Welcome!

News Feed Item

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.

     (Photo: http://photos.prnewswire.com/prnh/20140227/672149 )

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 http://www.esteve.es/EsteveFront/PressRoom.do?op=DN&div=idi&con=3426&lng=en)

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.

Watch video

https://www.youtube.com/watch?v=iSwT53qofKM

More Stories By PR Newswire

Copyright © 2007 PR Newswire. All rights reserved. Republication or redistribution of PRNewswire content is expressly prohibited without the prior written consent of PRNewswire. PRNewswire shall not be liable for any errors or delays in the content, or for any actions taken in reliance thereon.

Latest Stories
SYS-CON Events announced today that Juniper Networks (NYSE: JNPR), an industry leader in automated, scalable and secure networks, will exhibit at SYS-CON's 20th International Cloud Expo®, which will take place on June 6-8, 2017, at the Javits Center in New York City, NY. Juniper Networks challenges the status quo with products, solutions and services that transform the economics of networking. The company co-innovates with customers and partners to deliver automated, scalable and secure network...
Cognitive Computing is becoming the foundation for a new generation of solutions that have the potential to transform business. Unlike traditional approaches to building solutions, a cognitive computing approach allows the data to help determine the way applications are designed. This contrasts with conventional software development that begins with defining logic based on the current way a business operates. In her session at 18th Cloud Expo, Judith S. Hurwitz, President and CEO of Hurwitz & ...
Everyone wants to use containers, but monitoring containers is hard. New ephemeral architecture introduces new challenges in how monitoring tools need to monitor and visualize containers, so your team can make sense of everything. In his session at @DevOpsSummit, David Gildeh, co-founder and CEO of Outlyer, will go through the challenges and show there is light at the end of the tunnel if you use the right tools and understand what you need to be monitoring to successfully use containers in your...
The explosion of new web/cloud/IoT-based applications and the data they generate are transforming our world right before our eyes. In this rush to adopt these new technologies, organizations are often ignoring fundamental questions concerning who owns the data and failing to ask for permission to conduct invasive surveillance of their customers. Organizations that are not transparent about how their systems gather data telemetry without offering shared data ownership risk product rejection, regu...
@GonzalezCarmen has been ranked the Number One Influencer and @ThingsExpo has been named the Number One Brand in the “M2M 2016: Top 100 Influencers and Brands” by Analytic. Onalytica analyzed tweets over the last 6 months mentioning the keywords M2M OR “Machine to Machine.” They then identified the top 100 most influential brands and individuals leading the discussion on Twitter.
Grape Up is a software company, specialized in cloud native application development and professional services related to Cloud Foundry PaaS. With five expert teams that operate in various sectors of the market across the USA and Europe, we work with a variety of customers from emerging startups to Fortune 1000 companies.
Financial Technology has become a topic of intense interest throughout the cloud developer and enterprise IT communities. Accordingly, attendees at the upcoming 20th Cloud Expo at the Javits Center in New York, June 6-8, 2017, will find fresh new content in a new track called FinTech.
New competitors, disruptive technologies, and growing expectations are pushing every business to both adopt and deliver new digital services. This ‘Digital Transformation’ demands rapid delivery and continuous iteration of new competitive services via multiple channels, which in turn demands new service delivery techniques – including DevOps. In this power panel at @DevOpsSummit 20th Cloud Expo, moderated by DevOps Conference Co-Chair Andi Mann, panelists will examine how DevOps helps to meet th...
With major technology companies and startups seriously embracing Cloud strategies, now is the perfect time to attend @CloudExpo | @ThingsExpo, June 6-8, 2017, at the Javits Center in New York City, NY and October 31 - November 2, 2017, Santa Clara Convention Center, CA. Learn what is going on, contribute to the discussions, and ensure that your enterprise is on the right path to Digital Transformation.
SYS-CON Events announced today that Interoute, owner-operator of one of Europe's largest networks and a global cloud services platform, has been named “Bronze Sponsor” of SYS-CON's 20th Cloud Expo, which will take place on June 6-8, 2017 at the Javits Center in New York, New York. Interoute is the owner-operator of one of Europe's largest networks and a global cloud services platform which encompasses 12 data centers, 14 virtual data centers and 31 colocation centers, with connections to 195 add...
@DevOpsSummit at Cloud taking place June 6-8, 2017, at Javits Center, New York City, is co-located with the 20th International Cloud Expo and will feature technical sessions from a rock star conference faculty and the leading industry players in the world. The widespread success of cloud computing is driving the DevOps revolution in enterprise IT. Now as never before, development teams must communicate and collaborate in a dynamic, 24/7/365 environment. There is no time to wait for long developm...
SYS-CON Events announced today that Hitachi, the leading provider the Internet of Things and Digital Transformation, will exhibit at SYS-CON's 20th International Cloud Expo®, which will take place on June 6-8, 2017, at the Javits Center in New York City, NY. Hitachi Data Systems, a wholly owned subsidiary of Hitachi, Ltd., offers an integrated portfolio of services and solutions that enable digital transformation through enhanced data management, governance, mobility and analytics. We help globa...
The age of Digital Disruption is evolving into the next era – Digital Cohesion, an age in which applications securely self-assemble and deliver predictive services that continuously adapt to user behavior. Information from devices, sensors and applications around us will drive services seamlessly across mobile and fixed devices/infrastructure. This evolution is happening now in software defined services and secure networking. Four key drivers – Performance, Economics, Interoperability and Trust ...
Blockchain is a shared, secure record of exchange that establishes trust, accountability and transparency across supply chain networks. Supported by the Linux Foundation's open source, open-standards based Hyperledger Project, Blockchain has the potential to improve regulatory compliance, reduce cost and time for product recall as well as advance trade. Are you curious about Blockchain and how it can provide you with new opportunities for innovation and growth? In her session at 20th Cloud Exp...
Five years ago development was seen as a dead-end career, now it’s anything but – with an explosion in mobile and IoT initiatives increasing the demand for skilled engineers. But apart from having a ready supply of great coders, what constitutes true ‘DevOps Royalty’? It’ll be the ability to craft resilient architectures, supportability, security everywhere across the software lifecycle. In his keynote at @DevOpsSummit at 20th Cloud Expo, Jeffrey Scheaffer, GM and SVP, Continuous Delivery Busin...