AVROBIO Receives Orphan-Drug Designation from the U.S. FDA for AVR‑RD‑02 for the Treatment of Gaucher Disease
AVROBIO Receives Orphan-Drug Designation from the U.S. FDA for AVR‑RD‑02 for the Treatment of Gaucher Disease
“Under the existing standard of care, patients with Gaucher disease are bound to a lifelong infusion schedule of enzyme replacement therapies, and still experience painful and progressive symptoms such as debilitating musculoskeletal pain and fatigue,” said
Orphan-drug designation is granted by the
About AVR-RD-02
AVR-RD-02 is an ex vivo lentiviral-based investigational gene therapy designed to provide durable and potentially life-long therapeutic benefit for patients with Gaucher disease with a single dose of the patient’s own hematopoietic stem cells. The stem cells are genetically modified to express functional glucocerebrosidase (GCase), which reduces levels of glucosylceramide and glucosylsphingosine, the accumulated substances which cause the symptoms of Gaucher disease.
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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 AVROBIO’s expectations regarding the development and the continued progress of AVROBIO’s programs, including the intended incentives conferred by orphan-drug designation, the therapeutic potential of our product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, including the Phase 1/2 clinical trial of the Company’s AVR-RD-02 investigational gene therapy, the anticipated benefits of our gene therapy platform, the expected safety profile of our product candidates, timing and likelihood of success of our current or future product candidates, and the market opportunity for our product candidates. 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.
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Investor Contact:
Christopher F. Brinzey
Westwicke, an ICR Company
339-970-2843
chris.brinzey@westwicke.com
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Ten Bridge Communications
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