Webcast on Wednesday, Dec. 7,
2022, at 8 am ET
AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene
therapy company working to free people from a lifetime of genetic
disease, today announced that it will host a virtual Gaucher
Disease Program Update for analysts and investors on Wednesday,
Dec. 7, 2022, starting at 8 am ET.
AVROBIO will share updates on its clinical program using
hematopoietic stem cell (HSC) gene therapy to target the most
common lysosomal disorder, Gaucher disease. The comprehensive data
update will include new pharmacodynamic and clinical efficacy data
out to two years post gene therapy. Additionally, the company will
provide an update on a potential Phase 2/3 Gaucher disease type 3
clinical trial and related regulatory interactions, as well as the
overall Gaucher disease clinical development strategy.
In addition to members of AVROBIO management, guest speakers
will include leading experts in lysosomal disorders and pediatric
stem cell transplants.
A live webcast of the presentation and accompanying slides will
be available under “Events and Presentations” on the Investors
section of the company’s website at www.avrobio.com. An archived
webcast recording of the event will be available on the website for
approximately 30 days.
If you are a member of the investment community and would like
to attend, please RSVP to aaron.sobell@westwicke.com.
About Gaucher disease Gaucher disease is a rare,
inherited lysosomal disorder characterized by the toxic
accumulation of glucosylceramide (GlcCer) and glucosylsphingosine
(GlcSph) in macrophages. Macrophages enlarged with these fatty
substances are called Gaucher cells which amass primarily in the
spleen, liver and bone marrow. This results in a variety of
potential symptoms, including grossly enlarged liver and spleen,
bone issues, fatigue, low hemoglobin levels and platelet counts and
an adjusted lifetime relative risk of developing Parkinson's
disease that may be more than 20 times greater than the general
population. Even on enzyme replacement therapy (ERT) – the current
standard of care – people with Gaucher disease typically have a
shortened life expectancy and may experience debilitating symptoms
that significantly reduce their quality of life.
About AVROBIO Our vision is to bring personalized gene
therapy to the world. We target the root cause of genetic disease
by introducing a functional copy of the affected gene into
patients’ own hematopoietic stem cells (HSCs), with the goal to
durably express the therapeutic protein throughout the body,
including the central nervous system. Our first-in-class pipeline
includes clinical programs for cystinosis and Gaucher disease type
1, as well as preclinical programs for Gaucher disease type 3,
Hunter syndrome and Pompe disease. Our proprietary plato® gene
therapy platform is designed to be scaled to support late-stage
clinical development and commercialization globally. We are
headquartered in Cambridge, Mass. For additional information, visit
avrobio.com, and follow us on Twitter and LinkedIn.
Forward-Looking Statements This press release contains
forward-looking statements, including statements made pursuant to
the safe harbor provisions of the Private Securities Litigation
Reform Act of 1995. These statements may be identified by words and
phrases such as “aims,” “anticipates,” “believes,” “could,”
“designed to,” “estimates,” “expects,” “forecasts,” “goal,”
“intends,” “may,” “plans,” “possible,” “potential,” “seeks,”
“will,” and variations of these words and phrases or similar
expressions that are intended to identify forward-looking
statements. These forward-looking statements include, without
limitation, statements regarding our business strategy for and the
potential therapeutic benefits of our preclinical and clinical
product candidates, including AVR-RD-02 for the treatment of
Gaucher disease, the potential benefits and incentives provided by
FDA’s rare pediatric disease designation for AVR-RD-02, the
potential benefits provided by FDA’s priority review voucher, the
design, planning, commencement, enrollment and timing of current or
anticipated clinical trials, preclinical or clinical trial results,
product approvals and regulatory pathways, our plans and
expectations with respect to interactions with regulatory agencies
including interactions relating to a potential Phase 2/3 Gaucher
disease type 3 clinical trial, anticipated benefits of our gene
therapy platform including potential impact on our
commercialization activities, timing and likelihood of success, the
expected benefits and results of our implementation of the plato®
platform in our clinical trials and gene therapy programs, and the
expected safety profile of our preclinical and investigational gene
therapies. Any such statements in this press release that are not
statements of historical fact may be deemed to be forward-looking
statements. Results in preclinical or early-stage clinical trials
may not be indicative of results from later stage or larger scale
clinical trials and do not ensure regulatory approval. You should
not place undue reliance on these statements, or the scientific
data presented.
Any forward-looking statements in this press release are based
on AVROBIO’s current expectations, estimates and projections about
our industry as well as management’s current beliefs and
expectations of future events only as of today and are subject to a
number of risks and uncertainties that could cause actual results
to differ materially and adversely from those set forth in or
implied by such forward-looking statements. These risks and
uncertainties include, but are not limited to, the risk that any
one or more of AVROBIO’s product candidates will not be
successfully developed or commercialized, the risk of cessation or
delay of any ongoing or planned clinical trials of AVROBIO or our
collaborators, the risk that AVROBIO may not successfully recruit
or enroll a sufficient number of patients for our clinical trials,
the risk that AVROBIO may not realize the intended benefits of our
gene therapy platform, including the features of our plato®
platform, the risk that our product candidates or procedures in
connection with the administration thereof will not have the safety
or efficacy profile that we anticipate, the risk that prior
results, such as signals of safety, activity or durability of
effect, observed from preclinical or clinical trials, will not be
replicated or will not continue in ongoing or future studies or
trials involving AVROBIO’s product candidates, the risk that we
will be unable to obtain and maintain regulatory approval for our
product candidates, the risk that the size and growth potential of
the market for our product candidates will not materialize as
expected, risks associated with our dependence on third-party
suppliers and manufacturers, risks regarding the accuracy of our
estimates of expenses and future revenue, risks relating to our
capital requirements and needs for additional financing, risks
relating to clinical trial and business interruptions resulting
from the COVID-19 outbreak or similar public health crises,
including that such interruptions may materially delay our
enrollment and development timelines and/or increase our
development costs or that data collection efforts may be impaired
or otherwise impacted by such crises, and risks relating to our
ability to obtain and maintain intellectual property protection for
our product candidates. For a discussion of these and other risks
and uncertainties, and other important factors, any of which could
cause AVROBIO’s actual results to differ materially and adversely
from those contained in the forward-looking statements, see the
section entitled “Risk Factors” in AVROBIO’s most recent Quarterly
Report, as well as discussions of potential risks, uncertainties
and other important factors in AVROBIO’s subsequent filings with
the Securities and Exchange Commission. AVROBIO explicitly
disclaims any obligation to update any forward-looking statements
except to the extent required by law.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20221117005291/en/
Investors: Christopher F. Brinzey Westwicke, an ICR Company
339-970-2843 chris.brinzey@westwicke.com
Media: Kit Rodophele Ten Bridge Communications 617-999-9620
krodophele@tenbridgecommunications.com
AVROBIO (NASDAQ:AVRO)
Historical Stock Chart
From Feb 2023 to Mar 2023
AVROBIO (NASDAQ:AVRO)
Historical Stock Chart
From Mar 2022 to Mar 2023