Meet Shoukhrat Mitalipov He's using breakthrough gene therapy techniques to cure disease. Learn more at http://ohsu.info/LNCf. By: OHSUvideo … Continue reading →

The western worlds first gene therapy drug is expected to go on sale in Germany next year. Photograph: Eliseo Fernandez/Reuters The western worlds first gene therapy drug is set to go on sale in Germany, with a price tag that could amount to an 870,000 cost to treat a single patient. Glybera, a treatment for the rare genetic condition lipoprotein lipase deficiency (LPLD), which clogs the blood with fat, has been developed by Dutch biotech firm UniQure and Italian marketing marketing partner Chiesi. It is undergoing an assessment of benefits by Germanys federal joint committee, which will report by April 2015. But the company is seeking a retail price of 53,000 (42,000) per phial, which equates to 1.1m (870,000) for a course of treatment for a typical LPLD patient. This price will be subject to a discount under Germanys drug pricing system. A Chiesi spokeswoman confirmed the launch price and added that a final figure would be set after the German authorities gave their verdict and negotiations are held with health insurance funds. First commercial treatments are expected in the first half of 2015, she said. UniQure, which will get a net royalty of between 23% and 30% on sales, … Continue reading →

London - The Western world's first gene therapy drug is set to go on sale in Germany with a 1.1m ($1.4m) price tag, a new record for a medicine to treat a rare disease. The sky-high cost of Glybera, from Dutch biotech firm UniQure and its unlisted Italian marketing partner Chiesi, shows how single curative therapies to fix faulty genes may upend the conventional pharmaceutical business model. After a quarter century of experiments and several setbacks, gene therapy is finally throwing a life-line to patients by inserting corrective genes into malfunctioning cells - but paying for it poses a challenge. The new drug fights an ultra-rare genetic disease called lipoprotein lipase deficiency (LPLD) that clogs the blood with fat. The medicine was approved in Europe two years ago but its launch was delayed to allow for the collection of six-year follow-up data on its benefits. Now Chiesi has filed a pricing dossier with Germany's Federal Joint Committee, or G-BA, which will issue an assessment of the drug's benefits by the end of April 2015. The company is seeking a retail price of 53 000 per vial, or 43 870 ex-factory. That equates to 1.11m for an typical LPLD patient, averaging … Continue reading →