Sentences with phrase «gene therapy the treatment of»

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 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.

Luxturna is the first of a crop of treatments that target diseases caused by mutations in specific genes, and thus is referred to by many as the first gene therapy in the U.S.

Just this year, over 50 U.S. genetics and gene therapy startups raised at least $ 1 million to support genetics - based treatments, including speeding trails, improving accuracy of tests, and providing better platforms.

«Today's approval marks another first in the field of gene therapy — both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss — and this milestone reinforces the potential of this breakthrough approach in treating a wide - range of challenging diseases,» FDA Commissioner Scott Gottlieb said in a statement.

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.

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.

Gene therapies promise to revolutionize the treatment of many diseases, including neurological diseases such as ALS.

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.

Patients with Parkinson's disease may be the first group to benefit from gene therapy, the much hyped technique that has yet to result in a single reliable treatment despite nearly two decades of experimentation.

«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.»

The SMA model pig was created using a gene therapy approach by knocking down the levels of pig SMN, followed by treatment with human SMN at early and late time points.

Researchers who previously showed that a gene therapy treatment could save the lives of dogs with a deadly disease called myotubular myopathy — a type of muscular dystrophy that affects the skeletal muscles — have found that the therapy is long - lasting.

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

Now a paper in the November 2009 issue of Science suggests that the long - sought cure may come from gene therapy — a famously hyped approach to treatment that tragically caused the death of a teenage experimental subject in 1999.

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 achievement could open the way to a genetic treatment against the growth of smooth muscle tissue that can block arteries in heart patients, and ultimately to other targeted gene therapies.

The breakthrough has resulted from the merger of interlocking fields — gene therapy and cell therapy — which are now spawning near - miraculous treatments and cures.

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.

After this incident, and the death in 1999 of a young man in a clinical trial, apparently because of an immune reaction to the treatment, gene therapy research grounds nearly to a halt.

But he warns that gene therapy is still in its infancy worldwide and that even aside from the safety concerns, many technical problems must be solved before hopes of successful treatments can be raised.

«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.

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.

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.

«We report here a gene therapy dose - finding study in a large animal model of a severe muscle disease where a single treatment resulted in dramatic rescue,» said Childers.

After a year of treatment, in the 62 patients who received the gene therapy, FEV1 was 3.7 % greater compared to placebo.

The emergency treatment would be the first test of their gene therapy approach over such a large and severely damaged area.

Overall, the gene therapy was well tolerated and patients in the treatment and placebo groups experienced similar rates of adverse events.

«Today's approval marks another first in the field of gene therapy — both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss — and this milestone reinforces the potential of this breakthrough approach in treating a wide - range of challenging diseases,» FDA Commissioner Scott Gottlieb said in a statement.

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.

A new gene therapy treatment has restored some sight in a handful of blind patients suffering from Leber's congenital amaurosis, a syndrome in which, because of a broken or missing gene called RPE65, retinal photoreceptor cells malfunction and eventually die.

By targeting the fundamental DNA of the gene, the zinc finger therapy also has the advantage over other potential Huntington's therapies of needing less frequent treatments.

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.

The authors looked to the future of gene therapies, and the challenges of delivering these complex treatments to patients.

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.