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Dr. Haiyan Fu’s team in the Center for Gene Therapy has developed a systemic gene delivery approach to restore the activity of the enzyme (α-N-acetylglucosaminidase) in the central nervous system and somatic system, showing functional benefits in treating MPS IIIB I mouse model.  Supported by a translational grant from the National Institutes of Health (Haiyan Fu/Kevin Flanigan), the team is working toward obtaining an investigational new drug approval from the Food and Drug Administration for a Phase I/II clinical trial in patients with MPS IIIB. Dr. Fu’s team is also working on developing tissue-specific biomarkers for MPS IIIB, which will be critical for assessing disease severity and therapeutic responsiveness as therapies become available.

Haiyan Fu, PhD, is a principal investigator at the Center for Gene Therapy, the Research Institute at Nationwide Children’s Hospital. She did her PhD training in virology in the Institute for Animal Health, UK and postdoc training in the laboratory of Dr. Joseph Muenzer, in the University of North Carolina at Chapel Hill. She initiated her own lab at the Center for Gene Therapy, the Research Institute at Nationwide Children’s Hospital in 2004. Her major research focus has been on the development of gene therapy approaches for neuropathic lysosomal storage diseases. Her work have been supported by multiple patient foundations and grants from the NIH, leading to recent IND approvals by the FDA for two Phase 1/2 gene therapy clinical trials in patients with MPS IIIA and MPS IIIB. Her research interests also include better understanding the neuropathology mechanisms and identifying biomarkers, especially neurological biomarkers for neuropathic lysosomal storage diseases.

 

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Joseph Muenzer, MD, PhD is Professor of Pediatrics and Genetics in the Division of Genetics and Metabolism, Department of Pediatrics at the University of North Carolina at Chapel Hill, USA, where he has practiced since 1993. He is actively involved in the diagnosis, management and treatment of patients with inborn errors of metabolism, especially mucopolysaccharidosis (MPS) and infants detected by tandem mass spectrometry newborn screening. Dr Muenzer received his medical degree (1976) and PhD (Biochemistry, 1979) from Case Western Reserve University in Cleveland, Ohio. He completed his residency in pediatrics at the University of Wisconsin Hospital in Madison, and a genetics/endocrine fellowship at the National Institutes of Health in Bethesda, Maryland. Dr Muenzer is board certified in pediatrics and clinical biochemical/molecular genetics. Dr Muenzer’s clinical research is directed toward the development of enzyme replacement therapy (ERT) for MPS. He was a principal investigator for the recombinant enzyme replacement clinical trials for MPS I and MPS II. Dr Muenzer is currently a principal investigator for the Phase I/II and Phase II/III clinical trials evaluating intrathecal ERT for the severe form of Hunter syndrome and a Phase IIb intrathecal trial for MPS IIIA all sponsored by Shire.