Sentences with phrase «disease gene therapy drugs»

This week's headlines include: GSK slims portfolio with sale of rare disease gene therapy drugs, The...

This week's headlines include: GSK slims portfolio with sale of rare disease gene therapy drugs, The Focus Shifts in Alzheimer's Research, Healing process after breast cancer surgery may trigger...

LONDON, April 12 - GlaxoSmithKline is divesting its rare disease gene therapy drugs to Orchard Therapeutics, it said on Thursday, as Chief Executive Emma Walmsley makes good on her promise to prune the drugmaker's pharmaceuticals portfolio.

LONDON, April 12 - GlaxoSmithKline is divesting its rare disease gene therapy drugs to private biotech company Orchard Therapeutics as Chief Executive Emma Walmsley makes good on her promise to prune the drugmaker's pharmaceuticals portfolio.

Not exact matches

Shares of biotech Spark Therapeutics rose 4.5 % in early Monday trading after the Food and Drug Administration (FDA) agreed to review its gene therapy for vision loss patients who have vision loss due to a rare genetic condition called biallelic RPE65 - mediated inherited retinal disease (IRD).

U.S. drug giant Pfizer and California - based biotech Sangamo Therapeutics are partnering to develop a gene therapy to treat ALS, or Lou Gehrig's disease.

Approved treatments included gene therapies and the first new drugs for rare diseases in many years.

Gene therapy delivered to a specific part of the brain reverses symptoms of depression in a mouse model of the disease — potentially laying the groundwork for a new approach to treating severe cases of human depression in which drugs are ineffective.

AveXis, Inc., a clinical - stage gene therapy company developing treatments for patients suffering from rare and life - threatening neurological genetic diseases, announced in July 2016 that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for the treatment based on preliminary clinical results from the trial of AVXStherapy company developing treatments for patients suffering from rare and life - threatening neurological genetic diseases, announced in July 2016 that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for the treatment based on preliminary clinical results from the trial of AVXSTherapy Designation for the treatment based on preliminary clinical results from the trial of AVXS - 101.

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.

This achievement will also contribute to accelerating the research applications of RNAi such as to the development of RNA - based next - generation drugs, for example as gene therapy to suppress the production of a disease - causing protein.

«With more than 1.7 million people dying globally from TB each year and the rise of strains that are resistant to drug treatment, we need a better way to prevent this disease,» said the study's principal investigator Louis Picker, M.D., who is the associate director of the OHSU Vaccine and Gene Therapy Institute and a professor of pathology, molecular microbiology, and immunology in the OHSU School of Medicine.

Dr Wenxin Wang is trying to uncover therapies for diseases such as diabetic ulcers and Epidermolysis Bullosa, which causes chronic skin conditions: «We are currently investigating the use of these new materials for biomedical applications such as drug / gene delivery, cross linkable hydrogel materials and skin adhesives.

Genes that increase risk for AD are potential targets for new disease - modifying AD drug therapies.

Professor Moffatt said: «The genes we identified represent new potential drug targets for allergic diseases as well as biomarkers that may predict which patients will respond to existing expensive therapies.»

«Database of disease genes shows potential drug therapies.»

One of those could be a widely applicable drug or nutritional supplement, says Tsang, who has been involved in developing gene therapy for treating eye diseases.

This will give way to the development of a drug to be used in gene therapy against neurodegenerative diseases based on small molecules which enhance the expression of the gene and / or the use of fragments of the Klotho protein itself.

The next step will be to optimize the approach and to extend it to protein - protein interactions, protein - gene interactions, disease - protein interactions, disease - drug interactions all with a view to improving diagnostics and tailoring therapy for the individual patient based on the outcomes of their personal biological network analysis.

The mice will help scientists to understand how the disease develops and enable them to test new drugs and gene therapies.

According to Sinclair, all of the mammalian SIRT genes (and their proteins) are possible drug targets for therapies aimed at extending life, as well as staving off age - related illnesses, such as Alzheimer's disease, cancers and metabolic disorders, like diabetes.

It is expected that the new technique will be applied to gene therapy in the future in addition to providing a powerful tool for breeding useful organisms and conducting disease and drug - discovery research.

April 23, 2018 Ultragenyx Announces Filing and FDA Clearance of an Investigational New Drug Application for DTX401, a Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia

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.

«With our enhanced method of direct cardiac reprogramming, we hope to combine gene therapy with drugs to create better treatments for patients suffering from this devastating disease.»

Chicago, Ill., July 20, 2016 — AveXis, Inc., a clinical - stage gene therapy company developing treatments for patients suffering from rare and life - threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for AVXS - 101, the company's lead development candidate for the treatment of spinal muscular atrophy (SMA) Type 1 in pediatric patherapy company developing treatments for patients suffering from rare and life - threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for AVXS - 101, the company's lead development candidate for the treatment of spinal muscular atrophy (SMA) Type 1 in pediatric paTherapy Designation for AVXS - 101, the company's lead development candidate for the treatment of spinal muscular atrophy (SMA) Type 1 in pediatric patients.

Cleveland and the members of his lab use these therapies, also known as designer DNA drugs, to silence genes involved in the development of various neurodegenerative diseases such as Alzheimer's, Huntington's and Lou Gehrig's disease.

The current competence fields and main contributions of Daniel Scherman concern drug gene therapy, drug delivery, in vivo imaging, and rare drug diseases.