AVR‑RD-04 is designed as one-time gene therapy and is being investigated for the treatment of cystinosis by inserting the functional gene for human cystinosin (CTNS), and is designed to maximize the likelihood of sustained CTNS production in patients to correct for the single-gene defect in the CTNS gene that causes cystinosis.
"We are pleased to collaborate with Dr. Stephanie Cherqui of the University of California, San Diego, and we share the vision of her and her team, and the dedicated patient advocates in the cystinosis community, to develop what we expect will be a single-dose and potentially transformative gene therapy for the treatment of people with cystinosis," said Geoff MacKay, President and CEO of AVROBIO. "We believe there is a significant opportunity for gene therapy for a range of lysosomal storage diseases, and we are building a pipeline of product candidates to bring this new treatment paradigm to patients."
Cystinosis is a rare, genetic, autosomal recessive, lysosomal storage disease caused by the accumulation of the amino acid cystine. Cystine crystals build up and cause complications in many organs and tissues. The most severe form of cystinosis begins in infancy, causing poor growth and a particular type of kidney damage. Untreated children with cystinosis may experience kidney failure, and other symptoms include muscle deterioration, blindness, inability to swallow, diabetes, thyroid and nervous system problems. More than 90% of untreated patients require a kidney transplant before the age of 20. It is estimated that cystinosis disease is diagnosed in approximately one in 170,000 people.
About AVROBIO, Inc.
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 lentiviral-based gene therapiesemploy 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 Fabry disease, as well as additional gene therapy programs in other lysosomal storage disordersincluding Gaucher disease, Cystinosis and Pompe disease. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON. For additional information, visit www.avrobio.com.
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 business strategy, product candidates, the design, enrollment and timing of planned clinical trials, including those conducted by our collaborators, the potential of our gene therapy paradigm, 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.
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 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.
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