Mucopolysaccharidosis type I (MPS I) - Healing Genes

Mucopolysaccharidosis type I (MPS I)

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RGX-111 Gene Therapy in Patients With MPS I

A Phase I Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of RGX-111 in Subjects With MPS I

Phase 1


Doctors at Children’s Hospital of Philadelphia and the University of Pennsylvania seek patients with Mucopolysaccharidosis type I (MPS I) to trial therapeutic gene therapy. The therapy uses a harmless virus to deliver a deliver a functional copy of the α-L-iduronidase (IDUA) gene to the central nervous system, to correct its deficiency in MPS I patients.

Study participants will receive the investigatory treatment via a single infusion to the central nervous system while under anesthesia. Follow up will require lab testing every few weeks for 2 years, with visits less frequent in the second year.


  • Be 6years of age or older
  • Have documented evidence of early- stage neurocognitive deficit due to MPS I
  • Be able to receive immunosuppressive therapy in conjunction with the investigative therapy


  1. Screening to confirm acceptance to the study.
  2. Injection of the therapy, under anesthesia, guided by CT into the spinal fluid.
  3. Close observation immediately after the treatment.
  4. Follow up for 2 years with weekly, then monthly check-ins, spacing out to twice a year.

The trial will take place at the Children’s Hospital of Philadelphia, PA. Map.

Patient Advocacy  |  866-860-0117  |  [email protected]

Regenxbio Inc.


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