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AVROBIO Highlights the plato™ Platform and Gaucher and Cystinosis Programs at the 2019 Annual Meeting of the American Society of Gene and Cell Therapy

BusinessWire

CAMBRIDGE, Mass.–(BUSINESS WIRE)–AVROBIO,
Inc. (NASDAQ: AVRO) (the “Company”), a Phase 2 clinical-stage gene
therapy company, today announced the participation of its senior
management in sessions at the Annual Meeting of the American Society of
Gene and Cell Therapy (ASGCT). These include sessions on advances in
gene therapy research, manufacturing challenges during the development
of gene therapy products, and the potential benefits of the Company’s
plato™ platform. In addition, multiple oral presentations with
preclinical data from the Company’s gene therapy programs in Gaucher
disease and cystinosis are also on the program at the ASGCT Annual
Meeting.

“After more than three and a half years of development, we continue to
be on track towards our anticipated introduction of plato™ upgrades into
our clinical programs in the second half of 2019,” said Geoff
MacKay, AVROBIO’s President and CEO. “The plato platform includes a
proprietary vector system, an automated cell manufacturing solution and
refined conditioning regimen with therapeutic drug monitoring. We’re
especially pleased that our Head of Operations, Kim
Warren, PhD, is able to share our manufacturing and operations
experience with the gene and cell therapy community at ASGCT. The plato
platform is designed to overcome many of the identified constraints and
challenges of developing, manufacturing and commercializing gene
therapies.”

Dr. Warren is an invited expert panelist at the Pre-approval
Commercialization Workshop on “Manufacturing Challenges During Late
Phase Development of Gene Therapy Products.” During a session on the
latest advances in cell therapy research, AVROBIO’s Senior Director of
Manufacturing Operations, Azadeh Golipour, PhD, will present “Automated
Gene Modified Cell Production: Concept to Process Verification” which
describes the development of the automated cell process.

In several oral presentations at ASGCT, preclinical data will be
presented by AVROBIO’s academic collaborators and their colleagues,
related to the Company’s gene therapy programs in Gaucher disease and
cystinosis.

Gaucher disease ASGCT oral presentation and gene therapy program

An oral presentation will be made at ASGCT with preclinical data results
related to the gene therapy program for Gaucher disease led by Stefan
Karlsson, MD, PhD, Professor at the Division of Molecular Medicine and
Gene Therapy at Lund University in Sweden. AVROBIO has received
clearance for its clinical trial application (CTA) from Health Canada.
The Company plans to initiate its GAU-201 Phase 1/2 clinical trial of
AVR-RD-02 in patients with Gaucher disease in the second half of 2019
utilizing the plato platform.

Cystinosis ASGCT oral presentations and gene therapy program

Three oral presentations will be made at ASGCT related to the cystinosis
gene therapy program led by Stephanie Cherqui, PhD, Associate Professor
at the Department of Pediatrics at University of California, San Diego
(USCD). In January 2019, AVROBIO announced that the U.S. Food and Drug
Administration (FDA) had cleared the investigational new drug (IND)
application for the planned investigator-sponsored Phase 1/2 clinical
trial for AVR-RD-04 gene therapy in cystinosis to be conducted by UCSD.
Rights are licensed to AVROBIO to develop AVR-RD-04 as a
potential one-time gene therapy for the treatment of cystinosis.

“We believe that Dr. Karlsson’s Gaucher mouse model proof-of-concept
work shows multiple positive preclinical findings, including prevention
and reversal of disease manifestations, as well as improvement trends in
multiple bone parameters. Bone data are important because according to
The National Gaucher Foundation bone problems are common in people
living with Gaucher disease,” said Birgitte
Volck, MD, PhD, President of Research and Development at AVROBIO.
“Stephanie Cherqui, PhD, and her team are also sharing valuable insights
into ex vivo gene therapy for cystinosis. Both of these programs, for
Gaucher and cystinosis, are expected to begin clinical studies in the
second half of 2019.”

About AVROBIO, Inc.

AVROBIO,
Inc., is a Phase 2 clinical-stage gene therapy company developing
gene therapies to potentially cure rare diseases with a single dose.
AVROBIO’s lentiviral-based
gene therapies employ hematopoietic stem cells that are collected
from the patient and then modified with a lentiviral vector to insert
functional copies of the gene that is defective in the target
disease. AVROBIO is focused on the development of its gene therapy,
AVR-RD-01, in Fabry
disease, as well as additional gene therapy programs in other lysosomal
storage disorders including Gaucher
disease, cystinosis and Pompe
disease. The Company’s plato platform is a proprietary vector system
and automated, closed cell manufacturing solution designed to support
worldwide commercialization. AVROBIO is headquartered in Cambridge,
MA and has offices in Toronto, ON. For additional information, visit www.avrobio.com.

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 such as “aims,” “anticipates,” “believes,” “could,”
“estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,”
“possible,” “potential,” “seeks,” “will,” and variations of these words
or similar expressions that are intended to identify forward-looking
statements. These forward-looking statements include, without
limitation, statements regarding our business strategy, prospective
products and goals, the therapeutic potential of our product candidates,
anticipated benefits of our gene therapy platform including potential
impact on our commercialization activities, the design, commencement,
enrollment and timing of ongoing or planned clinical trials, and the
timing, scope and likelihood of regulatory filings and approvals. 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 the date of this release 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 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. 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 Annual Report on Form 10-K for the fiscal year ended December
31, 2018, 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.

Contacts

Investor Contact:
Christopher F. Brinzey
Westwicke Partners
339-970-2843
chris.brinzey@westwicke.com

Media Contact:
Kathryn Morris
The Yates Network
914-204-6412
kathryn@theyatesnetwork.com

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