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.

EGT-101, the lead project in Esteve’s gene therapy platform, consists of an AAV9 vector containing the human sulfamidase (SGSH) transgene and it aims to restore SGSH functional deficiency in patients with Sanfilippo A syndrome. Esteve plans to initiate Phase I/II clinical trials for EGT-101 by the end of 2016. EGT-101 received orphan drug designation by the FDA and EMA in 2011.