Hunter’s Syndrome - Healing Genes

Hunter’s Syndrome

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RGX-121 Gene Therapy in Patients With MPS II (Hunter Syndrome)

A Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of RGX-121 in Pediatric Subjects With MPS II (Hunter Syndrome)

Phase 1/2


Doctors are seeking male participants aged 4 months to 5 years old for a trial gene therapy to treat severe Hunter’s Syndrome. This gene therapy will be delivered directly to the brain, using a harmless virus as a way to transport a normal gene to replace the mutated gene that causes Hunter’s Syndrome.

Study participants will undergo a single dose of the trial therapy and be closely observed for the first 24 weeks post the treatment, with re-assessments at 48, 78, and 104 weeks. The researchers hope to see patients begin to normally produce the iduronate-2-sulfatase enzymes lacking due to their disease.


  • Be 4 months to 5 years old and male
  • Must meet any of the following criteria:
    • Has a documented diagnosis of MPS II and a has a neurocognitive testing score > 55 and ≤ 77 (Bayley or Kaufman), OR
    • Has a documented diagnosis of MPS II AND has a decline of ≥ 1 standard deviation on serial neurocognitive testing (Bayley or Kaufman) and a testing score > 55, OR
    • Has a relative diagnosed with severe MPS II who has the same IDS mutation as the subject AND in the opinion of a geneticist has inherited a severe form of MPS II


  1. Screening before the treatment
  2. Under anesthesia, a CT scan-guided injection to the spinal cord will deliver the treatment to the brain, inside the blood-brain barrier
  3. The patients will be observed closely for 24 weeks, then have visits at 48, 78, and 104 weeks.


Trials will take place at the Children’s Hospital of Pittsburgh – UPMC: Program for Neurodevelopment in Rare Disorders. Map.

Contact: Jacob Wesley, Pharm D, MS  |  [email protected]  | 240-552-8181

Contact: Jodi Martin  |  412-692-6351  |  [email protected]

Contact: Dr. Maria Escolar |  [email protected]



Regenxbio Inc.


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