ESGCT 2021 | LYS-SAF302 AAV gene therapy for MPS IIIA

Michael Hocquemiller, PhD, Lysogene, Paris, France, provides an overview of the ongoing Phase II/III AAVance (NCT03612869) study of LYS-SAF302 in patients with Mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo type A. LYS-SAF302 is an adeno-associated virus serotype rh.10 (AAVrh.10) vector carrying functional human N-sulfoglucosamine sulfohydrolase (SGSH) cDNA. This study aims to evaluate the efficacy in improving the neurodevelopmental state of MPS IIIA patients following one-time intracerebral administration of LYS-SAF302. Cerebrospinal fluid biomarker data obtained at one-year follow up supports the biological activity and therapeutic potential of LYS-SAF302. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.