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18/03/2014
The public-private partnership ESTEVE-UAB is bearing fruit
ESTEVE has announced the signing of two agreements that will enable it to progress the development of its gene therapeutic for the treatment of 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.
 
Sanfilippo A is a rare and devastating neurodegenerative disease that affects approximately 1 in every 100.000 children that rarely survive past adolescence.

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).

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.

Press release

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