Esteve and UAB expand research to two new gene therapies for Sanfilippo B and Hunter syndromes

Privately held Spanish drugmaker Laboratorios Esteve is strengthening its gene therapy platform with the addition of two new investigational gene therapies, EGT-201 for the treatment of Sanfilippo B syndrome and EGT-301 for the treatment of Hunter syndrome, both developed in collaboration with the group of Professor Fàtima Bosch at the Universitat Autònoma de Barcelona (UAB). EGT-201 and EGT-301 join EGT-101, designed to treat Sanfilippo A syndrome.

Esteve says that the European Medicines Agency and the US Food and Drug Administration have granted Orphan drug designation to its novel gene therapy program EGT-301 for the treatment of Hunter syndrome or mucopolysaccharidosis II (MPS II). EGT-301 consists of an adeno-associated viral vector of serotype 9 (AAV9) containing the human Iduronate-2-sulfatase (I2S) transgene designed to restore I2S functional deficiency in patients with Hunter syndrome. ESTEVE is currently initiating regulatory preclinical development of EGT-301.