Sentences with phrase «developing gene therapy treatments»

The research suggests the possibility, Kahn explains, of developing gene therapy treatments using fat cells that aid in treating metabolic diseases, cancer or other conditions in the liver or other organs.

Not exact matches

The finding offers potential for developing both gene therapy and more effective protein replacement treatments for hemophilia A, the most common form of hemophilia.

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.

When conventional wisdom said only gene therapy could correct the root cause of cystic fibrosis, Vertex developed pill - based treatments that address the underlying disease mechanism, says Altshuler.

Researchers at the Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Children's Hospital and Houston Methodist have developed an alternative treatment in which virus - specific cells protect patients against severe, drug - resistant viral infections.

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

But for a gene therapy to be effective, one must know the precise gene responsible for a given individual's disorder and develop a tailored treatment.

A bone marrow transplant is the only standard treatment, but the gene therapy approach, developed by researchers at the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy, offers a new line of attgene therapy approach, developed by researchers at the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy, offers a new line of therapy approach, developed by researchers at the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy, offers a new line of attGene Therapy in Milan, Italy, offers a new line of Therapy in Milan, Italy, offers a new line of attack.

Noting the potential of gene therapy to be a one - time treatment for rare and serious diseases that otherwise cost hundreds of thousands, if not millions, of dollars in chronic care over a lifetime, Stuart Orkin, MD, and co-author Philip Reilly, MD, JD, of Third Rock Ventures, seek to «catalyze the discussion» by suggesting several new models for valuing, pricing and developing gene therapy.

«Gene therapy may represent a leading strategy to develop more efficient regenerative surgical treatments for numerous clinical needs,» they write.

A «miracle» gene therapy treatment for children suffering from the fatal «bubble boy» disease has been halted in France, after one of the patients developed leukaemia as a direct consequence of the treatment.

The goal of the Program in Human Gene Therapy is to develop gene transfer technologies and use them for hepatic gene therapy for the treatment of genetic and acquired diseaGene Therapy is to develop gene transfer technologies and use them for hepatic gene therapy for the treatment of genetic and acquired diTherapy is to develop gene transfer technologies and use them for hepatic gene therapy for the treatment of genetic and acquired diseagene transfer technologies and use them for hepatic gene therapy for the treatment of genetic and acquired diseagene therapy for the treatment of genetic and acquired ditherapy for the treatment of genetic and acquired diseases.

The team hopes to develop a single, injected, gene therapy treatment that could eliminate many severe allergic responses (Credit: University of Queensland)

The team hopes to develop a single, injected, gene therapy treatment that could eliminate many severe allergic responses

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

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.

The treatment, developed at the University of Pennsylvania with clinical trials carried out at Children's Hospital of Philadelphia and the University of Iowa, is the first time gene therapy is used to treat an inherited disease and could lead to treatments of other currently untreatable conditions.

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.

There are at least 265 different genes in which mutations can cause blindness; breakthroughs in gene therapy are allowing scientists to develop treatments for diseases that were previously untreatable, giving hope to millions.

AveXis is a clinical ‐ stage gene therapy company developing treatments for patients suffering from rare and life ‐ threatening neurological genetic diseases.