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UNC Pediatrics delivers investigational genome editing therapy in clinical trial for the rare Hunter Syndrome

Dr. Joseph Muenzer
Dr. Joseph Muenzer

First gene therapy performed by Dr. Joseph Muenzer and the UNC Clinical Translational and Research Center.

This week, a 40-year-old patient was treated at UNC's Clinical and Translational Research Center (CTRC) with SB-913, an investigational genome editing therapy for individuals with mucopolysaccharidosis type II (MPS II), a rare lysosomal storage disorder also known as Hunter syndrome.

This treatment marks a significant milestone for Joseph Muenzer, MD, PhD, and the CTRC. It was the first time that Muenzer, a pediatric biochemical geneticist who specializes in disorders such as Hunter syndrome, has had the opportunity to administer a gene therapy treatment to a patient with this condition.

"I developed a mouse model for Hunter syndrome at UNC about twenty years ago to help develop therapies for this rare disorder," said Dr. Muenzer. "I spent many years working on gene therapy in my UNC research lab and it is with great pleasure that I am able to finally perform gene therapy on an individual with Hunter syndrome. Gene therapy has the potential to dramatically improve the quality of life for individuals with Hunter syndrome."

The treatment, SB-913, was developed by Sangamo Therapeutics, a biotechnology company in the San Francisco Bay Area in California. Sangamo is currently evaluating the treatment in a Phase 1/2 clinical trial, called the CHAMPIONS study. The patient at UNC was the third patient to receive the treatment so far as part of the CHAMPIONS study.

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Michelle Maclay, Communications Director