AVROBIO, Inc. Announces Analyst and Investor Event and Upcoming Clinical Data Presentations at WORLDSymposium 2019

CAMBRIDGE, Mass., Jan. 17, 2019 (GLOBE NEWSWIRE) -- . (NASDAQ: AVRO) (the “Company”), a Phase 2 clinical-stage gene therapy company, today announced that clinical trial updates related to the Company’s gene therapy program for AVR-RD-01 in Fabry disease will be presented at WORLDSymposium, an annual conference dedicated to lysosomal diseases, being held February 4-8, 2019 in Orlando, Florida. AVROBIO will host an event for analysts and investors in conjunction with the conference, with a presentation and webcast beginning at 6:30 p.m. ET on Wednesday, February 6, 2019 in addition to the data presentations made as part of the WORLDSymposium program.   

AVROBIO’s analyst and investor event will expand on the presentations at the conference and highlight AVROBIO’s progress with the Phase 1 study and Phase 2 FAB-201 study¹ for its lead clinical program in Fabry disease; the Company’s optimized gene therapy platform; and other gene therapy pipeline programs in Gaucher, Cystinosis and Pompe. Featured speakers at the analyst and investor event include AVROBIO management and Dr. Mark Thomas, a specialist in Fabry disease and physician at the Department of Nephrology, Royal Perth Hospital and Clinical Professor at the University of Western Australia Medical School.

AVR‑RD‑01 is a gene therapy being investigated in patients with . Designed to be a one-time therapy, its mechanism of action is by inserting the GLA gene that encodes functional αgalactosidase A (AGA, the enzyme that is deficient in Fabry disease), with the goal of enabling continuous AGA production and distribution to tissues and organs. The data related to the AVR‑RD-01 clinical program in Fabry disease presented at WORLDSymposium includes:

Platform presentation: “FACTs Fabry gene therapy clinical trial: Phase 1 study update and two-year data for first patient”

Study: Investigator-sponsored Phase 1 study of AVR-RD-01 conducted  by the FACTs team (Fabry disease Clinical research and Therapeutics) in Canada 

Presenter: Jeffrey A. Medin, Ph.D., principal investigator of FACTs team and Vice Chair of Research Innovation and Professor, Pediatrics, Hematology/Oncology, Medical College of Wisconsin

Date: Wednesday, February 6, 2019 at 9:00 a.m. ET

Poster presentation: “Evaluating the Content Validity of the Diary of Irritable Bowel Syndrome Symptoms - Mixed (DIBSS-M) to Assess Gastrointestinal Symptoms Associated with Fabry”

Study: AVROBIO-sponsored Phase 2 trial of AVR-RD-01 (the FAB-201 Study¹)

Presenter: Dawn Phillips Ph.D., Senior Clinical Scientist, AVROBIO, Inc.

Date: Tuesday, February 5, 2019 at 4:30-6:30 p.m. ET

For AVROBIO’s analyst and investor event, a live webcast of the presentation and accompanying slides will be available under “” 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.

About AVROBIO, Inc.

., is a Phase 2 clinical-stage gene therapy company developing gene therapies to potentially cure rare diseases with a single dose. AVROBIO’s employ hematopoietic stem cells that are collected from the patient and then modified with a lentiviral vector to insert a functional copy of the gene that is defective in the target disease. AVROBIO is focused on the development of its gene therapy, AVR‑RD‑01, in , as well as additional gene therapy programs in other  including  ,  and  disease. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON. For additional information, visit . 

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 such as “aims,” “anticipates,” “believes,” “could,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,” “potential,” “seeks,” “will,” and variations of these words or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements about presenting at the WORLDSymposium annual conference and the types of data expected to be disclosed at the WORLDSymposium including the adjacent analyst and investor presentation. 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 of AVROBIO’s ongoing or planned clinical trials, the risk that AVROBIO may not realize the intended benefits of efforts to optimize its platform, and 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. 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 from those contained in the forward-looking statements, see the section entitled “Risk Factors” in AVROBIO’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2018, 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 The official name of the ‘FAB-201 study’ is AVRO-RD-01-201, which is a Phase 2 trial of AVROBIO’s investigational gene therapy AVR-RD-01, in Fabry disease.