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     114  0 Kommentare AVROBIO Expands Lentiviral Gene Therapy Pipeline with Program for Hunter Syndrome

    AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced an exclusive, worldwide license agreement and a collaborative research funding agreement with The University of Manchester for an investigational lentiviral gene therapy for mucopolysaccharidosis type II (MPS II), or Hunter syndrome, a rare and deadly lysosomal disorder that primarily affects young boys.

    Hunter syndrome, which affects an estimated one in 100,000 to one in 170,000 males worldwide, causes devastating complications throughout the body and brain, including severe cardiac and respiratory dysfunction, skeletal malformations and hearing impairment. Children with severe cases of Hunter syndrome typically show early symptoms in their toddler years and begin to regress developmentally around age six, losing basic motor skills and cognitive function. The current standard of care is weekly enzyme replacement therapy (ERT), which can delay some complications but does not halt overall progression of the disease and has not been demonstrated to address cognitive issues. Even with ERT, people with Hunter syndrome face life-limiting symptoms and a significantly reduced life span.

    “We believe a lentiviral gene therapy approach is well suited to treat a progressive and pervasive disease such as Hunter syndrome, which affects organs throughout the body and severely impairs cognitive function. If we treat children early, before their symptoms arise, we hope to prevent the tragic complications that rob these young children of their futures,” said Geoff MacKay, AVROBIO’s president and CEO. “We believe our deep experience with investigational gene therapies for lysosomal disorders will enable us to efficiently move the program through clinical development in collaboration with Prof. Brian Bigger, who has done tremendous work to develop and optimize this investigational gene therapy. We’re proud to add this program to our leading lysosomal disorder pipeline and excited about its potential to change the lives of patients and families living with Hunter syndrome.”

    The investigator-sponsored Phase 1/2 clinical trial for Hunter syndrome is expected to enter the clinic in the second half of 2021. The program was developed by Brian Bigger, Ph.D., a professor of cell and gene therapy at the University of Manchester, U.K. Prof. Bigger has published preclinical data demonstrating that the introduction of the transgene with an optimized, proprietary tag has the potential to correct peripheral disease and normalize brain pathology. Primary investigators for the Phase 1/2 clinical trial will be Prof. Robert Wynn, M.D., consultant pediatric hematologist at the Royal Manchester Children’s Hospital, and Dr. Simon Jones, MBChB, consultant pediatric physician for inherited metabolic diseases at the Willink Unit, Saint Mary’s Hospital and the Manchester Centre for Genomic Medicine.

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    AVROBIO Expands Lentiviral Gene Therapy Pipeline with Program for Hunter Syndrome AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced an exclusive, worldwide license agreement and a collaborative research funding agreement …