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AVROBIO Announces Clinical and Regulatory Progress for Gaucher Disease Program
AVROBIO Announces Clinical and Regulatory Progress for Gaucher Disease Program
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 plato™ 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
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).
About
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.
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
View source version on businesswire.com: https://www.businesswire.com/news/home/20200114005177/en/
Source:
Investor Contact:
Christopher F. Brinzey
Westwicke, an ICR Company
339-970-2843
chris.brinzey@westwicke.com
Media Contact:
Tom Donovan
Ten Bridge Communications
857-559-3397
tom@tenbridgecommunications.com