samedi 16 janvier 2021

AVROBIO Announces Clinical Data Presentation and Multiple Events at WORLDSymposium™ 2020

Par Rédaction , dans Communiqués , le 30 mars 2020

Tuesday, February 4th 2020 at 12:00pm UTC

Analyst and investor event to be webcast on Monday, Feb. 10, 2020, at 7:00 p.m. ET

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 the company will provide clinical data updates for its investigational programs in Fabry disease and cystinosis, as well as present data from the first clinical use of its plato™ gene therapy platform, including an optimized vector, automated manufacturing system and personalized conditioning regimen. These data will be presented at the WORLDSymposium™, an annual scientific meeting dedicated to lysosomal disorders, Feb. 10-13, 2020, in Orlando, Fla.

Analyst and investor event, Monday, Feb. 10, 2020, from 7:00-9:00 p.m. ET

In addition to members of AVROBIO management, featured speakers will include leading researchers in the area of lysosomal disorders and gene therapy:

  • 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; associate professor of pediatrics at University of California, San Diego, School of Medicine; and chair of the American Society of Gene and Cell Therapy (ASGCT) Gene and Cell Therapy of Genetic and Metabolic Diseases Committee;
  • 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; vice chair of research innovation and MACC Fund professor, pediatrics (hematology/oncology) at the Medical College of Wisconsin; and
  • Mark Thomas, M.D., lead investigator for the company-sponsored Phase 2 clinical trial of AVR-RD-01 (FAB-201) for Fabry disease; nephrologist at the Department of Nephrology, Royal Perth Hospital; and clinical professor at the University of Western Australia Medical School.

A live webcast of the presentation and accompanying slides will be available at 7:00 p.m. ET under “Events and Presentations” in the Investors section of the company’s website at An archived webcast recording of the event will be available on the website for approximately 30 days.

Satellite symposium, “Lysosomal disorders: Lentiviral gene therapy and the promise of freedom for life,” Tuesday, Feb. 11, 2020, from 6:30-8:30 p.m. ET

In addition to members of AVROBIO management, featured speakers will include Drs. Cherqui, Medin and Thomas, as well as:

  • Harry L. Malech, M.D., symposium chair and chief of the Genetic Immunotherapy Section, and deputy chief, Laboratory of Clinical Immunology and Microbiology in the National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health; and
  • Aneal Khan, M.D., FACTs project investigator; and assistant professor, Departments of Medical Genetics and Pediatrics at the University of Calgary Cumming School of Medicine.

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

  • Birgitte Volck, M.D., Ph.D., president of research & development, AVROBIO, will present an update on the AVROBIO-sponsored Phase 2 trial of AVR-RD-01 in Fabry disease (FAB-201).
  • Poster presentation: Wednesday, Feb. 12, 2020, at 4:30-6:30 p.m. ET.
  • Contemporary Forum platform presentation: Thursday, Feb. 13, 2020, at 1:45 p.m. ET.


AVROBIO’s 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, 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, and anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success. 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, 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 on Form 10-Q, 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.


Investor Contact:

Christopher F. Brinzey

Westwicke, an ICR Company


Media Contact:

Tom Donovan

Ten Bridge Communications



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