AVROBIO Announces First Patient Dosed Using plato Platform


Patient is the fourth dosed in Phase 2 trial of AVR-RD-01, an investigational gene therapy for Fabry disease

plato is AVROBIO's platform for worldwide gene therapy commercialization

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- 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 the first patient has been dosed using the Company's plato™ gene therapy platform, which is intended to provide the foundation for the potential worldwide commercialization of AVROBIO's gene therapies.

The plato platform includes a state-of-the-art lentiviral vector (LV2) designed to optimize vector copy number, transduction efficiency and resulting enzyme activity; a personalized conditioning regimen with precision dosing via therapeutic drug monitoring intended to enhance safety and engraftment; advanced cryopreservation to extend shelf life and enable flexible scheduling for patients; and an automated, closed-manufacturing process intended to improve consistency and predictability of the drug product. The Company expects that plato's self-contained manufacturing pods will facilitate global manufacturing site expansion.

The first patient dosed using plato is enrolled in FAB-201, the Company's Phase 2 trial of AVR-RD-01, an investigational gene therapy for Fabry disease. That trial is evaluating the safety and efficacy of AVR-RD-01 in 8 to 12 treatment-naïve patients, with ongoing recruitment in the U.S., Canada and Australia.

"Our plato platform sets AVROBIO apart. Early in AVROBIO's growth, we prioritized the development of plato to address and surmount key challenges facing gene therapy, and believe we have made great strides towards that goal," said Geoff MacKay, AVROBIO's President and Chief Executive Officer. "We have delivered improvements in manufacturing across the supply chain and into the clinic. We are proud to say that our gene therapy programs are powered by plato."

plato has received regulatory clearance for clinical use and will be used for all patients going forward in FAB-201, AVROBIO's Phase 2 Fabry disease trial, and in GAU-201, AVROBIO's Phase 1/2 clinical trial of AVR-RD-02, an investigational gene therapy for Gaucher disease.


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 progression 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 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 regarding our plato platform, including the expected benefits and results of our implementation of the plato platform in our FAB-201 trial and other programs, our business strategy, prospective products and goals, the therapeutic potential of our product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, and the anticipated benefits of our gene therapy platform including potential impact on our commercialization activities. 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 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.

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Investor Contact:
Christopher F. Brinzey
Westwicke, an ICR Company

Media Contact:
Tom Donovan
Ten Bridge Communications




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