Mucopolysaccharidosis (MPS) IIIB (MPSIIIB) - Healing Genes

Mucopolysaccharidosis (MPS) IIIB (MPSIIIB)

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Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB (MPSIIIB)

Phase I/II Gene Transfer Clinical Trial of rAAV9.CMV.hNAGLU for Mucopolysaccharidosis (MPS) IIIB

Phase 1 / 2


The Nationwide Children’s Hospital in Columbus, OH, is recruiting patients with Mucopolysaccharidosis (MPS) IIIB (MPSIIIB) for a clinical trial of a gene therapy that will attempt to impart a functional copy of the NAGLU gene. This therapy is a genetically-engineered virus that delivers the gene, and treatment will involve a one-time infusion in addition to a tapering course of steroids. Improved health and cognitive ability are some of the goals of this treatment.

Follow up for 2 years will include lab testing and clinical assessments to track NAGLU enzyme activities and overall health.


  • Be 6 months of age or older
  • Have a confirmed diagnosis of MPSIIIB by both of the following two methods:
    • No detectable or significantly reduced NAGLU enzyme activity by plasma, serum, or leukocyte assay.
    • Genomic DNA analysis demonstrating homozygous or compound heterozygous mutations in the NAGLU gene
  • Not have received prior treatment with NAGLU enzyme replacement therapy (ERT)


  1. Screening before the treatment, possibly including DNA assessments
  2. A single infusion of the gene therapy drug will be administered at one of two dose levels.
  3. Monitoring of the patient for a few hours after dosing, with lab, physical, and cognitive assessments required for up to 2 years post treatment.


Trials will take place at the Nationwide Children’s Hospital in Columbus, OH. Map.

Federica Rinaldi, PhD  |   614-355-2897  |  [email protected]
Tabatha Simmons, PhD  |  614-722-6921  |  [email protected]
Abeona Therapeutics, Inc
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