Gene Therapy on the mend as treatment gets western approval

gene_therapyEurope’s Committee for Medicinal Products for Human Use approved a gene therapy for a rare genetic disease, the first time a Western regulatory agency has okayed such a treatment, though gene therapies have been approved in China.

The virus-mediated treatment, called Glybera, which is being developed by Amsterdam-based UniQure, introduces a normal version of a gene needed to properly break down fats in the blood. Though the condition is rare, patients with dysfunctional copies of this gene have dramatically increased levels of fat in their blood, which can lead to fatal inflammation of the pancreas. The genetic repair lowers blood fat concentration and reduces the frequency of pancreatitis, according to clinical trial data.

Gene therapy hit a major milestone in 2012 when Glybera was approved by European authorities for the treatment of autosomal recessive lipoprotein lipase (LPL) deficiency. Gene therapy has been a promise in the making for decades, and as such, hopes are high for Glybera. Two other drugs had been approved several years ago in China as gene therapies for cancer, but with only very limited clinical success. We also had an early look at ongoing work to genetically reprogram cardiac cells to form a natural pacemaker. Meanwhile, genetic sequencing costs keep plummeting, and Life Sciences is confident that with its Ion Proton DNA sequencer it will be able to sequence so fast and cheap that it was the first to enter X-Prize 100 over 100 $1000 Genomes Challenge.

The European Medicines Agency took gene therapy a big step closer to normalization this week. The Agency reported on its website that it was recommending approval of the controversial techniques for the treatment of a very rare genetic disorder.

Providing the European Commission take on board this recommendation, and they have always listened to a medical body before, then it will make the first ever regulatory approval of a gene therapy medication, in the West. (A gene therapy medication to treat cancer has approval in China since ‘03). This will mean a metaphorical shot in the arm for a field of study that has for too long labored with too little support and funds.

The basic concept of gene therapy—replacing a defective gene or adding in a functional copy— is straightforward, but the trick is in the delivery. Gene therapies are often carried into a patient’s cells by viruses. Some of the early problems with the therapies were due to strong immune reaction to the transport viruses or cancer sparked by genomic changes induced by the viruses. But in recent years, researchers have found safer viruses and techniques for getting the replacement genes into a patient’s body.

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About thomasdinnocenzi

Thomas D'Innocenzi is a highly accomplished, results-focused senior international executive with extensive experience in global sourcing and market development worldwide to meet evolving business needs. Tom has proven ability in implementing and managing profitable global sourcing operations worldwide. Extensive experience in international market development operations to accommodate rapid growth. Skilled in building top-performing teams, benchmarking performance, and restricting organizations to improve efficiency, productivity, and profitability. Experienced transition leader and change agent. As principal of Nova Advisors, LLC I’ve assembled an exemplary team that brings with them the knowledge and experience gained from starting up a Global Sourcing program with multiple Fortune 500 companies as well as the largest supplier network throughout the Asia-Pacific region. We have experience and expertise in more than a thousand medical and pharmaceutical products in manufacturing and sourcing at the best value. The right product, the right price point and the right branding fueled these successes that resulted in double-digit growth for top line sales and bottom line net margins for our customers. What sets us apart: • Our reach includes a large network of suppliers & manufacturers spanning 13 countries in Asia-Pacific region • We understand the manufacturing process and the business of the supplier and the buyer • Our company culture is based on quality assurance and our process is based on local quality control Our commitment is to be your partner offering the best products and services at the lowest cost. Contact me to discuss how we can make the global marketplace work for you. thomas@novaadvisors.com In addition, I am open to discussing opportunities in global sourcing, international marketing & sales, logistics and medical/pharma in Thailand, Vietnam, Malaysia, Philippines & Japan. Aside from my work I enjoy piano, astronomy, physics, and assisting my daughters with their studies. SPECIALTIES: Global Sourcing, Supply Chain Management, Business Development, Marketing, Logistics, Global Networking, Market Development, Healthcare Solutions, Pharmaceuticals, Medical Devices, Technology, Asia, Southeast Asia, US and Canada
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