|By PR Newswire||
|December 11, 2012 02:01 AM EST||
AMSTERDAM, December 11, 2012 /PRNewswire/ --
uniQure B.V., a leader in the field of human gene therapy, today announced the start of its Phase I clinical trial in acute intermittent porphyria (AIP) with the treatment of the first patient. The study is conducted under the aegis of the AIPGENE consortium, a pan-European collaboration funded in part by the European Commission's Seventh Framework Program with the aim to develop a gene therapy for the treatment of AIP, a rare and devastating disease caused by mutations in the porphobilinogen deaminase gene (PBGD). AIP can be life-threatening and the long-term effects include irreversible nerve damage, liver cancer and kidney failure. uniQure was granted orphan drug designation for the treatment of AIP in 2009 from the European Medicines Agency.
"The start of the AIP Phase I study marks the first of four programs that will enter clinical trials over the next 12 months," says Jörn Aldag, CEO of uniQure. "After AIP we expect clinical trials to be initiated in Parkinson's disease, hemophilia B, and Sanfilippo B. After many years of building and developing our capabilities and competencies, and the approval in November of Glybera for LPLD as the first gene therapy in the Western world, we are highly motivated to expedite the clinical development of our other advanced gene therapies."
About the AIP Phase I study
The Phase I will enroll eight patients with severe AIP at two centers: the Clinical University of Navarra, Pamplona, Spain, and the 12 de Octubre University Hospital,, Madrid, Spain. The study's primary objective is the assessment of safety and determination of the maximum tolerated dose. Secondary objectives include tolerability of treatment, pharmacokinetics, changes in the levels of surrogate markers of activity including porphobilinogen (PBG) and delta-aminolevulinic acid (ALA), and assessment of symptom control, neuro-psychological changes and quality of life. All patients will be followed for one year, and the interim results of the Phase I are expected in Q3 2013.
About acute intermittent porphyria
Acute Intermittent porphyria (AIP) is a rare genetic disease which is caused by mutations in the porphobilinogen deaminase (PBGD) gene; one of the enzymes of the heme biosynthesis pathway. Mutations in this gene cause insufficient activity of the protein resulting in partially disruption of heme synthesis. This in turn leads to accumulation of toxic intermediates (ALA and PBG) giving rise to a wide variety of problems including acute, severe abdominal pains, psychiatric and neurological disorders, and muscular weakness. Acute porphyric attacks can be life-threatening and the long-term consequences include irreversible nerve damage, liver cancer and kidney failure. Currently, the only curative therapy is liver transplantation and thus, new curative options are urgently needed. Severe AIP patients are suffering poor quality of life with palliative treatments for the different symptoms including glucose or heme infusions for metabolic replacement and inhibition of toxic metabolic production.
AIPGENE is a European Commission Framework Programme 7-funded consortium (Grant Agreement number 261506) which was put together with the aim to develop the orphan gene therapy drug AAV5-AAT-PBGD (AMT-021) for the treatment of Acute Intermittent porphyria (AIP). The consortium's objective is to contribute to alleviating the negative impact of this disease on the quality of life of the patients and their families. Overall coordinator of the project is the Centre for Applied Medical Research (CIMA) at the University of Navarra, Pamplona, Spain. Apart from uniQure, other members of the consortium are the Clinical University of Navarra, Pamplona, Spain; Karolinska University Hospital, Stockholm, Sweden; German Cancer Research Center (NCT-DKFZ), Heidelberg, Germany; DIGNA Biotech, Pamplona, Spain; Servicio Madrileno de Salud, Madrid, Spain.
uniQure is a world leader in the development of human gene based therapies. uniQure's Glybera, a gene therapy for the treatment of lipoprotein lipase deficiency has been approved in the European Union, and is the first approved gene therapy in the Western world. uniQure's product pipeline of gene therapy products in development comprise hemophilia B, acute intermittent porphyria, Parkinson's disease and Sanfilippo B. Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate probably the world's first stable and scalable AAV manufacturing platform. This proprietary platform can be applied to a large number of rare (orphan) diseases caused by one faulty gene, and allows uniQure to pursue its strategy of focusing on this sector of the industry. uniQure's largest shareholders are Forbion Capital Partners and Gilde Healthcare, two of the leading life sciences venture capital firms in the Netherlands. Further information can be found at http://www.uniqure.com.
SOURCE uniQure B.V.
The 17th International Cloud Expo has announced that its Call for Papers is open. 17th International Cloud Expo, to be held November 3-5, 2015, at the Santa Clara Convention Center in Santa Clara, CA, brings together Cloud Computing, APM, APIs, Microservices, Security, Big Data, Internet of Things, DevOps and WebRTC to one location. With cloud computing driving a higher percentage of enterprise IT budgets every year, it becomes increasingly important to plant your flag in this fast-expanding bu...
Jul. 7, 2015 10:30 AM EDT Reads: 1,845
SYS-CON Events announced today that Dyn, the worldwide leader in Internet Performance, will exhibit at SYS-CON's 17th International Cloud Expo®, which will take place on November 3-5, 2015, at the Santa Clara Convention Center in Santa Clara, CA. Dyn is a cloud-based Internet Performance company. Dyn helps companies monitor, control, and optimize online infrastructure for an exceptional end-user experience. Through a world-class network and unrivaled, objective intelligence into Internet condit...
Jul. 7, 2015 10:30 AM EDT Reads: 2,177
IT data is typically silo'd by the various tools in place. Unifying all the log, metric and event data in one analytics platform stops finger pointing and provides the end-to-end correlation. Logs, metrics and custom event data can be joined to tell the holistic story of your software and operations. For example, users can correlate code deploys to system performance to application error codes. In his session at DevOps Summit, Michael Demmer, VP of Engineering at Jut, will discuss how this can...
Jul. 7, 2015 10:30 AM EDT Reads: 1,543
In their general session at 16th Cloud Expo, Michael Piccininni, Global Account Manager - Cloud SP at EMC Corporation, and Mike Dietze, Regional Director at Windstream Hosted Solutions, reviewed next generation cloud services, including the Windstream-EMC Tier Storage solutions, and discussed how to increase efficiencies, improve service delivery and enhance corporate cloud solution development. Michael Piccininni is Global Account Manager – Cloud SP at EMC Corporation. He has been engaged in t...
Jul. 7, 2015 10:15 AM EDT Reads: 2,372
"What Dyn is able to do with our Internet performance and our Internet intelligence is give companies visibility into what is actually going on in that cloud," noted Corey Hamilton, Product Marketing Manager at Dyn, in this SYS-CON.tv interview at 16th Cloud Expo, held June 9-11, 2015, at the Javits Center in New York City.
Jul. 7, 2015 10:00 AM EDT Reads: 1,501
In his General Session at 16th Cloud Expo, David Shacochis, host of The Hybrid IT Files podcast and Vice President at CenturyLink, investigated three key trends of the “gigabit economy" though the story of a Fortune 500 communications company in transformation. Narrating how multi-modal hybrid IT, service automation, and agile delivery all intersect, he will cover the role of storytelling and empathy in achieving strategic alignment between the enterprise and its information technology.
Jul. 7, 2015 10:00 AM EDT Reads: 2,419
Overgrown applications have given way to modular applications, driven by the need to break larger problems into smaller problems. Similarly large monolithic development processes have been forced to be broken into smaller agile development cycles. Looking at trends in software development, microservices architectures meet the same demands. Additional benefits of microservices architectures are compartmentalization and a limited impact of service failure versus a complete software malfunction. Th...
Jul. 7, 2015 10:00 AM EDT Reads: 1,734
SYS-CON Events announced today that JFrog, maker of Artifactory, the popular Binary Repository Manager, will exhibit at SYS-CON's @DevOpsSummit Silicon Valley, which will take place on November 3–5, 2015, at the Santa Clara Convention Center in Santa Clara, CA. Based in California, Israel and France, founded by longtime field-experts, JFrog, creator of Artifactory and Bintray, has provided the market with the first Binary Repository solution and a software distribution social platform.
Jul. 7, 2015 10:00 AM EDT Reads: 1,888
SYS-CON Events announced today that CommVault has been named “Bronze Sponsor” of SYS-CON's 17th International Cloud Expo®, which will take place on November 3–5, 2015, at the Santa Clara Convention Center in Santa Clara, CA. A singular vision – a belief in a better way to address current and future data management needs – guides CommVault in the development of Singular Information Management® solutions for high-performance data protection, universal availability and simplified management of data...
Jul. 7, 2015 09:45 AM EDT Reads: 2,175
SYS-CON Events announced today that Cloudian, Inc., the leading provider of hybrid cloud storage solutions, will exhibit at SYS-CON's 17th International Cloud Expo®, which will take place on November 3–5, 2015, at the Santa Clara Convention Center in Santa Clara, CA. Cloudian, Inc., is a Foster City, California - based software company specializing in cloud storage software. The main product is Cloudian, an Amazon S3-compliant cloud object storage platform, the bedrock of cloud computing system...
Jul. 7, 2015 09:30 AM EDT Reads: 2,127
"We help to transform an organization and their operations and make them more efficient, more agile, and more nimble to move into the cloud or to move between cloud providers and create an agnostic tool set," noted Jeremy Steinert, DevOps Services Practice Lead at WSM International, in this SYS-CON.tv interview at @DevOpsSummit, held June 9-11, 2015, at the Javits Center in New York City.
Jul. 7, 2015 09:30 AM EDT Reads: 1,573
SYS-CON Events announced today that Secure Infrastructure & Services will exhibit at SYS-CON's 17th International Cloud Expo®, which will take place on November 3–5, 2015, at the Santa Clara Convention Center in Santa Clara, CA. Secure Infrastructure & Services (SIAS) is a managed services provider of cloud computing solutions for the IBM Power Systems market. The company helps mid-market firms built on IBM hardware platforms to deploy new levels of reliable and cost-effective computing and hig...
Jul. 7, 2015 09:00 AM EDT Reads: 1,973
"We have a tagline - "Power in the API Economy." What that means is everything that is built in applications and connected applications is done through APIs," explained Roberto Medrano, Executive Vice President at Akana, in this SYS-CON.tv interview at 16th Cloud Expo, held June 9-11, 2015, at the Javits Center in New York City.
Jul. 7, 2015 09:00 AM EDT Reads: 1,887
In his session at 16th Cloud Expo, Simone Brunozzi, VP and Chief Technologist of Cloud Services at VMware, reviewed the changes that the cloud computing industry has gone through over the last five years and shared insights into what the next five will bring. He also chronicled the challenges enterprise companies are facing as they move to the public cloud. He delved into the "Hybrid Cloud" space and explained why every CIO should consider ‘hybrid cloud' as part of their future strategy to achie...
Jul. 7, 2015 09:00 AM EDT Reads: 1,891
Internet of Things is moving from being a hype to a reality. Experts estimate that internet connected cars will grow to 152 million, while over 100 million internet connected wireless light bulbs and lamps will be operational by 2020. These and many other intriguing statistics highlight the importance of Internet powered devices and how market penetration is going to multiply many times over in the next few years.
Jul. 7, 2015 09:00 AM EDT Reads: 2,556