2022-07-13 | NDAQ:AVRO | Press release

AVR-RD-05 is the fourth AVROBIO gene therapy to receive orphan drug designation

The FDA previously granted Rare Pediatric Disease Designation to AVR-RD-05

AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a common goal of freeing people from a lifetime of genetic disease, today announced that the Food and Drug Administration ( US FDA) has granted orphan drug designation to AVR-RD-05, its gene therapy for the treatment of mucopolysaccharidosis type II (MPSII), or Hunter syndrome, a rare and severely debilitating lysosomal storage disease that affects mainly young boys.

Orphan drug designation is granted by the FDA to drugs and biologics intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug designation offers certain incentives, which may include tax credits. to cover the cost of clinical trials and waiver of prescription drug user fees.

Hunter syndrome is caused by a deficiency in the lysosomal enzyme iduronate-2-sulfatase (IDS), which is essential for breaking down large sugar molecules. Gene therapy uses the patient’s own hematopoietic stem cells (HSCs) which are transduced ex-vivo with a lentiviral vector encoding the human IDS enzyme. The company-planned, collaborator-sponsored Phase 1/2 clinical trial for Hunter Syndrome is expected to begin in 2023 as part of the company’s collaboration with the University of Manchester, UK. The program was developed by Brian Bigger, Ph.D., Professor of Cell and Gene Therapy at the University of Manchester, who previously published preclinical data demonstrating that HSC gene therapy deploying an optimized proprietary tag has the potential to correct peripheral diseases and normalize brain pathology.

About Hunter Syndrome

Hunter syndrome, which affects approximately one in 100,000 to one in 170,000 men worldwide, causes devastating complications throughout the body, including severe neurological, cardiac and respiratory dysfunctions, skeletal malformations and hearing impairment. Children with severe cases of Hunter syndrome typically show early symptoms of the disease during infancy and childhood and begin to regress developmentally, losing basic motor skills and cognitive function within a few years. The current standard of care is weekly enzyme replacement therapy (ERT), which may delay some health complications but does not arrest overall disease progression and has not been shown to resolve central nervous system (CNS) problems. ). Even with ERT, people with Hunter syndrome face life-limiting symptoms and a significantly reduced lifespan.


Our vision is to bring personalized gene therapy to the world. Our goal is to prevent, halt or reverse disease throughout the body with a single dose of gene therapy designed to promote long-lasting expression of therapeutic proteins, even in hard-to-reach tissues and organs, including the brain , muscles and bones. AVROBIO’s pipeline is powered by our industry-leading platform® gene therapy platform, our foundation designed to deliver gene therapy worldwide. It includes clinical programs in cystinosis and Gaucher disease type 1, as well as preclinical programs in Gaucher disease type 3, Hunter syndrome and Pompe disease. Our headquarters are located in Cambridge, Massachusetts. For more 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. Such statements may be identified by words and phrases such as “objective”, ” “anticipates,” “believes,” “could,” “designed for,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible ,” “potential,” “seeks,” “will,” and variations of these similar words and phrases or phrases that are intended to identify forward-looking statements. These forward-looking statements include, but are not limited to, statements regarding our business strategy and the potential therapeutic benefits of our preclinical and clinical product candidates, including AVR-RD-05 for the treatment of Hunter syndrome, the potential benefits and incentives provided by the FDA’s orphan drug designation for AVR-RD-05, the design, initiation, enrollment and timing of planned clinical trials, results of preclinical or clinical trials, product approvals and regulatory pathways, our plans and expectations regarding interactions with regulators, anticipated benefits of our gene therapy platform, including potential impact on our commercialization activities, timing and likelihood of success, benefits and expected outcomes of our implementation of the plato platform in our clinical trials and research programs gene therapy, and the expected safety profile of our preclinical and investigational gene therapies. Such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results of preclinical or early-stage clinical trials may not be indicative of results of later-stage or larger clinical trials and do not guarantee regulatory approval. You should not place undue reliance on these statements or the scientific data presented.

All forward-looking statements contained in this press release are based on AVROBIO’s current expectations, estimates and projections regarding our industry as well as management’s current beliefs and expectations regarding future events only as of the date and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that one or more of AVROBIO’s product candidates will not be successfully developed or commercialized, the risk of termination or delay of any ongoing clinical trial or of AVROBIO or our collaborators, the risk that AVROBIO will fail to recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO will not realize the expected benefits of our gene therapy platform, including features of our plato® platform, the risk that our product candidates or the procedures related to their administration will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as safety signals, activity or durability of effect, observed from preclinical studies or clinical trials, will not be replicated or continued in ongoing or future studies or trials involving AVROBIO’s product candidates, the risk that we will not our ability 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 relating to our dependence on – vis-à-vis third-party suppliers and manufacturers, risks relating to the accuracy of our estimates of future expenses and revenues, risks relating to our capital requirements and our need for additional financing , clinical trial risks, and business interruptions resulting from the COVID-19 outbreak, public health or similar crises, including that such interruptions may significantly delay our enrollment and development timelines and/or increase our costs development or data collection efforts may be impaired or otherwise affected by such crises, and risks 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, each 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, 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 forward-looking statements, except as required by law.

About Hector Hedgepeth

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