Ongoing Phase 2/3 clinical trial for Sanfilippo syndrome Type A (MPS IIIA)

In October 2018, Lysogene, a company focused on pioneering gene therapies for CNS disorders, partnered with Sarepta Therapeutics to move forward on the AAVance MPS IIIA clinical trial. The Phase 2/3 gene therapy trial started in December 2018. AAVance is currently recruiting patients for the trial.

The trial will assess the efficacy of LYS-SAF302 in improving or stabilizing the neurodevelopmental status of MPS IIIA patients. LYS-SAF302 is an AAV-mediated gene therapy, the goal of which is to replace the faulty SGSH gene with a healthy copy of the gene. LYS-SAF302 employs the AAVrh10 virus, chosen for its ability to target the CNS. For more information, visit or (NCT03612869)