AVROBIO to Present New Data from Phase 1/2 Clinical Trial in Cystinosis at WORLDSymposium™ 2022

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- 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 18^th 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.

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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).

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