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.
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