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AVROBIO to Present Clinical and Preclinical Data from Lysosomal Disorder Gene Therapy Pipeline at WORLDSymposium™ 2023

Four platform presentations and two posters with data on AVROBIO’s lysosomal disorder programs to be presented

AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today announced that updated data suggesting complete biochemical correction in the first pediatric Gaucher disease type 3 (GD3) patient treated with hematopoietic stem cell (HSC) gene therapy, including improvements in major refractory elements of disease, will be presented at the 19th Annual WORLDSymposium™ in Orlando, Florida, Feb. 22-26, 2023.

The company also announced that data from the fully enrolled, collaborator-sponsored Phase 1/2 clinical trial in cystinosis will be presented at the conference.

The company’s planned activities are listed below and the full preliminary program is available online on the WORLDSymposium™ website.


Validation of a GMP stem cell gene therapy manufacturing process for mucopolysaccharidosis type II (MPS II) in preparation for an approved Phase 1/2 clinical trial

Thursday, Feb. 23, 2023, 8:00a.m. ET

  • Stuart Ellison, Ph.D., University of Manchester, UK, will present murine data for a central nervous system (CNS)-targeted HSC gene therapy and plans for a Phase 1/2 clinical trial in MPS II or Hunter syndrome.

Sustained improvement of clinical CNS and somatic features of GD3 after HSC gene therapy: A first-in-world report

Thursday, Feb. 23, 2023, at 2:00p.m. ET

  • Aimee Donald, M.D., Ph.D., University of Manchester, UK, will present clinical data from the first pediatric GD3 patient dosed with investigational AVR-RD-02.

Phase 1/2 clinical trial of autologous hematopoietic stem and progenitor cell (HSPC) gene therapy for cystinosis

Saturday, Feb. 25, 2023, 9:00a.m. ET

  • Stephanie Cherqui, Ph.D., University of California San Diego and principal investigator of the collaborator-sponsored Phase 1/2 ongoing clinical trial of CTNS-RD-04 for cystinosis, will present updated data from the fully enrolled trial.

The Guard1 clinical trial – A first in-human, Phase 1/2 study evaluating AVR-RD-02, an HSC gene therapy for Gaucher disease: Preliminary safety, pharmacodynamic and clinical efficacy results from the subjects observed for up to 24 months post-infusion

Saturday, Feb. 25, 2023, 9:00a.m. ET

  • Mathews Adera, M.D., AVROBIO, will present safety and efficacy data on AVR-RD-02, an investigational gene therapy for Gaucher disease type 1.


Validation of an assay to measure iduronate-2-sulfatase activity in cerebrospinal fluid to assess the efficacy of an HSC gene therapy (#123)

Thursday, Feb. 23, 2023, 3:00-4:00p.m. ET

Using IVIM/SAGA as screening tools during lentiviral vector lead selection for detection of clinically translatable insertional transformational risk (#283)

Saturday, Feb. 25, 2023, 4:00-5:00p.m. ET


Our vision is to bring personalized gene therapy to the world. We target the root cause of genetic disease by introducing a functional copy of the affected gene into the patient’s own hematopoietic stem cells (HSCs), with the goal of durably expressing the therapeutic protein throughout the body, including the central nervous system. Our first-in-class pipeline includes clinical programs for Gaucher disease and cystinosis, as well as preclinical programs for Hunter syndrome and Pompe disease. Our proprietary plato® gene therapy platform is scalable for planned global commercialization. We are headquartered in Cambridge, Mass. 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,” “continue,” “could,” “designed to,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,” “potential,” “predicts,” “projects,” “seeks,” “strives,” “should,” “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 preclinical and clinical product candidates, including without limitation the use of AVR-RD-02 for the treatment of Gaucher disease in both a clinical and a compassionate use or named patient setting, the design, commencement, enrollment and timing of planned clinical trials, our plans and expectations with respect to the development of our clinical and preclinical product candidates, including timing, design, and initiation of our potential clinical and registration trials and anticipated interactions and expectations with regulatory agencies, the timing of patient recruitment and enrollment activities, preclinical, compassionate use or 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 our plato® platform in our clinical trials and gene therapy programs, and the expected safety profile of our preclinical and 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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