources to enable it to fund its
operations and development plans. 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. 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.
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Sangamo Contact Investor Relations & Media Inquiries
Louise Wilkie ir@sangamo.com media@sangamo.com
Voyager Contact Voyager Investors: Andrew Funderburk,
afunderburk@kendallir.com Voyager Media: Peg Rusconi,
prusconi@vergescientific.com
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