Sentences with phrase «gene therapy treatment for»

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.

4 / 18 / 2018First Gene Therapy Treatment for Cerebral ALD Wins National Clinical Research Excellence Award

Led by Florian Eichler, MD, from Harvard Medical School and Massachusetts General Hospital, the study tested the first successful gene therapy treatment for cerebral adrenoleukodystrophy (ALD), a fatal degenerative brain disease that most severely affects boys.

Washington, D.C. — April 18, 2018 — The Clinical Research (CR) Forum, a non-profit membership association of top clinical research experts and thought leaders from the nation's leading academic health centers, today awarded its most prestigious honor to a Massachusetts General Hospital research team for its discovery of the first successful gene therapy treatment for a fatal brain disease, cerebral adrenoleukodystrophy (ALD).

Spark Therapeutics announced yesterday that they will be taking a gene therapy treatment for LHON (Leber Hereditary Optic Neuropathy) into clinical trials.

UC San Diego Health joins national clinical trial testing a gene therapy treatment for individuals with hemophilia B.

Gene therapy: Généthon starts a clinical trial to test a gene therapy treatment for a rare liver disease, Crigler - Najjar Syndrome

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.

French teams from CIC Biothérapie (AP - HP / Inserm), from pediatric hematology department of Necker Hospital for Children (AP - HP), led by Marina Cavazzana, Salima Hacein Bey Albina and Alain Fischer and from Genethon led by Anne Galy (Genethon / Inserm UMR - S951), and English teams from UCL Institute of Child Health and Great Ormond Street Hospital in London led by Adrian Thrasher and Bobby Gaspar demonstrated the efficacy of gene therapy treatment for Wiskott - Aldrich Syndrome (WAS).

«Both of the designed linker proteins may possibly be used in the future as a gene therapy treatment for congenital muscular dystrophy,» says Rüegg.

These human genes were also protective against alpha - synuclein - induced death, suggesting that they could be worth testing as gene therapy treatments for Parkinson's disease, Lu says.

The Jan. 12 issue of the journal Science provides an in - depth and timely review of the key developments that have led to several successful gene therapy treatments for patients with serious medical conditions.

There a team has been working to create gene therapy treatments for GM1 in cats.

Not exact matches

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.

The treatment, named Kymriah, was hailed by doctors and the life sciences community as a major advance in medicine and a boon to children and young adults with a certain form of leukemia (the group for whom the gene therapy is approved).

The first gene therapy in the U.S. now has a price tag: $ 850,000 for the one - time treatment, or, more specifically, $ 425,000 per eye for a retinal disorder.

The treatment is delivered just once, a facet of gene therapy that poses unique pricing questions in an industry fueled by steady payments for chronic therapies.

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

And a third, different kind of gene therapy from Spark Therapeutics — to treat a rare inherited form of blindness — is likely to be approved by the end of 2017, too, making this year a landmark for treatments that manipulate the body's own biological mechanisms in novel new ways to fight deadly diseases.

Katherine High, Spark's president and chief scientific officer, expressed her enthusiasm for the early clinical data related to SPK - 8011: «The encouraging start of our SPK - 8011 clinical trial reinforces the strength of our gene therapy platform, delivers human proof - of - concept in a second liver - mediated disease — a significant achievement in the gene therapy field — and positions us well to potentially transform the current treatment approach for this life - altering disease with a one - time intervention.»

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.

He and his lab, for example, along with the company Neurologix based in Fort Lee, N.J., are conducting a clinical trial to test a similar gene - therapy treatment for Parkinson's disease.

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

«Together, our data strongly suggest that cutaneous gene therapy with inducible expression of GLP1 can be used for the treatment and prevention of diet - induced obesity and pathologies,» the authors wrote.

This study represents a significant step towards the development of clinical trials in gene therapy for the curative treatment of hereditary deafness and balance loss in humans.

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.

New treatments for spinal cord injury, including stem cells, gene therapy and electrical stimulation, are being studied.

«Steep funding cuts for the federal health agencies are counterproductive at a time when innovative research is moving us closer to identifying solutions for rare diseases, new prevention strategies to protect Americans from deadly and costly conditions, advances in gene therapy, new technologies for understanding the brain, and treatments that harness the ability of our immune system to fight cancer.»

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.

An extended transplant window could make time for drug or stem - cell based treatments — even gene therapy.

A year after a government - appointed committee gave the green light to gene therapy, Britain still has no mechanism for approving any experimental treatments.

«It's a method that is much more feasible and safer for patients than traditional gene therapy, which uses modified viruses to carry out the treatment.»

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.

The findings could help improve treatments for HIV infection and make gene therapy safer and more efficient.

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.

Gene therapy death The Food and Drug Administration stopped a gene therapy trial this week after a patient undergoing the experimental treatment for a severe form of arthritis had a fatal reactGene therapy death The Food and Drug Administration stopped a gene therapy trial this week after a patient undergoing the experimental treatment for a severe form of arthritis had a fatal reactgene therapy trial this week after a patient undergoing the experimental treatment for a severe form of arthritis had a fatal reaction.

A gene therapy treatment called CAR - T immunotherapy has been approved for use in a rare type of leukemia.

The nanotechnology approach that is being tested for inhibitor control could also improve the haemophilia treatment that is now at the cutting edge of clinical research: gene therapy.

One potential treatment for CF is gene therapy, and a major challenge in gene therapy is packaging replacement genes so they can be delivered to the target cells.

Gene therapy suffered a setback in 1993 when a person undergoing such treatment for CF almost died.

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.

«Regarding the first part of the question, gene therapy is approaching clinical realization for the treatment of neoplastic and metabolic diseases.

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.

Although several experimental trials of gene therapy are under way, some of them for more than a decade, uniQure is the first in the West to win regulatory approval for a commercial 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.

As a new generation of gene therapy clinical trials shows promise to cure or halt the progression of several rare diseases, the time has come to explore ways to pay for the cutting edge treatments, a pediatric hematologist - oncologist from Dana - Farber / Boston Children's Cancer and Blood Disorders Center argues in a commentary published by the journal Science.

Showing that the LCA2 gene therapy treatment works best in children is «a big step» for inherited blindness, says geneticist Frans Cremers of Radboud University Nijmegen Medical Center in the Netherlands, who wrote an accompanying commentary in The Lancet.

For more than two decades, researchers have explored using gene therapy, an experimental treatment, to replace the flawed gene with a healthy copy.

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.