This week in therapeutics




Licensing status

Publication and contact information

Endocrine/metabolic disease


N-sulfoglucosamine sulfohydrolase (SGSH; HNS)

Animal studies suggest SGSH gene therapy could help treat mucopolysaccharidosis IIIA (MPS IIIA). In mouse models for MPS IIIA and in normal dogs, intracerebral injection of an adeno-associated virus serotype 9 (AAV9) vector encoding murine or canine Sgsh increased Sgsh activity in the brain compared with injection of empty vector. In the mouse models, the gene therapy decreased behavioral deficits and disease symptoms in peripheral organs and increased locomotor function and survival compared with empty vector. Ongoing studies include testing intracerebral delivery of an AAV9 vector-based gene therapy in animal models for MPS IIIB.
Esteve S.A. has the AAV9 vector-based SGSH gene therapy in preclinical testing to treat MPS IIIA.
Lysogene's SAF-301, an intraparenchymally administered AAV10 vector encoding human SGSH and sulfatase modifying factor 1 (SUMF1), has completed Phase I/II testing to treat MPS IIIA.

SciBX 6(29); doi:10.1038/scibx.2013.759
Published online Aug. 1, 2013

Patented by the Autonomous University of Barcelona and Esteve; licensed to Esteve

Haurigot, V. et al. J. Clin. Invest.; published online July 1, 2013;
Contact: Fátima Bosch, Autonomous University of Barcelona, Bellaterra, Spain