Dyne Therapeutics’ Preclinical Data Demonstrating FORCE™ Platform Delivery to CNS Featured in Oral Presentation at ASGCT Annual Meeting
17 Maio 2023 - 8:30AM
Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle
disease company focused on advancing innovative life-transforming
therapeutics for people living with genetically driven diseases, is
delivering an oral presentation today at the American Society of
Gene & Cell Therapy (ASGCT) 26th Annual Meeting in Los Angeles
highlighting new preclinical data demonstrating the FORCE™ platform
achieved delivery to the central nervous system (CNS) in non-human
primates and robust pharmacological effects in the brain in a model
of myotonic dystrophy type 1 (DM1).
Oral Presentation: FORCE™ Platform Delivers
Oligonucleotides to the Brain in a DM1 Mouse Model and in NHPs
(abstract #82)Session: Nucleic Acid
TherapeuticsDate/Time: Wednesday, May 17, 2023, at
4:00 p.m. PTPresenter: Stefano Zanotti, Ph.D.,
Executive Director, Head of Neuromuscular Research, Dyne
The presentation will be available in the Scientific
Publications & Presentations section of Dyne’s website
following the session.
The FORCE platform was designed to overcome the limitations of
delivering oligonucleotide therapeutics to muscle tissue by
leveraging transferrin receptor 1 (TfR1). TfR1-mediated delivery
also has been shown by the field to facilitate uptake of
therapeutics by the CNS. Many people living with rare muscle
diseases experience CNS symptoms that contribute to the burden of
disease, including cognitive deficits and dysregulated sleep, which
affect individuals with DM1.
Data being presented at ASGCT show that intravenous (IV)
administration of FORCE conjugate, a TfR1-binding Fab antibody
conjugated to an antisense oligonucleotide (ASO), achieved delivery
to the CNS via TfR1 in both non-human primates (NHPs) and the
innovative hTfR1/DMSXL mouse model. The hTfR1/DMSXL model,
developed by Dyne, expresses the human TfR1 and carries a human
DMPK gene with more than 1,000 CTG repeats that represents a severe
DM1 phenotype. In these studies, FORCE conjugate was well
tolerated.
Highlights from the ASGCT data include:
- In NHPs, FORCE conjugate achieved
superior delivery compared to unconjugated ASO when both were
administered via IV. In addition, IV administration of FORCE showed
broader distribution throughout the brain compared to intrathecal
(IT) administration of unconjugated ASO.
- FORCE conjugate also delivered
to the brain of hTfR1/DMSXL mice and demonstrated robust knockdown
of toxic human nuclear DMPK RNA and foci reduction.
“We have previously demonstrated that the FORCE platform enables
delivery of oligonucleotides to skeletal, smooth and cardiac muscle
in multiple, well-validated preclinical models, and we are excited
to now highlight TfR1-mediated delivery to the CNS in these data at
ASGCT. The CNS manifestations of neuromuscular disorders contribute
significantly to the disease burden, and we look forward to further
evaluating the potential of the FORCE platform in this area and
advancing our commitment to delivering life-transforming therapies
to patients,” said Oxana Beskrovnaya, Ph.D., chief scientific
officer of Dyne.
About Dyne Therapeutics
Dyne Therapeutics is a clinical-stage muscle disease company
focused on advancing innovative life-transforming therapeutics for
people living with genetically driven diseases. With its
proprietary FORCE™ platform, Dyne is developing modern
oligonucleotide therapeutics that are designed to overcome
limitations in delivery to muscle tissue. Dyne has a broad pipeline
for serious muscle diseases, including clinical programs for
myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy
(DMD), and a preclinical program for facioscapulohumeral muscular
dystrophy (FSHD). For more information, please visit
https://www.dyne-tx.com/, and follow us on Twitter, LinkedIn and
Facebook.
Forward-Looking Statements
This press release contains forward-looking statements that
involve substantial risks and uncertainties. All statements, other
than statements of historical facts, contained in this press
release, including statements regarding Dyne’s strategy, future
operations, prospects and plans, objectives of management, and the
potential of the FORCE platform, constitute forward-looking
statements within the meaning of The Private Securities Litigation
Reform Act of 1995. The words “anticipate,” “believe,” “continue,”
“could,” “estimate,” “expect,” “intend,” “may,” “might,”
“objective,” “ongoing,” “plan,” “predict,” “project,” “potential,”
“should,” or “would,” or the negative of these terms, or other
comparable terminology are intended to identify forward-looking
statements, although not all forward-looking statements contain
these identifying words. Dyne may not actually achieve the plans,
intentions or expectations disclosed in these forward-looking
statements, and you should not place undue reliance on these
forward-looking statements. Actual results or events could differ
materially from the plans, intentions and expectations disclosed in
these forward-looking statements as a result of various important
factors, including: uncertainties inherent in the identification
and development of product candidates, including the initiation and
completion of preclinical studies and clinical trials;
uncertainties as to the availability and timing of results from
preclinical studies and clinical trials; the timing of and Dyne’s
ability to initiate and enroll patients in clinical trials; whether
results from preclinical studies will be predictive of the results
of later preclinical studies and clinical trials; whether Dyne’s
cash resources will be sufficient to fund the Company’s foreseeable
and unforeseeable operating expenses and capital expenditure
requirements; as well as the risks and uncertainties identified in
Dyne’s filings with the Securities and Exchange Commission (SEC),
including the Company’s most recent Form 10-Q and in subsequent
filings Dyne may make with the SEC. In addition, the
forward-looking statements included in this press release represent
Dyne’s views as of the date of this press release. Dyne anticipates
that subsequent events and developments will cause its views to
change. However, while Dyne may elect to update these
forward-looking statements at some point in the future, it
specifically disclaims any obligation to do so. These
forward-looking statements should not be relied upon as
representing Dyne’s views as of any date subsequent to the date of
this press release.
Contacts:
InvestorsAmy
Reillyareilly@dyne-tx.com857-341-1203
MediaStacy Nartkersnartker@dyne-tx.com
781-317-1938
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