Company remains on track to have three muscular
dystrophy programs in clinic by end of year
SAN DIEGO, Sept. 7, 2022 /PRNewswire/ -- Avidity
Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company
committed to delivering a new class of RNA therapeutics called
Antibody Oligonucleotide Conjugates (AOCs™), honors National
Muscular Dystrophy Awareness Month, an annual observance that
raises awareness for families across the nation who are living with
neuromuscular diseases. This month Avidity will also raise
awareness and support patient-focused activities for World Duchenne
Awareness Day on September 7 and
International Myotonic Dystrophy Awareness Day on September 15. Avidity is on track to have three
rare muscular dystrophy programs in the clinic by the end of this
year for myotonic dystrophy type 1 (DM1), Facioscapulohumeral
muscular dystrophy (FSHD) and Duchenne muscular dystrophy
(DMD).
"We look forward to engaging with patient communities and their
families this month to better understand their unique journeys and
experiences. We are excited to welcome many of our partners here to
San Diego where we will be
participating at the MDF Annual Conference, FSHD Society's Walk and
Roll to Cure FSHD, and the Global Genes RARE Patient Advocacy
Summit," said Sarah Boyce, president
and chief executive officer. "At Avidity, we are committed to
listening, learning and partnering with the patient and advocacy
community as part of our mission to improve the lives of people
affected by serious rare diseases. We are on track to have three
rare muscular dystrophy programs in the clinic by the end of this
year."
In support of Muscular Dystrophy Awareness Month, Avidity will
be engaging in various activities with patient and advocacy
communities including:
- Welcoming patients and families to tour Avidity's office during
the 2022 Myotonic Dystrophy Foundation (MDF) Annual Conference, an
event to unite community, care and a cure for myotonic
dystrophy
- Supporting Jett Foundation's Stronger than Duchenne World
Duchenne Awareness event
- Recognizing International Myotonic Dystrophy Awareness
Day as a proud member of the Global Alliance for Myotonic
Dystrophy Awareness
- Joining in the FSHD Society's 2022 Walk & Roll to Cure
FSHD in San Diego, the only
international event focused solely on funding progress for
FSHD
- Participating in the 2022 Global Genes RARE Patient Advocacy
Summit, one of the world's largest gatherings of rare disease
patients, caregivers, advocates and healthcare professionals
About Myotonic Dystrophy Type 1
Myotonic dystrophy type 1 (DM1) is an underrecognized, progressive
and often fatal disease caused by a triplet-repeat in the DMPK
gene, resulting in a toxic gain of function mRNA. The disease is
highly variable with respect to severity, presentation and age of
onset, however all forms of DM1 are associated with high levels of
disease burden and may cause premature mortality. DM1 primarily
affects skeletal and cardiac muscle, however patients can suffer
from a constellation of manifestations including myotonia and
muscle weakness, respiratory problems, fatigue, hypersomnia,
cardiac abnormalities, severe gastrointestinal complications, and
cognitive and behavioral impairment. Currently, there are no
treatments for people living with DM1.
About Facioscapulohumeral muscular dystrophy
(FSHD)
Facioscapulohumeral muscular dystrophy (FSHD) is
characterized by progressive and often asymmetric skeletal muscle
loss that initially causes weakness in muscles in the face,
shoulders, arms and trunk and progresses to weakness in muscles in
lower body. FSHD is an autosomal dominant genetic disease, meaning
a single copy of the disease-associated gene, DUX4 (double homeobox
4), is enough to cause the disease. The abnormal expression of DUX4
leads to a series of downstream events that result in skeletal
muscle wasting and compromised muscle function, including an
inability to lift arms for more than a few seconds, loss of ability
to show facial expressions and serious speech impediments. These
symptoms cause many people affected by FSHD to become dependent on
the use of a wheelchair for mobility. Currently there are no
approved treatments for people living with FSHD.
About Duchenne muscular dystrophy (DMD)
Duchenne
muscular dystrophy (DMD) causes a lack of functional dystrophin
that leads to stress and tears of muscle cell membranes, resulting
in muscle cell death and the progressive loss of muscle function.
The dystrophin protein maintains the integrity of muscle fibers and
acts as a shock absorber through its role as the foundation of a
group of proteins that connects the inner and outer elements of
muscle cells. People living with DMD suffer from progressive muscle
weakness that typically starts in boys at a very young age. Those
living with the condition often require special aid and assistance
throughout their lives and have significantly shortened life
expectancy. While there are treatments approved to treat people
with DMD, it remains a very high unmet need.
About Avidity
Avidity Biosciences, Inc.'s mission is to profoundly improve
people's lives by delivering a new class of RNA therapeutics -
Antibody Oligonucleotide Conjugates (AOCs™). Avidity's proprietary
AOCs are designed to combine the specificity of monoclonal
antibodies with the precision of oligonucleotide therapies to
target the root cause of diseases previously untreatable with RNA
therapeutics. Avidity is on track to have three programs in
clinical development by the end of 2022. The company's lead product
candidate, AOC 1001, is designed to treat patients with myotonic
dystrophy type 1 (DM1). AOC 1001 is currently in Phase 1/2
development with the ongoing MARINA™ trial and MARINA-OLE™ in
adults with DM1. The next programs in the company's advancing and
expanding pipeline are AOC 1044, the lead of three programs for the
treatment of DMD, and AOC 1020, designed to treat people living
with FSHD. Avidity anticipates both programs will enter the clinic
by the end of 2022. Avidity is also broadening the reach of AOCs
beyond muscle tissues through both internal discovery efforts and
key partnerships as the company continues to deliver on the RNA
revolution. Avidity is headquartered in San Diego,
CA. For more information about our science, pipeline and
people, please visit www.aviditybiosciences.com and
engage with us on LinkedIn and Twitter.
Forward-Looking Statements
Avidity cautions readers
that statements contained in this press release regarding matters
that are not historical facts are forward-looking statements. These
statements are based on the company's current beliefs and
expectations. Such forward-looking statements include, but are not
limited to, statements regarding: the progression of clinical
programs for AOC 1001, AOC 1044 and AOC 1020 and timing thereof;
and the broad potential of AOCs to treat serious diseases of
skeletal muscle and other tissues and cell types. The inclusion of
forward-looking statements should not be regarded as a
representation by Avidity that any of these plans will be achieved.
Actual results may differ from those set forth in this press
release due to the risks and uncertainties inherent in the
business, including, without limitation: Avidity is early in its
development efforts; Avidity's approach to the discovery and
development of product candidates based on its AOC platform is
unproven, and the company does not know whether it will be able to
develop any products of commercial value; potential delays in the
commencement, enrollment and completion of preclinical studies or
clinical trials; the success of its preclinical studies and
clinical trials for the company's product candidates; the results
of preclinical studies and early clinical trials are not
necessarily predictive of future results; Avidity's dependence on
third parties in connection with preclinical and clinical testing
and product manufacturing; unexpected adverse side effects or
inadequate efficacy of its product candidates that may limit their
development, regulatory approval and/or commercialization, or may
result in recalls or product liability claims; regulatory
developments in the United States
and foreign countries, including acceptance of INDs and similar
foreign regulatory filings and the proposed design of future
clinical trials; Avidity could use its available capital resources
sooner than it currently expects; disruption to its operations from
the COVID-19 pandemic or the war in Ukraine; and other risks described in prior
press releases and in filings with the Securities and Exchange
Commission (SEC). Avidity cautions readers not to place undue
reliance on these forward-looking statements, which speak only as
of the date hereof, and the company undertakes no obligation to
update such statements to reflect events that occur or
circumstances that exist after the date hereof. All forward-looking
statements are qualified in their entirety by this cautionary
statement, which is made under the safe harbor provisions of the
Private Securities Litigation Reform Act of 1995.
Investor Contact:
Kathleen
Gallagher
(858) 401-7900 x550
investors@aviditybio.com
Media Contact:
Navjot
Rai
(858) 401-7900 x550
media@aviditybio.com
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SOURCE Avidity Biosciences, Inc.