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ESTEVE and U.A.B. Advance in Their Program to Develop a Cure for Sanfilippo A Syndrome

BARCELONA, Spain, March 11, 2014 /PRNewswire/ --

  • This gene therapy program is being developed in a public-private partnership between ESTEVE and the research team of Dr. Fàtima Bosch at the Center for Biotechnology and Gene Therapy of the Universitat Autònoma de Barcelona (UAB)
  • ESTEVE announce the signing of agreements with REGENX and GÉNÉTHON which will allow it to initiate its phase I/II clinical trial in 2015
  • Sanfilippo A is a rare and devastating neurodegenerative disease that affects approximately 1 in every 100.000 children that rarely survive past adolescence

ESTEVE has announced the signing of two agreements that will enable it to progress the development of its gene therapeutic for the treatment of Mucopolysaccharidosis type IIIA (MPSIIIA or Sanfilippo A Syndrome) and begin a phase I/II clinical trial in 2015. The agreements are with the North American biotechnology company REGENX Biosciences, LLC (REGENX) and with the French non for profit organization GÉNÉTHON.

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The license agreement with REGENX grants ESTEVE the right to use the adeno-associated viral vector, NAV rAAV9, in the development and commercialization of its investigational gene therapy for the treatment of Sanfilippo A Syndrome. The vector NAV rAAV9 is an integral part of the investigational therapeutic and enables the gene for the enzyme Sulfamidase, missing or defective in patients with Sanfilippo A Syndrome, to be delivered to and enter cells such as neurons and hepatocytes. Once inside the cells the gene expresses the Sulfamidase enzyme stably, compensating for its absence hence addressing the cause of the disease.

The agreement with GÉNÉTHON is for the development of the manufacturing process of the investigational gene therapeutic and its production for clinical trial use. The process to be developed will allow the production of the therapeutic for preclinical toxicology studies, the clinical trial and eventually for commercial use.

Public-private partnership ESTEVE-UAB

The Sanfilippo project was initiated by the research team of Dr. Fàtima Bosch at the Center for Biotechnology and Gene Therapy (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.

The investigational gene therapeutic consists of the viral NAV rAAV9, which contains a version of the gene that codes for Sulfamidase that has been optimized to improve its expression levels. Experimentation using preclinical disease models performed by the CBATEG have validated the potential efficacy of this therapeutic approach (see

At this time the project, which has been granted orphan status by the European Medicines Agency (EMA) and the US Food and Drug Agency (FDA), is in the preclinical development phase, with the manufacturing of early batches expected to begin shortly to support the required preclinical toxicology studies, which, once completed, will allow us to initiate the phase I/II clinical trial in 2015.

This project has received financial support from the Spanish Ministry of Health, Social Policy and Equality, and from the Spanish Ministry of Economy and Competitiveness.

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