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Pediatric Gene Therapy

08/02/2017 by Andrea

The European approvals of Glybera® in 2012 and Strimvelis® in 2016 seem likely to be the vanguard of the therapeutic revolution heralded by the decoding of the human genome. Both of these treatments originated from academic centers of excellence and became the subjects of agreements with pharmaceutical companies (Uniqure/Chiesi and GSK, respectively) with the experience to navigate the manufacturing infrastructure and regulatory approvals processes that led to the products’ commercialization. The EMA has indicated the level of follow-up it expects to see for these patients, and the FDA’s perspective will likely be revealed with the first US approvals of gene therapies anticipated in 2017. The pharmaceutical industry has been responsible for the successful execution of hundreds of thousands of trials of new drugs and vaccines. Gene therapy trials are unique, combining established experience and new approaches previously
unnecessary for developing new drugs.

 

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Filed Under: Clinical Channel White Papers, Pharma Channel White Papers, R&D Channel White Papers

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