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AVROBIO to Present Clinical and Preclinical Data at the ASGCT 24th Annual Meeting

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 two oral presentations and two posters featuring data from its pipeline of lysosomal disorder gene therapy programs will be shared at the 24th virtual Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), May 11-14, 2021.

Accepted abstracts will include new data points from an investigator-sponsored Phase 1/2 trial1 in cystinosis and from AVROBIO’s preclinical program in Pompe disease. Encore data from AVROBIO’s clinical programs in Fabry disease and Gaucher disease type 1 will also be presented.

The presentations are listed below and the full preliminary program is available online at the ASGCT website.


“Long-term hematopoietic stem cell lentiviral gene therapy corrects neuromuscular manifestations in preclinical study of Pompe mice”

Tuesday, May 11, 2021, 6:30-6:45 p.m. ET

Niek van Til, Ph.D., senior director, gene therapy, AVROBIO, will present new preclinical data for AVR-RD-03, a gene therapy for Pompe disease, and answer questions in a live Q&A.

“AVR-RD-01, an investigational lentiviral gene therapy for Fabry disease: Clinical data trends from Phase 1 and Phase 2 studies up to 3.5 years”

Friday, May 14, 2021, 12:30 p.m. - 12:45 p.m. ET

Mark Thomas, M.D., principal investigator of the AVROBIO-sponsored FAB-GT Phase 2 trial of AVR-RD-01, an investigational gene therapy for Fabry disease, nephrologist at the Department of Nephrology, Royal Perth Hospital and clinical professor at the University of Western Australia Medical School, will present encore data and answer questions in a live Q&A.


“Hematopoietic stem cell lentiviral gene therapy for cystinosis: Updated results from a Phase 1/2 clinical trial”

Stephanie Cherqui, Ph.D., principal investigator of the Phase 1/2 clinical trial of AVR-RD-04 (CTNS-RD-04), an investigational gene therapy for cystinosis, and associate professor of pediatrics at University of California, San Diego, School of Medicine, will present new data on the third patient dosed in the trial and recap existing data on the first two patients dosed.

“Preliminary results from the Phase 1/2 Guard1 trial: A first-in-human study of ex vivo lentiviral gene therapy (AVR-RD-02) in people with Gaucher disease type 1.”

Leslie Jacobsen, M.D., vice president, clinical development lead, AVROBIO, will present encore data from the first patient dosed in the Phase 1/2 trial of AVR-RD-02, an investigational therapy for Gaucher disease type 1.


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. Our ex vivo lentiviral gene therapy pipeline includes clinical programs in Fabry disease, Gaucher disease type 1 and cystinosis, as well as preclinical programs in Hunter syndrome, Gaucher disease type 3 and Pompe disease. AVROBIO is powered by our industry leading plato® gene therapy platform, our foundation designed to deliver gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit, 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 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.


1 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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