The Food and Drug Administration (FDA) has granted Fast Track designation to LYS-SAF302 (Lysogene), an investigational gene therapy for the treatment of mucopolysaccharidosis Type IIIA (MPS IIIA).

MPS IIIA, also known as Sanfilippo syndrome type A, is a rare autosomal recessive genetic disease caused by mutations in the SGSH (N-sulfoglucosamine sulfohydrolase) gene. The disorder usually begins during early childhood and can result in progressive dementia, aggressive behavior, hyperactivity, seizures, deafness, and loss of vision.

LYS-SAF302 works by delivering a functional copy of the SGSH gene through an adeno-associated vector directly to the CNS in one neurosurgical procedure. The Company believes the replacement of the SGSH gene will reverse the phenotypic abnormalities of CNS neural cells.

A phase 2/3 trial of LYS-SAF302 is underway to assess the efficacy of the gene therapy in improving or stabilizing the neurodevelopmental state of patients with MPS IIIA. At this time, the Company reports that 17 of the 20 enrolled patients have been treated with the investigational therapy. The primary outcome measure of the study is the change from baseline in development quotient, compared to regression reported in natural history studies, at 24 months.

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