LYS-SAF302 Gene Therapy | Type IIIA | Phase II-III | Lysogene

March 9, 2020
Page Last Updated Feb. 15, 2021

LYS-SAF302 is a gene therapy by Lysogene which is intended to deliver a working copy of the SGSH gene to deliver the gene therapy to the patient’s brain. The normal gene is placed inside the shell of a non-disease-causing virus (rhAAV10).

This is a phase 2-3 study to assess the efficacy in improving or stabilizing the neurodevelopmental state of MPS IIIA patients.

20 patients are being recruited. There are eight trial locations globally, including four in the United States. The others are in France, Germany, Netherlands, and the United Kingdom.

This trial is currently on clinical hold.

The gene therapy is directly injected into tissue of the brain. This is designed to be a one-time treatment.

Read this clinical trial’s information on, which includes enrollment criteria, trial locations, and study contact information.

Read Lysogene’s information about its gene therapy approach.

Recent press releases and articles regarding this clinical trial

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