SAN FRANCISCO, Feb. 28, 2018 /PRNewswire/ -- Audentes
Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company
focused on developing and commercializing innovative gene therapy
products for patients living with serious, life-threatening rare
diseases, today announced its support for Rare Disease Day®.
The global theme of this year's Rare Disease Day is research.
Rare disease research contributes to the development of diagnostic
tools, treatments and cures, as well as improved health and social
care for patients and their families.
"Audentes is proud to join the global rare disease community to
recognize and support Rare Disease Day 2018," said Matthew R. Patterson, President and Chief
Executive Officer of Audentes. "By raising awareness, we
improve the chances that new treatments are made available for the
millions of people who live with these diseases every day."
Audentes will be supporting Rare Disease Day through employee
activities that remind us about everyday life for patients and
their families touched by a rare disease. Audentes employees
embrace the company's mission of bringing innovative gene therapy
products to patients living with serious, life-threatening rare
diseases and the day's activities will serve as a reminder that our
work is vital and urgent.
About Rare Disease Day
Rare Disease Day takes place
on the last day of February, a month known for having a 'rare'
number of days. It was first launched in Europe by EURORDIS and its Council of National
Alliances in 2008 and is now observed in more than 94
countries. Rare Disease Day is sponsored in the United States by the National Organization
for Rare Disorders (NORD)®, a leading independent, non-profit
organization committed to the identification, treatment and cure of
rare diseases.
For more information about Rare Disease Day in the United States, go to
www.rarediseaseday.us. For information about global
activities, go to www.rarediseaseday.org.
About Audentes Therapeutics, Inc.
Audentes
Therapeutics (Nasdaq: BOLD) is a biotechnology company focused on
developing and commercializing innovative gene therapy products for
patients living with serious, life-threatening rare diseases.
We are currently conducting Phase 1/2 clinical studies of our lead
product candidates AT132 for the treatment of X-Linked Myotubular
Myopathy (XLMTM), and AT342 for the treatment of Crigler-Najjar
Syndrome. We have two additional product candidates in
development, including AT982 for the treatment of Pompe disease,
and AT307 for the treatment of the CASQ2 subtype of
Catecholaminergic Polymorphic Ventricular Tachycardia
(CASQ2-CPVT). We are a focused, experienced and passionate
team committed to forging strong, global relationships with the
patient, research and medical communities.
For more information regarding Audentes, please visit
www.audentestx.com.
About AT342 for X-Linked Myotubular Myopathy
AT132 is
the Audentes product candidate being developed to treat XLMTM, a
rare monogenic disease characterized by extreme muscle weakness,
respiratory failure and early death, with an estimated 50%
mortality rate by 18 months of age. XLMTM is caused by
mutations in the MTM1 gene, which encodes the protein
myotubularin. Myotubularin plays an important role in the
development, maintenance and function of skeletal muscle
cells. AT132 is comprised of an AAV8 vector containing a
functional copy of the MTM1 gene. In January 2018, Audentes reported positive interim
data from the first dose cohort of ASPIRO, a multicenter, ascending
dose Phase 1/2 clinical study to evaluate the safety and
preliminary efficacy of AT132 in approximately 12 XLMTM patients
less than five years of age. The preclinical development of AT132
was conducted in collaboration with Genethon (www.genethon.fr).
About AT342 for Crigler-Najjar Syndrome
AT342 is an
AAV8 vector containing a functional copy of the UGT1A1 gene for the
treatment of Crigler-Najjar Syndrome, a rare monogenic disease
characterized by severely high levels of unconjugated bilirubin in
the blood and risk of irreversible neurological damage and death.
The current standard of care for Crigler-Najjar Syndrome is
daily, persistent phototherapy, usually for longer than 10 to 12
hours per day. Phototherapy wanes in effectiveness as children age,
and a liver transplant may be required for survival. Data
from LUSTRO, a prospective natural-history run-in study in
Crigler-Najjar patients, demonstrate that even with strict
adherence to a persistent daily phototherapy regimen, bilirubin may
only be reduced to levels just below those considered to be
neurotoxic. In February 2018
Audentes announced it had initiated VALENS, a multicenter,
ascending dose Phase 1/2 clinical study to evaluate the safety and
preliminary efficacy of AT342 in approximately 12 Crigler-Najjar
patients greater than or equal to one year of age.
Forward Looking Statements
This press release contains
forward-looking statements within the meaning of the "safe harbor"
provisions of the Private Securities Litigation Reform Act of 1995,
including, but not limited to: the timing of key program
milestones, including plans to dose patients in, and report
clinical data from, ASPIRO, the Phase 1/2 study of AT132 in XLMTM,
and VALENS, the Phase 1/2 study of AT342 in Crigler-Najjar
Syndrome. All statements other than statements of historical
fact are statements that could be deemed forward-looking
statements. Although the company believes that the
expectations reflected in such forward-looking statements are
reasonable, the company cannot guarantee future events, results,
actions, levels of activity, performance or achievements, and the
timing and results of biotechnology development and potential
regulatory approval is inherently uncertain. Forward-looking
statements are subject to risks and uncertainties that may cause
the company's actual activities or results to differ significantly
from those expressed in any forward-looking statement, including
risks and uncertainties related to the company's ability to advance
its product candidates, obtain regulatory approval of and
ultimately commercial its product candidates, the timing and
results of preclinical and clinical trials, the company's ability
to fund development activities and achieve development goals, the
company's ability to protect intellectual property and
other risks and uncertainties described under the heading
"Risk Factors" in documents the company files from time to time
with the Securities and Exchange Commission. These
forward-looking statements speak only as of the date of this press
release, and the company undertakes no obligation to revise or
update any forward-looking statements to reflect events or
circumstances after the date hereof.
Audentes Contacts:
Investor Contact:
Andrew Chang
415.818.1033
achang@audentestx.com
Media Contact:
Paul Laland
415.519.6610
plaland@audentestx.com
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SOURCE Audentes Therapeutics, Inc.