Press Release
Details

Updated data will be presented on first patient dosed in Phase 1/2 clinical trial for cystinosis and first patient treated using plato™ gene therapy platform in Phase 2 Fabry disease clinical trial

Company will present new data from preclinical AVR-RD-03 program for Pompe disease

CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 5, 2020-- 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 the company, along with its academic collaborators, will present four abstracts, including three oral and one poster presentation, and participate in two pre-meeting programs, at the 23rd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), which will be held virtually May 12-15, 2020. The oral presentations will include data updates for its investigational programs for Fabry disease and cystinosis, as well as preclinical data for its investigational program for Pompe disease.

Details on oral presentations:
Hematopoietic stem cell gene therapy for cystinosis: initial results from a Phase I/II clinical trial

• Stephanie Cherqui, Ph.D., principal investigator of the Phase 1/2 clinical trial of AVR-RD-04 for cystinosis, associate professor of pediatrics at University of California, San Diego (UCSD) School of Medicine and former chair of the ASGCT Gene and Cell Therapy of Genetic and Metabolic Diseases Committee. The collaborator-sponsored Phase 1/2 clinical trial of AVR-RD-04 for cystinosis is funded by grants to UCSD from the California Institute for Regenerative Medicine (CIRM), Cystinosis Research Foundation (CRF) and National Institutes of Health (NIH)
• Wednesday, May 13, 2020, 4:30-4:45 p.m. ET

Gb3 substrate in endothelial cells of renal peritubular capillaries was reduced in a previously untreated classic Fabry male patient treated with AVR-RD-01 investigational lentiviral gene therapy

• Includes updated data on first patient treated using plato gene therapy platform in AVROBIO’s Phase 2 Fabry clinical trial
• Birgitte Volck, M.D., Ph.D., president of R&D, AVROBIO
• Wednesday, May 13, 2020, 5:00-5:15 p.m. ET

Hematopoietic stem cell gene therapy corrects neuromuscular manifestations in preclinical study of Pompe mice

• Niek van Til, Ph.D., senior director, AVROBIO
• Thursday, May 14, 2020, 4:30-4:45 p.m. ET

Additional presentations:
Development and implementation of lentivirus-mediated gene therapy for Fabry disease

• Jeffrey A. Medin, Ph.D., principal investigator of the Fabry Disease Clinical Research and Therapeutics (FACTs) clinical study of AVR-RD-01, AVROBIO’s investigational gene therapy for Fabry disease, MACC Fund Professor, Pediatrics and Biochemistry, at the Medical College of Wisconsin
• Pre-meeting program: Monday, May 11, 2020, 8:00-8:30 a.m. ET

Commercial development of lentiviral vectors and ex vivo gene therapies

• Chris Mason, M.D., Ph.D., FRCS, FMedSci, chief scientific officer, AVROBIO
• Pre-meeting program: Monday, May 11, 2020, 3:35-3:55 p.m. ET

Macrophage tunneling nanotube-mediated HSPC transplantation therapy for the lysosomal storage disorder cystinosis remains effective in preclinical mouse study following SHPK elimination

• Spencer M. Goodman, graduate student, UCSD
• Poster session: Wednesday, May 13, 2020, 5:30-6:30 p.m. ET

The abstracts for the oral presentations and poster are available on the ASGCT Annual Meeting website.

About AVROBIO
Our mission is to free people from a lifetime of genetic disease with a single dose of gene therapy. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a program in Pompe disease. AVROBIO is powered by the plato™ gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. 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 prospective 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 development timeline 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 Annual or 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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Investor Contact:
Christopher F. Brinzey
Westwicke, an ICR Company
339-970-2843
chris.brinzey@westwicke.com

Media Contact:
Tom Donovan
Ten Bridge Communications
857-559-3397
tom@tenbridgecommunications.com

Source: AVROBIO, Inc.