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Newsletter November 2012

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News: First Gene Therapy Approved in Europe
Amsterdam, The Netherlands – November 2, 2012 – uniQure announced it has received approval from the European Commission for the gene therapy Glybera® (alipogene tiparvovec), a treatment for patients with lipoprotein lipase deficiency (LPLD, also called familial hyperchylomicronemia) suffering from recurring acute pancreatitis. Patients with LPLD, a very rare, inherited disease, are unable to metabolize the fat particles carried in their blood, which leads to inflammation of the pancreas (pancreatitis), an extremely serious, painful, and potentially lethal condition. The approval makes Glybera the first gene therapy approved by regulatory authorities in the Western world.

“Glybera’s approval means LPLD patients, for the first time, have a medical treatment option for a very complex and severe disease,” said Professor John Kastelein of the Department of Vascular Medicine at the Academic Medical Center of the University of Amsterdam, the Netherlands. “LPLD leads to acute and recurrent pancreatitis attacks, and in many patients causes early onset diabetes and cardiovascular complications. This therapy will have a dramatic impact on the lives of these patients. Currently their only recourse is to severely restrict the amount of fat they consume. By helping to normalize the metabolism of fat, Glybera prevents inflammation of the pancreas thereby averting the associated pain and suffering and, if administered early enough, the associated co-morbidities.”

As part of the approval, patients will receive treatment with Glybera through dedicated centers of excellence and by specially trained doctors. uniQure will also build a patient registry to further improve the understanding of this devastating, under-researched disease and the effects of Glybera treatment. Marketing Authorisation covers all 27 European Union member states. uniQure is preparing to apply for regulatory approval in the US, Canada, and other markets.

“The final approval of Glybera from the EC marks a major step forward in making gene therapies available not only for LPLD but also for a large number of rare diseases with a very high unmet medical need,” says Jörn Aldag, CEO of uniQure. “The EC’s approval is an important validation of our innovative product platform and offers strong support for our other advanced development programs, which focus on acute intermittent porphyria, Sanfilippo B, hemophilia B and Parkinson’s disease.”

uniQure has developed Glybera as a therapy for patients with the genetic disorder lipoprotein lipase deficiency, an orphan disease for which no treatment existed. The disease is caused by mutations in the LPL gene, resulting in highly decreased or absent activity of LPL enzyme in patients. This enzyme is needed in order to break down large fat-carrying particles that circulate in the blood after each meal. When such particles, called chylomicrons, accumulate in the blood, they may obstruct small blood vessels. Excess chylomicrons result in recurrent and severe acute inflammation of the pancreas, called pancreatitis, the most debilitating complication of LPLD. Glybera has orphan drug designation in the EU and US. LPL Deficiency affects 1-2 persons per million.

Glybera has been tested in three interventional clinical studies conducted in the Netherlands and in Canada, in which a total of 27 LPLD patients participated. In all three clinical trials, Glybera was well tolerated, with no relevant safety issues observed. Data from these clinical trials indicate that a single dose administration of Glybera resulted in a long-term biological activity of the LPL protein. For further information on LPLD visit www.lpldeficiency.com.

Lipoprotein lipase is a key ‘first step’ enzyme in the metabolism of lipoproteins following fat intake with diet. In clinical studies a transient reduction in triglycerides for up to 12 weeks in individual patients could be observed. Furthermore, Glybera allows expression of the LPL protein in injected muscle which is reflected by the improvement of postprandial chylomicron (CM) metabolism observed in a small subset of patients. Glybera (alipogene tiparvovec) contains the human lipoprotein lipase (LPL) gene variant LPLS447X in a vector. The vector comprises a protein shell derived from adeno-associated virus serotype 1 (AAV1), the promoter, a posttranscriptional regulatory element and AAV2 derived inverted terminal repeats.

Glybera is indicated for adult patients diagnosed with familial lipoprotein lipase deficiency (LPLD) and suffering from severe or multiple pancreatitis attacks despite dietary fat restrictions. The diagnosis of LPLD has to be confirmed by genetic testing. The indication is restricted to patients with detectable levels of LPL protein.

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Poll: Future Gene Therapy Approvals
The market approval of gene therapy product Glybera in Europe will accelerate regulatory approvals of other gene medicines. What do you think? Vote now!
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2- No, regulatory process is still a big hurdle


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Conferences
- 19 – 21 November 2012, International Conference on Genetic Syndromes & Gene Therapy, San Antonio, USA
- 23 November 2012, 6th Annual Meeting of Korean Society of Gene and Cell Therapy, Seoul, Korea
- 5 - 7 December 2012, DNA Vaccines 2012, San Diego, LA
- 28 - 30 January 2013, Phacilitate 9th Annual Cell & Gene Therapy Forum 2013, Washington DC, MD
- 13 - 15 February 2013, Gene Transfer Technologies into Multicellular Eukaryotes, Vienna, Austria
- 28 February - 2 March 2013, 19th Annual Meeting German Society for Gene Therapy (DG-GT e.V.), Munich, Germany
- 15 - 16 March 2013, Spring Symposium of the Netherlands Society of Gene & Cell Therapy (NVGCT), Lunteren, The Netherlands
- 17 - 19 April 2013, Annual conference of the British Society for Gene Therapy (BSGT), London, UK
- 22 - 25 April 2013, International Society for Cellular Therapy (ISCT) 19th Annual Meeting, Auckland, New Zealand
- 8 – 10 May 2013, 8th Australasian Gene Therapy Society Meeting (AGTS), Sydney, Australia
- 15 – 18 May 2013, American Society of Gene and Cell Therapy (ASGCT) 16th Annual Meeting, Salt Lake City, Utah
- 4 – 6 July 2013, The 19th Annual Meeting of the Japan Society of Gene Therapy (JSGT), Okayama, Japan
- 24 – 28 October 2013, VII Congress of the Spanish Society of Gene (SETGyC) and Cell Therapy, and XXI Congress of the European Society of Gene and Cell Therapy (ESGCT), Madrid, Spain

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