AVROBIO Receives Orphan Drug Designation from U.S. FDA for AVR‑RD‑04 for Cystinosis
AVROBIO Receives Orphan Drug Designation from U.S. FDA for AVR‑RD‑04 for Cystinosis
AVR-RD-04 is the third
“People living with cystinosis need new treatment options to keep cystine from accumulating in the lysosomes of cells, which leads to corneal damage and kidney deterioration, among other complications. Although the current standard of care has improved the outlook for this community, it does not halt disease progression or a wide range of debilitating complications which can severely impact daily lives,” said
Orphan drug designation is granted by FDA to drugs and biologics which are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that affect fewer than 200,000 people in the
About Cystinosis
Cystinosis is a rare, progressive disease marked by the accumulation of cystine in cellular organelles known as lysosomes, causing debilitating symptoms including eye complications (such as severe photophobia), muscle wasting and kidney failure, which often lead to a shortened lifespan. More than 90 percent of untreated patients require a kidney transplant before age 20. The current standard of care for cystinosis, a burdensome treatment regimen that causes severe halitosis and can amount to dozens of pills a day, does not halt disease progression.
About AVR-RD-04
AVR-RD-04 is an investigational, lentiviral-based gene therapy designed to potentially halt or reverse the progression of cystinosis with a single dose of the patient’s own hematopoietic stem cells. Before being transplanted into the patient, the stem cells are collected and genetically modified to express functional cystinosin, a transport protein which reduces the cystine build-up in the lysosomes of cells that cause the symptoms of cystinosis.
The open-label, single-arm Phase 1/2 clinical trial evaluating the safety and efficacy of AVR-RD-04 is being conducted under the name CTNS-RD-04 by the company’s academic collaborators at
About
Our 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 also are 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 the development and continued progress of our 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 ongoing Phase 1/2 trial of the AVR-RD-04 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.
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
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
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