Sentences with phrase «first gene therapy product»

Not exact matches

China was the first country to approve a gene therapy product for commercial use, in 2004.

The product, dubbed tisagenlecleucel (pronounced tis - a-gen-LEK-loo-sell), is the first gene therapy of any kind to be approved in the United States.

China has made crucial breakthroughs in the field, including creating the world's first commercialized gene therapy product, Gendicine.

Under his leadership from 2009 - 2015, uniQure received the first - ever approval of a gene therapy product by the European Medicines Agency, built a pipeline of gene therapy products across several disease areas, obtained approximately $ 200M through its NASDAQ - listing and follow - on, and closed a multi-billion dollar collaboration in cardiovascular gene therapy.

Endari, the first new treatment for patients with sickle cell disease in almost 20 years, Genentech's Hemlibra, the first - ever non-blood product to treat patients with hemophilia A with inhibitors, Actemra, the first treatment for adults diagnosed with giant cell arteritis, BioMarin's Brineura, the first treatment for a form of Batten disease, Benznidazole, the first U.S. treatment for Chagas disease, Novartis» Kymriah to treat certain children and young adults with B - cell acute lymphoblastic leukemia, which is also the first gene therapy to become available in the United States, are some of the drugs that received the FDA's stamp of approval in 2017.

Aiuti A, Roncarolo MG, Naldini L. Gene therapy for ADA - SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal produGene therapy for ADA - SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal produgene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products.

First academic GLP center for performing biodistribution, toxicology / tumorigenicity and validation studies on gene and cell therapy products.