Gene Therapy Slows Decline in Childhood Dementia

Lysogene’s investigational gene therapy LYS-SAF302 preserved cognitive function and brain volume in a small set of infants with a devastating type of childhood dementia, mucopolysaccharidosis Type IIIA (MPS IIIA).

Overall, the 12-patient phase 3 study didn’t achieve its primary endpoints of cognition, language development or motor development. But positive signals did appear in a sub analysis of six patients who were younger than 30 months when they received the treatment, the company said.

Instead of regarding it as a failed trial, Lysogene is concentrating on the positive results in children younger than 30 months.

LYS-SAF302 uses an adeno-associated viral carrier to deliver a functional copy of the gene that makes sulfamidase. The therapy is delivered directly into the brain via six intracerebral infusions.

The LYS-SAF302 program has received Fast Track, Rare Pediatric Disease and Orphan Drug designations in the U.S. and Orphan Medicinal Product designation in the EU.