Mucopolysaccharidosis Type IIIA - Healing Genes

Mucopolysaccharidosis Type IIIA

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Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) (AAVance)

Open-label, Single-arm, Multi-center Study of Intracerebral Administration of Adeno-associated Viral (AAV) Serotype rh.10 Carrying Human N-sulfoglucosamine Sulfohydrolase (SGSH) cDNA for Treatment of Mucopolysaccharidosis Type IIIA

Phase 2/3

Researchers in TX, NY. MN, and CA seek patients with Mucopolysaccharidosis Type 3 A Sanfilippo Syndrome to trial an investigatory gene therapy, LYS-SAF302, a virus engineered to transfer a functional SGSH gene to the patient, so that their bodies can process heparan sulfate normally.

Participants will undergo a single intracerebral (into the brain, guided by MRI) infusion of the investigatory gene therapy, then be assessed at 6, 12, 18, and 24 months.


  • Be 6 months of age or older
  • Have confirmed diagnosis of MPS IIIA
  • Meet specific genetic screening criteria regarding their type of MPS mutation
  • Not have past use of SGSH enzyme replacement therapy for a period exceeding 3 months. If so, a period of at least 2 months without its use is required prior to screening.


  1. Screening to confirm eligibility.
  2. Participants receive a single infusion of the investigatory gene therapy, as well as a steroid prescription.
  3. Lab and physical assessments will continue 24 months at 6-month intervals.

The study sites include:
CHOC Children’s in Orange, CA. Map.
University of Minnesota in Minneapolis, MA. Map.
Weill Cornell Medical College in New York, NY. Map.
Baylor College of Medicine / Texas Children’s Hospital in Houston, TX. Map.
Lysogene trial inquiries  |  +33 (0) 1 41 43 03 90 (international)  |  [email protected]
Or go online:

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