Analyst and investor conference call scheduled
for Wednesday, Feb. 9, 2022, at 8:00 a.m. ET
Five platform presentations and three posters
on the company’s clinical and preclinical programs for lysosomal
disorders are scheduled throughout the symposium
AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene
therapy company with a mission to free people from a lifetime of
genetic disease, today announced that updated clinical data from
its ongoing, collaborator-sponsoredi Phase 1/2 clinical trial in
cystinosis will be presented on Feb. 9, 2022, at the 18th Annual
WORLDSymposium™ in San Diego, Calif. Additionally, preclinical data
from its Pompe disease and Hunter syndrome programs, as well as
data on its ongoing work to monitor the nature and dynamics of gene
therapy cell products and their impact on the composition of blood
cell populations in lysosomal disorders, will be presented during
the four-day conference starting Feb. 7.
The company’s planned activities are listed below and the full
preliminary program is available online at the WORLDSymposium™
website.
ANALYST AND INVESTOR EVENT: Members of AVROBIO management
will host an Analyst and Investor conference call and webcast at
8:00 a.m. ET on Wednesday, Feb. 9, to discuss the updated data. The
event can be accessed under “Events and Presentations” in the
Investors section of the company’s website or by dialing 1 (866)
939-3921 from locations in the U.S. The conference ID number is
50279190.
An archived recording of the event will be available on the
website for approximately 30 days. To RSVP or for additional
information on the event, please email us here.
PLATFORM PRESENTATIONS: Enhanced transduction and
immunophenotyping demonstrates preclinical safety and efficacy of
hematopoietic stem cell gene therapy for mucopolysaccharidosis type
II using an IDS.ApoEII brain targeted therapy
Tuesday, Feb. 8, 2022, 9:30 a.m. PST (Session 9-10)
- Stuart Ellison, Ph.D., University of Manchester, UK, will
present new preclinical data for AVR-RD-05, a gene therapy program
for Hunter syndrome (MPS II).
Hematopoietic stem cell gene therapy for cystinosis: updated
results from a Phase I/II clinical trial
Wednesday, Feb. 9, 2022, 1:45 p.m. PST (Session 1-2)
- Stephanie Cherqui, Ph.D., principal investigator of the
collaborator-sponsored Phase 1/2 trial of AVR-RD-04, an
investigational gene therapy for cystinosis, and associate
professor at University of California, San Diego, will present new
clinical data from the patients dosed in the ongoing trial.
Long-term hematopoietic stem cell lentiviral gene therapy
rescues neuromuscular manifestations in preclinical study of Pompe
disease mice
Thursday, Feb. 10, 2022, 9:45 a.m. PST (Session 9-10)
- Niek van Til, Ph.D., will present new preclinical data for
AVR-RD-03, a gene therapy program for Pompe disease.
High-resolution cellular and molecular follow up of lysosomal
disorder patients treated with hematopoietic stem cell lentiviral
gene therapy
Thursday, Feb. 10, 2022, 2:30 p.m. PST (Session 2-3)
- Mariana Loperfido, Ph.D., AVROBIO, will present data from
exploratory studies based on high-resolution cellular and molecular
analysis of patients’ bone marrow and peripheral blood.
High throughput monitoring of safety, potency and stability
of gene therapy cell products in lysosomal disease patients
Thursday, Feb. 10, 2022, 2:45 p.m. PST (Session 2-3)
- Luca Biasco, Ph.D., AVROBIO, will present data on a novel
analytical platform to monitor the nature and dynamics of gene
therapy cell products.
POSTER PRESENTATIONS: Ex-vivo autologous stem cell gene
therapy for MPSII (Hunter syndrome) (#25) Tuesday, Feb. 8,
2022, between 3:00-5:00 p.m. PST
Systematic literature review of the clinical effectiveness,
safety, quality of life, epidemiology and economic burden
associated with cystinosis (#14) Thursday, Feb. 10, 2022,
between 3:00-5:00 p.m. PST
Industry working with rare disease patient advocacy
organizations to further the awareness of lentiviral gene therapy
clinical studies for Fabry disease and Gaucher disease type 1
(#305) Thursday, Feb. 10, 2022, between 3:00-5:00 p.m. PST
About AVROBIO Our vision is to bring personalized gene
therapy to the world. We aim to prevent, halt or reverse disease
throughout the body with a single dose of gene therapy designed to
drive durable expression of therapeutic protein, even in
hard-to-reach tissues and organs including brain, muscle and bone.
AVROBIO’s pipeline is powered by our industry-leading plato® gene
therapy platform, our foundation designed to deliver gene therapy
worldwide. It includes clinical programs in cystinosis and Gaucher
disease type 1, as well as preclinical programs in Gaucher disease
type 3, Hunter syndrome and Pompe disease. 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 product candidates, the
design, commencement, enrollment and timing of ongoing or planned
clinical trials, clinical trial results, product approvals and
regulatory pathways, 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 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.
______________________________ i Collaborator-sponsored Phase
1/2 clinical trial of AVR-RD-04 is funded in part by grants to UCSD
from the California Institute for Regenerative Medicine (CIRM),
Cystinosis Research Foundation (CRF) and National Institutes of
Health (NIH).
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220127005397/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
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