ESTEVE and UAB Expand Their Research to Two New Gene Therapies for Sanfilippo B Syndrome and Hunter Syndrome

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BARCELONA, Spain, February 25, 2016 /PRNewswire/ --

ESTEVE strengthens 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.

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ESTEVE announced that the EMA and the U.S. FDA 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.

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. ESTEVE is currently initiating regulatory preclinical development of EGT-201. EGT-201 received orphan drug designation by the FDA and EMA in 2013.

"With the addition of EGT-201 and EGT-301 to create a strong pipeline of projects, ESTEVE reaffirms its commitment to developing gene therapies to treat severe and debilitating rare diseases. ESTEVE plans to expand its platform to other currently untreated disease conditions with the aim of bringing cures to underserved patients and becoming a reference in the field of gene therapy for rare diseases," said Dr. Carlos Plata, Chief Scientific Officer at ESTEVE.

"The Orphan Drug Designations obtained for all three projects underscore the excellence of the preclinical discovery capabilities at the UAB, while reinforcing the public-private partnership between the University and ESTEVE", said Fà tima Bosch, Director at the Center for Animal Biotechnology & Gene Therapy of UAB.

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 Type A and B and Hunter syndromes are lysosomal storage disease in which a given enzyme has lost its functional activity, leading to the accumulation of glycosaminoglycan substrates. This leads to a disruption in cellular function in multiple tissues and organs, amongst them the brain. The incidence the incidence is between 0.5 and 1 in 100,000 births. In all cases, quality of life may be significantly affected and in the severe cases, life expectancy is heavily reduced.

More information about Sanfilippo and Hunter syndrome:

http://www.esteve.es/EsteveFront/CargarPagina.do?pagina=idi_rd_portfolio_sanfilippo.jsp&div=idi&lng=en

SOURCE ESTEVE