AVROBIO Announces Multiple Clinical Data Presentations, Posters and Events at 17th Annual WORLDSymposium™ 2021

Analyst and investor event scheduled for Monday, Feb. 8 at 8:00 a.m. ET

Four platform presentations and two posters on the company’s clinical and preclinical programs for lysosomal disorders are scheduled throughout 17th annual WORLDSymposium™

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Feb. 1, 2021-- 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 it will host a virtual investor event to discuss new data from AVROBIO’s clinical programs in Fabry disease, cystinosis and Gaucher disease type 1 presented at the virtual 17th annual WORLDSymposium™, a scientific meeting dedicated to lysosomal disorders, Feb. 8-12, 2021.

The company also will host two medical symposia featuring perspectives from world-renowned clinicians on the latest developments in ex vivo lentiviral gene therapy for lysosomal disorders and the unmet need in Gaucher disease type 1.


Virtual investor event, Monday, Feb. 8, 2021, from 8:00-9:30 a.m. ET

Members of AVROBIO management will share new clinical data across the company’s Fabry disease, cystinosis and Gaucher disease type 1 programs. The conference call and presentation will begin at 8:00 a.m. ET and can be accessed under “Events and Presentations” in the Investors section of the company’s website at avrobio.com or by dialing (866) 353-0165 from locations in the U.S. and (409) 217-8080 from outside the U.S. The conference ID number is 3287052.

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.


“Long-term hematopoietic stem cell gene therapy corrects neuromuscular manifestations in preclinical study of Pompe mice,” Thursday, Feb. 11, 2021, at 11:00 a.m. ET

  • Niek van Til, Ph.D., senior director, gene therapy, AVROBIO, will present additional preclinical data for AVR-RD-03, an investigational gene therapy for Pompe disease.

“Analysis of genetically engineered stem cell product and follow up of gene therapy patients through high-throughput single cell technologies,” Thursday, Feb. 11, 2021, at 1:24 p.m. ET

  • Cristina Baricordi, Ph.D., scientist, AVROBIO, will present the company’s advanced analytics for characterizing drug product.

“Hematopoietic stem cell gene therapy for cystinosis: Updated results from a phase I/II clinical trial,” Friday, Feb. 12, 2021, at 11:00 a.m. ET

  • Stephanie Cherqui, Ph.D., principal investigator of the Phase 1/2 clinical triali 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) Genetic and Metabolic Diseases Committee, will present an update on a Phase 1/2 trial of AVR-RD-04 in cystinosis.

“AVR-RD-01, an investigational lentiviral gene therapy for Fabry disease: Overview of clinical data from phase 1 and phase 2 studies,” Friday, Feb. 12, 2021, at 11:24 a.m. ET

  • Mark Thomas, M.D., principal investigator of the AVROBIO-sponsored Phase 2 clinical 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 updates on both the Phase 1 and Phase 2 (FAB-201/FAB-GT) trials of AVR-RD-01 for Fabry disease.


The Guard1 clinical trial: A first-in-human, open-label, multinational phase 1/2 study of AVR-RD-02 ex vivo lentiviral vector, autologous gene therapy for Gaucher disease,” Thursday, Feb. 11, 2021, between 2:30-3:30 p.m. ET

  • Leslie Jacobsen, M.D., vice president, clinical development lead, AVROBIO, will present new data from the AVROBIO-sponsored Phase 1/2 trial of AVR-RD-02, an investigational gene therapy for Gaucher disease type 1.

“Uncovering the burden of Gaucher disease type 1: Patient perspectives on unaddressed symptoms, impact of disease, and the future of treatment,” Thursday, Feb. 11, 2021, between 2:30-3:30 p.m. ET

  • Meghan White, patient advocate, Ottawa, Canada and Lori Ann Correia, associate director, patient advocacy and engagement, AVROBIO, will present patient perspectives on the unmet needs of Gaucher disease type 1.

The presentations and posters are available online for WORLD attendees on the conference website.


“Harnessing the power of ex vivo lentiviral gene therapy: A personalized approach,” symposium, Wednesday, Feb. 10, 2021, from 12:00-1:00 p.m. ET

  • Dominique P. Germain, M.D., Ph.D., APHP – University Paris Saclay, France
  • Robert J. Hopkin, M.D., Cincinnati Children’s Hospital, U.S.
  • Paul J. Orchard, M.D., University of Minnesota, U.S.
  • Brian Bigger, Ph.D., University of Manchester, U.K.

“Unmet need in Gaucher disease: Future possibilities with ex vivo lentiviral gene therapy,” symposium, Thursday, Feb. 11, 2021, from 4:00-5:20 p.m. ET

  • Timothy M. Cox, M.Sc., M.D., FRCP, MAE, FMedSci, University of Cambridge, U.K.
  • Cyndi Frank, Co-Founder and Co-President of Gaucher Community Alliance, U.S.
  • Roy Alcalay, M.D., M.S., Columbia University Medical Center, U.S.
  • Aneal Khan, M.D., FRCPC, FCCMG, University of Calgary, Canada

WORLD attendees can attend the symposia as part of the regular meeting program online on the conference website.


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 functional protein, even in hard-to-reach tissues and organs including the 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 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, and anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success.

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 preclinical or 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 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.

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).

Christopher F. Brinzey
Westwicke, an ICR Company

Stephanie Simon
Ten Bridge Communications

Source: AVROBIO, Inc.

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