New Preclinical Data from Dyne Therapeutics’ Myotonic Dystrophy Type 1 Program to be Featured in Presentations During Upcom...
April 27 2021 - 4:35PM
Dyne Therapeutics, Inc. (Nasdaq: DYN), a muscle disease
company focused on advancing innovative life-transforming
therapeutics for people living with genetically driven diseases,
today announced that new preclinical data from its myotonic
dystrophy type 1 (DM1) program will be featured in presentations
during the American Society of Gene & Cell Therapy (ASGCT) 24th
Annual Meeting, to be held virtually May 11-14, 2021. ASGCT
abstracts are available on the meeting website, and Dyne’s
presentations are noted below.
Presentation: Splice Correction
and Reduction of Toxic DMPK RNA In Vitro and In Vivo Utilizing
Novel Antibody Targeted Antisense Oligonucleotides
Scientific Symposium: Hot Topics and Remaining
Challenges in RNAi and Oligonucleotide Therapy for
2021Date/Time: Friday, May 14, 2021 at 10:26 a.m.
ETPresenter: Romesh Subramanian, Ph.D., Chief
Scientific Officer
Oral Presentation: The
FORCETM Platform Achieves Robust Knock Down of Toxic Human
Nuclear DMPK RNA and Foci Reduction in DM1 Cells and in
Newly Developed hTfR1/DMSXL Mouse Model (Abstract
#247)Session: Oligonucleotide
TherapeuticsDate/Time: Friday, May 14, 2021 at
1:15 p.m. ETPresenter: Stefano Zanotti, Ph.D.,
Director, Mechanistic Biology
The presentations will include new data, expanding
upon initial findings reported in January 2021 utilizing Dyne’s
innovative hTfR1/DMSXL mouse model expressing human TfR1 and
carrying a human DMPK gene that represents a severe DM1
phenotype with more than 1,000 CTG repeats, as well as results
from in vitro studies.
Dyne’s FORCE™ platform leverages the importance of
transferrin 1 receptor, TfR1, in muscle biology as the
foundation for its novel approach. TfR1, which is highly expressed
on the surface of muscle cells, is required for iron transport into
muscle cells. Dyne links therapeutic payloads to its TfR1-binding
fragment antibody (Fab) to develop targeted therapeutics for muscle
diseases. Dyne’s lead DM1 candidate consists of a Fab conjugated to
an antisense oligonucleotide (ASO) to enable targeted delivery to
muscle tissue to reduce accumulation of toxic DMPK RNA in
the nucleus, release splicing proteins, allow normal mRNA
processing and translation of normal proteins, and potentially stop
or reverse disease progression.
DM1 Program Webcast
Following the presentations on May 14, 2021, Dyne
plans to host a live webcast event at 4:00 p.m. ET to review the
company’s DM1 program and preclinical data, and to provide an
overview of the disease and treatment challenges. Joining
management on the webcast will be Charles Thornton, M.D., the
Saunders Distinguished Professor of Neuromuscular Research at the
University of Rochester. Dr. Thornton directs the Muscular
Dystrophy Cooperative Research Center in Rochester and the Myotonic
Dystrophy Clinical Research Network, a multicenter consortium for
clinical research and therapeutic trials. He has been engaged in
bench and clinical research on myotonic dystrophy for 30 years.
The live and archived webcast can be accessed in
the Investors & Media section of Dyne’s website
at: https://investors.dyne-tx.com/news-and-events/events-and-presentations.
The archived webcast will be made available shortly after the event
and accessible for 90 days.
About Dyne Therapeutics
Dyne Therapeutics is building a leading muscle
disease company dedicated to 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 seen with
other approaches. Dyne’s broad portfolio of therapeutic
candidates for serious muscle diseases includes programs for
myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy
(DMD) and 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, 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 conduct of
research activities and the initiation and completion of
preclinical studies and clinical trials; uncertainties as to the
availability and timing of results from preclinical studies; the
timing of and Dyne’s ability to submit and obtain regulatory
approval for investigational new drug applications; 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; the impact of the COVID-19 pandemic on Dyne’s
business and operations; as well as the risks and uncertainties
identified in Dyne’s filings with the Securities and Exchange
Commission (SEC), including the Company’s Annual Report on Form
10-K for the year ended December 31, 2020 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.
Contact:
Dyne TherapeuticsAmy
Reillyareilly@dyne-tx.com857-341-1203
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