The US Food and Drug Administration (FDA) has approved the first gene therapy for children with Sanfilippo syndrome type A, a rare inherited condition that causes progressive damage to the brain and nervous system.
Sanfilippo syndrome type A is caused by changes in the SGSH gene, which lead to a deficiency of the enzyme sulfamidase. This causes heparan sulphate to build up in cells, contributing to progressive loss of cognitive and developmental abilities.
The treatment, called Fayuvi, was developed by US pharmaceutical company Ultragenyx. Fayuvi is given as a single intravenous infusion and uses an adeno-associated virus 9 vector to deliver a working copy of the SGSH gene to cells.
Pharmaceutical Technology has more information on the FDA approval.