Sanfilippo Syndrome type A (MPS IIIA) Gene Therapy Trial is Enrolling Participants

Lysogene is excited to announce the launch of its phase 2-3, single-arm trial to assess the efficacy of direct-to-CNS delivery of LYS-SAF302 in improving or stabilizing the neurodevelopmental status of MPS IIIA patients.

The search is on for MPS IIIA patients who fit the eligibility criteria for this trial, which is entitled “Multi-Center Study of AAVrh10-h.SGSH Gene Therapy in Patients with MPS IIIA (AAVance).” Details are available on under its study number NCT03612869, or contact: