Sentences with phrase «gene therapy products»

DNA - based gene therapy products have been in clinical development since the 1990s.
GlaxoSmithKline has sold a number of gene therapy products — including its approved autologous ex vivo product Strimvelis — to Orchard Therapeutics.
Most recently, she has led the planning and execution of a program for a new gene therapy product candidate, from ideation through proof - of - concept study, and mapping out the development plan.
We entered into an agreement with Bayer to research, develop and commercialize AAV gene therapy products for treatment of hemophilia A.
Although currently there are no FDA - approved gene therapy products, an effective gene therapy will probably gain FDA approval within the next three to five years.
China has made crucial breakthroughs in the field, including creating the world's first commercialized gene therapy product, Gendicine.
The MAA is in the validation stage and once approved, GLYBERA ® will be the first gene therapy product to be marketed in Europe.
Supported by: Hear from leading expert Dr David DiGiusto, Stanford University School of Medicine, USA, as he shares his extensive experience in developing and optimizing manufacturing processes for cell and gene therapy products including:
Among other things, we plan to include certain gene therapy products that permanently alter tissue and produce a sustained therapeutic benefit as part of the products that will meet the definition of being eligible to come under the pathway enabled by RMAT.
In 1998 he co-founded Amsterdam Molecular Therapeutics (AMT; currently Uniqure), a gene therapy company that develops AAV - based gene therapy products.
China was the first country to approve a gene therapy product for commercial use, in 2004.
The clinical trial of nine adult hemophilia B patients, aged 18 to 52 years, used a single dose of a gene therapy product engineered to enter patients» liver cells and direct the production of the blood clotting factor that they lack.
Previous hemophilia gene therapy trials have been frustrated by an immune response to the gene therapy product that limited the success of the therapy.
George notes, «Our goal in this trial was to evaluate the safety of the gene therapy product and secondarily, to determine if we could achieve levels of factor IX that could decrease bleeding events in patients.»
Two additional patients received the gene therapy product too recently to determine quality - of - life measures.
Although there are currently no gene therapy products on the market in the U.S., recent studies in both Parkinson's disease and Leber congenital amaurosis, a rare form of blindness, have returned very promising results.
The complexity of the development of cells and gene therapy products are addressed by designing tailored studies to allow safety assessment of GTMPs and to fulfil GLP requirements and OECD principles.
The trial, sponsored by Généthon, will include 17 patients in four centres in Europe, and will assess safety and therapeutic efficacy of the gene therapy product developed by Généthon.
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.
This is followed by a presentation by Dr Alaina Schlinker, Fresenius Kabi, as she discusses practical approaches to addressing some of the key processing challenges for cell and gene therapy products.
«Money back guarantee» for cell - gene therapy drug High prices for marketed cell / gene therapy products is a hot and controversial topic.
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