AVROBIO Announces Clinical and Regulatory Progress for Gaucher Disease Program

FDA clears IND application for AVR-RD-02

First patient consented in GAU-201, a global Phase 1/2 trial of AVR-RD-02 in Gaucher disease type 1

All global clinical sites will be supported by proprietary platoplatform

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jan. 14, 2020-- AVROBIO (NASDAQ: AVRO) (the “Company”), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that it has received notice of clearance from the U.S. Food and Drug Administration (FDA) regarding an Investigational New Drug (IND) application for AVR-RD-02, its investigational gene therapy for the treatment of Gaucher disease. This follows receipt of FDA orphan drug designation status for AVR-RD-02, and now clears AVROBIO to expand its ongoing Phase 1/2 clinical trial in Gaucher disease to the United States, supported by the Company’s proprietary plato™ gene therapy platform.

“Many people with Gaucher disease type 1 experience life-limiting symptoms even while on chronic enzyme replacement therapy. Our investigational therapy is designed to address these unmet needs with a single dose,” said Geoff MacKay, AVROBIO’s President and Chief Executive Officer. “We are eager to bring patients a fundamentally new approach with the potential to halt the progression of their disease and alleviate or even reverse symptoms not addressed by the standard of care. As we work toward that goal, we are pleased to announce important milestones in our Gaucher program, including IND clearance in the U.S. and consent from the first patient in our global GAU-201 trial.”

The standard of care for Gaucher disease type 1 is enzyme replacement therapy (ERT), which often does not halt disease progression or adequately address common life-limiting symptoms reported by Gaucher disease type 1 patients on ERT, such as fatigue and bone pain. Unlike ERT, AVR-RD-02 aims to provide a therapeutic benefit both systemically and throughout the central nervous system (CNS). AVROBIO believes AVR-RD-02 could slow, halt or potentially reverse symptoms throughout the entire body and brain, such as GBA-related Parkinson’s disease which occurs more frequently in people with Gaucher disease type 1.

AVROBIO has received consent from the first patient to enroll in GAU-201, a global Phase 1/2 trial of AVR-RD-02. The trial is actively recruiting in Australia and Canada, with additional sites planned in the United States. It is intended to recruit 8 to 16 patients between the ages of 16 and 35 with Gaucher disease type 1, including both those who are treatment-naïve and those who are stable on enzyme replacement therapy. AVROBIO’s gene therapy platform, plato, has now been cleared by regulators in Canada, Australia and the United States for use in the AVR-RD-02 clinical program.


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 are also advancing a program in Pompe disease. AVROBIO is powered by plato™, our streamlined platform for worldwide gene therapy commercialization. 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 prospective product candidates, including AVR-RD-02, our investigational gene therapy for Gaucher disease, the design, commencement, enrollment and timing of ongoing or planned clinical trials, including our GAU-201 Phase 1/2 clinical trial of AVR-RD-02 for Gaucher disease, 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, including our GAU-201 Phase 1/2 clinical trial of AVR-RD-02 for Gaucher disease, 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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