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Newsletter August 2012

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News: European Agency Recommends Gene Therapy Approval
European regulators have recommended approval of the Western world's first gene therapy drug - after rejecting it on three previous occasions - in a significant advance for the novel medical technology. More than 20 years since the first experiments with the ground-breaking method for fixing faulty genes, scientists and drug companies are still struggling to apply gene therapy in practice. Friday's decision by the European Medicines Agency (EMA) is a win for the drug's maker, the small Dutch biotech company uniQure, and a potential lifeline for patients with the ultra rare genetic disorder lipoprotein lipase deficiency (LPLD).

News: LinkedIn discussion by Carlos R. Camozzi
First gene therapy in Western world receives positive opinion in Europe from CHMP the 19th Jul 2012. uniQure’s gene therapy Glybera® is recommended for approval. The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP), supported by the Committee for Advance Therapies (CAT), has issued a positive opinion (19 July 2012) that recommends marketing authorization of Glybera® (alipogene tiparvovec) as a treatment for lipoprotein lipase deficiency (LPLD) under exceptional circumstances. LPLD is an ultra-rare inherited metabolic disease. Patients with this disorder are unable to handle fat particles (chylomicrons) in their blood, which leads to recurring severe abdominal pain and lifethreatening pancreatitis attacks. The first gene therapy in the Western world reached the most important regulatory approval milestone that recognises an outstanding progress after years of gene therapy research and development to treat a severe disease (LPL deficiency) with no alternative treatment. This CHMP positive opinion validates uniQure’s unique AAV-based gene therapy platform, consisting of a modular, plug-and-play vector system and unrivaled GMP manufacturing capabilities on a commercial scale. This innovative technology platform is ready to bring therapeutic solutions for many severe genetic and other disorders.

The positive recommendation from the CHMP for Glybera represents the major breakthrough for both LPLD patients and for medicine as a whole. uniQure is developing treatments for other rare diseases such as hemophilia, acute intermittent porphyria and Sanfilippo B. But the potential of gene therapy stretches far beyond rare diseases such as Parkinson’s. As part of the approval, treatment with Glybera will be offered through dedicated centers of excellence with expertise in treating LPLD and by specially trained doctors to ensure ongoing safety of this novel treatment paradigm. uniQure has also committed to building a patient registry for continued understanding of this devastating, under-researched disease. The Company is now preparing to apply for regulatory approval in the US, Canada, and other markets. In summary, this CHMP positive opinion is a historic event as it demonstrates that regulatory frame follows the pace of innovative research and development that brings safe and effective solutions to patients

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- 4 – 6 October 2012, International Society for Cell & Gene Therapy of Cancer - 2012 Singapore Conference, Singapore
- 26 – 29 October 2012, European Society Of Gene & Cell Therapy (ESGCT) XXth annual meeting and the French Society of Cell and Gene Therapy (SFTCG) collaborative Congress 2012, Versailles, France
- 19 – 21 November 2012, International Conference on Genetic Syndromes & Gene Therapy, San Antonio, USA
- 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,
- 17 - 19 April 2013, Annual conference of the British Society for Gene Therapy (BSGT), London, UK
- 15 – 18 May 2013, American Society of Gene and Cell Therapy (ASGCT) 16th Annual Meeting, Salt Lake City, Utah


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