Sentences with phrase «on gene therapy treatments»

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Spark Therapeutics» stock soared 20 % in Wednesday trading on the heels of its second quarter 2017 earnings report and some (very) early data on the gene therapy - focused firm's treatment for the blood disorder hemophilia A.

That means the treatment could be approved on or before January 12, 2018, which would make it the first - ever gene therapy cleared to treat a genetic condition in the U.S..

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 type of treatment is what we call gene therapy and researchers have been working on developing techniques to accomplish this for many years.

Tsigelny added: «The clinical treatments that do not take into consideration the specific mutations in genes, but rather treat all mutations within a specific gene such as EGFR as the same, are a «hit - or - miss» game because they are based solely on observational data on how other patients previously have reacted to drug therapies.»

Based on these promising results, work has begun on the necessary developments to propose to patients suffering from Friedreich's ataxia and presenting a progressive cardiomyopathy a treatment by gene therapy.

Although gene therapy has since proved successful against rare diseases of the immune system and shown promise against AIDS (see «Genetic treatment closes door on HIV»), it has proved much harder to use it to treat CF.

LONDON (Reuters)- The science of gene therapy is finally delivering on its potential, and drugmakers are now hoping to produce commercially viable medicines after tiny sales for the first two such treatments in Europe.

Under the proposed law, gene therapy will be approved only for the treatment of people with genetic diseases such as cystic fibrosis and will not be allowed in germ cells, where genetic alterations would be passed on to the next generation.

According to Saura, «this study opens up new perspectives on therapeutic prevention and treatment of Alzheimer's disease, given that we have demonstrated that a gene therapy which activates the Crtc1 protein is effective in preventing the loss of memory in lab mice».

Dr. Giatsidis and coauthors reviewed the state of the art in research on gene therapy techniques for treatment of local disorders and injuries — the first such review in more than a decade.

Gene Therapy Approaches Studied for «Almost Every Tissue»... Dr. Giatsidis and coauthors reviewed the state of the art in research on gene therapy techniques for treatment of local disorders and injuries — the first such review in more than a decGene Therapy Approaches Studied for «Almost Every Tissue»... Dr. Giatsidis and coauthors reviewed the state of the art in research on gene therapy techniques for treatment of local disorders and injuries — the first such review in more than a Therapy Approaches Studied for «Almost Every Tissue»... Dr. Giatsidis and coauthors reviewed the state of the art in research on gene therapy techniques for treatment of local disorders and injuries — the first such review in more than a decgene therapy techniques for treatment of local disorders and injuries — the first such review in more than a therapy techniques for treatment of local disorders and injuries — the first such review in more than a decade.

This specificity in treatment fits with an emerging approach in cancer treatment nationwide, known as personalized medicine, in which the therapies for each patient are selected based on the genes altered in their tumors.

When tested on mice that already had the disease, the treatment significantly reversed the symptoms just eight days after the gene therapy was delivered.

Paris, France, April 3, 2018, 5.35 pm CET — GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA - PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced topline results from the REVERSE Phase III clinical trial evaluating the safety and efficacy of a single intravitreal injection of GS010 (rAAV2 / 2 - ND 4) in 37 subjects whose visual loss due to 11778 - ND4 Leber Hereditary Optic Neuropathy (LHON) commenced between 6 and 12 months prior to study treatment.

A significant percentage of children and young adults with treatment - resistant B - cell leukemia achieved remission through a new CAR T - cell gene therapy that destroys cancer cells with the CD22 molecule on its surface.

The FDA has signed off on Ultragenyx Pharmaceutical's (RARE -1.1 %) IND for gene therapy DTX401 for the potential treatment of glycogen storage...

Clinical research activities are focused on the implementation of experimental protocols based on gene or cell therapy for the treatment of genetic blood disorders such as ADA - SCID, Wiskott - Aldrich Syndrome, Metachromatic Leukodystrophy, Beta - thalassemia, Chronic granulomatous disease, Mucopolysaccharidosis Type I (Hurler).

This work, published in «Blood», was carried out by the CNIO Telomeres and Telomerase Group The treatment is based on the transport of the telomerase gene to the bone marrow cells using gene therapy, a completely new strategy in the treatment of aplastic anaemia

«Gene therapy, or any treatment that relies on tight regulation of gene expression, could be improved by precisely targeting molecules to the right place in the nucleus,» says KarGene therapy, or any treatment that relies on tight regulation of gene expression, could be improved by precisely targeting molecules to the right place in the nucleus,» says Kargene expression, could be improved by precisely targeting molecules to the right place in the nucleus,» says Karpen.

Up to 50 million Americans are estimated to have an autoimmune condition, * and annual direct healthcare treatment costs for these patients total $ 100 billion.1 Nearly 100 different diseases have been classified as autoimmune in nature, with 40 more suspected.1 IFM's 2018 Annual International Conference (AIC), «Solving the Puzzle of Autoimmunity: The Interplay of Gut, Genes, and Environment,» focuses on the exploding research and therapies around the interconnected roles of intestinal permeability, genetic disruptors, and various environmental triggers that coalesce to create autoimmune disease.

Auburn University College of Veterinary Medicine, UMass Medical School to test gene therapy treatment for Tay - Sachs and Sandhoff diseases Researchers at Auburn University College of Veterinary Medicine and UMass Medical School are nearing human clinical trials on a genetic therapy for two rare neurological diseases that are fatal to children.

Research done on cartilage transplantation and gene cell therapy in the Orthopaedics Laboratory has been utilized in the treatment of knee injuries in people.

«We look forward to helping the company realize the tremendous promise of its gene therapy platform, which has the potential to provide patients suffering from these severe eye diseases with treatments that offer life - long clinical benefits, potentially based on a one - time therapeutic administration.»