Date: 2016-02-25
Type of information: R&D agreement
Compound: EGT-201, EGT-301
Company: Esteve (Spain) Universitat Autònoma de Barcelona (Spain)
Therapeutic area: Rare diseases - Genetic diseases
Type agreement: R&D
Action mechanism: gene therapy. 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. EGT-201 consists of an AAV9 vector containing the alpha-N-acetyglucosaminidase (NAGLU) transgene and it aims to restore NAGLU functional deficiency in patients with Sanfilippo B syndrome.
Disease: mucopolysaccharidosis type II (Hunter's syndrome), mucopolysaccharidosis type IIIB (Sanfilippo B syndrome)
Details: * On February 25, 2016, Esteve announced that the company has strengthened 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, to create a promising gene therapy platform. 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. The company 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. The European Medicines Agency (EMA) and the FDA have also granted Orphan Drug Designation to EGT-301 for the treatment of Hunter syndrome. Esteve is currently initiating regulatory preclinical development of EGT-301. These projects have received financial support from the Spanish Ministry of Health, Social Policy and Equality, and from the Spanish Ministry of Economy and Competitiveness. Sanfilippo Project has also received financial support from European Union FEDER funds. The gene therapy platform was initiated by the research team of Professor Fàtima Bosch at the CBATEG of UAB and since 2009 is being developed within the framework of a public-private partnership between ESTEVE and the University. In this partnership, ESTEVE leads all activities associated with the management and protection of intellectual property, regulatory activities, the coordination and supervision of GMP manufacturing, the preclinical toxicology studies as well as all clinical development. The CBATEG research team at the UAB brings to the partnership their scientific know-how and expertise in gene therapy including viral vector design and the development of preclinical disease models. CBATEG/UAB and ESTEVE have recently strengthened their collaboration by signing a long-term contract to pursue the discovery and development of gene therapies for other rare diseases.
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