Sentences with phrase «of gene therapy products»

GlaxoSmithKline has sold a number of gene therapy products — including its approved autologous ex vivo product Strimvelis — to Orchard Therapeutics.

The researchers assessed LPLD - related acute abdominal events that required hospital care in a small group of patients treated with a single dose of the gene therapy product Glybera ®.

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.»

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.

Not exact matches

Abeona Therapeutics (ABEO)- The company announced receipt of Orphan Drug Designation from the European Medicines Agency Committee for Orphan Medicinal Products for gene therapy program ABO - 202.

Risk Versus Reward: The Value of Cell Therapy for Patients and Investors Source: Streetwise Reports (4/25/18) The cell therapy space, encompassing disruptive new treatment including stem cell therapy, immunotherapy and gene editing, has begun to mature, with a handful of product approvals and others in late - stage develTherapy for Patients and Investors Source: Streetwise Reports (4/25/18) The cell therapy space, encompassing disruptive new treatment including stem cell therapy, immunotherapy and gene editing, has begun to mature, with a handful of product approvals and others in late - stage develtherapy space, encompassing disruptive new treatment including stem cell therapy, immunotherapy and gene editing, has begun to mature, with a handful of product approvals and others in late - stage develtherapy, immunotherapy and gene editing, has begun to mature, with a handful of product approvals and others in late - stage development.

The cell therapy space, encompassing disruptive new treatment including stem cell therapy, immunotherapy and gene editing, has begun to mature, with a handful of product approvals and others in late - stage development.

These include the ability to bring new, innovative products to the market; progress in oncology, such as the approval of Genentech's drug Avastin for breast cancer and advances in the use of gene therapy, despite some setbacks; continuing progress in research on stem cells; the emergence of treatments for previously untreated diseases; and solutions for food and fuel shortages, such as biocrops and biofuels.

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.

They do social science, health, philosophical, policy, or legal research on topics such as privacy, confidentiality, the psychological impact of genetic information, informed - consent issues in genomics research, commercialization of genetic products, genetically modified foods, behavioral genetics, gene testing, and gene therapy.

Previous hemophilia gene therapy trials have been frustrated by an immune response to the gene therapy product that limited the success of the therapy.

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.

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.

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.

Our definition is similar to the European Medicines Agency (EMA) definition of Advanced Therapy Medicinal Product (ATMP): «Medicinal product for human use that is a gene therapy medicinal product, a somatic cell therapy medicinal product or tissue engineered product» (EMA ATTherapy Medicinal Product (ATMP): «Medicinal product for human use that is a gene therapy medicinal product, a somatic cell therapy medicinal product or tissue engineered product» (EMA ATProduct (ATMP): «Medicinal product for human use that is a gene therapy medicinal product, a somatic cell therapy medicinal product or tissue engineered product» (EMA ATproduct for human use that is a gene therapy medicinal product, a somatic cell therapy medicinal product or tissue engineered product» (EMA ATtherapy medicinal product, a somatic cell therapy medicinal product or tissue engineered product» (EMA ATproduct, a somatic cell therapy medicinal product or tissue engineered product» (EMA ATtherapy medicinal product or tissue engineered product» (EMA ATproduct or tissue engineered product» (EMA ATproduct» (EMA ATMP Reg.

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.

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:

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.

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.

American Society of Gene and Cell Therapy Annual Meeting Speaker: Olivia Kelly, Ph.D., Senior Director, Cell Biology Presentation: Developing a Cell Therapy Combination Product for Diabetes Date / Time: May 16, 8:30 AM EDT Location: Hilton Chicago, Chicago

We entered into an agreement with Bayer to research, develop and commercialize AAV gene therapy products for treatment of hemophilia A.

Part 1 of this two - part report on that meeting describes factors influencing the choices of lot - release assays for vaccines and gene - therapy products (1).

The idea there is that by identifying genes that cause, or contribute to glaucoma, and understanding what the protein products of those genes do, we can use new techniques to target those proteins to develop therapies that actually approach the actual disease mechanisms that cause the disease.

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.

Targets of research range from basic molecular and cellular mechanisms, the manipulation of these mechanisms in animal models, analyses of the genes and gene products in cardiovascular disease, and clinical research that seeks to improve diagnosis and therapy for patients.

With the currently approved FDA cancer therapeutics drugs, we are only targeting the tip of the iceberg (85 targeted therapies for 105 gene products (1)-RRB- and with over 22,000 genes in our genome many genes are being neglected!

Preparation and global prosecution of patent applications directed to diagnostic and therapeutic products including antibodies, gene therapies, vaccines, stem cells, pharmaceutical formulations, nanotechnology, nucleic acid molecules, biologics, medical devices, mouse platforms for antibody development, and methods of treating diseases.

Alachua, Fla. - based AGTC is a clinical - stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe inherited orphan diseases in ophthalmology.

AGTC plans to use the net proceeds of the offering to fund preclinical investigation and Phase 1/2 trials of potential product candidates for treatment of wet AMD; to expand its manufacturing capabilities and create a pilot manufacturing group; to in - license, acquire or invest in complementary gene therapy, technologies, products or assets; and for working capital and other general corporate purposes.