Welcome to Gene Therapy Net

Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials. For other useful information on pain management and minimally invasive spine treatment, consider Inspired Spine, who are specialists in these areas.

Gene therapy for beta-thalassemia safe, effective in people

Posted on: 19 April 2018, source: EurekAlert
In a powerful example of bench-to-bedside science showing how observations made in the lab can spark life-altering therapies in clinic, an international team of clinician-investigators has announced that gene therapy for patients with a severe form of the blood disorder beta-thalassemia can be safe and effective. Led by study director Philippe Leboulch, MD, a sponsored collaborator in Brigham and Women's Hospital's Division of Genetics and lecturer in medicine at Harvard Medical School, the research team reports that a one-time treatment with the gene therapy known as LentiGlobin BB305 vector reduced or eliminated the need for blood transfusions in 22 patients with severe beta-thalassemia. The team's results are published in the April 19 issue of The New England Journal of Medicine.

13-Year-Old Boy Is First Person in US to Receive Newly Approved Gene Therapy for Blindness

Posted on: 22 March 2018, source: Gizmodo
On Tuesday, a 13-year-old boy from New Jersey was at the center of medical history as he became the first person in the US to receive an FDA-approved gene therapy for an inherited disease. The event marks the beginning of a new era of medicine, one in which devastating genetic conditions that we are born with can be simply edited out of our DNA with the help of modern biomedical technologies. The therapy, Luxturna, from Spark Therepeutics, was approved by the FDA in December to treat a rare, inherited form of blindness. Its price tag, set at $850,000—or $425,000 per eye—made it the most expensive drug in the US and sparked mass sticker-shock. But the therapy, which in high-profile clinical trials has allowed patients to see the stars for the first times, also offered the almost miraculous possibility of giving sight to the blind.

Gene therapy found safe in trial of Pompe disease treatment

Posted on: 22 February 2018, source: UPI news
The first human trial of gene therapy to treat respiratory problems in early-onset Pompe disease of children was found safe, according to health researchers in Florida. University of Florida Health scientists conducted a successful trial of nine participants who had the incurable disease, which damages muscle and nerve cells throughout the body. Results of the treatment, which utilizes an adeno-associated virus, were published in the journal Human Gene Therapy Clinical Development.

High-dose AAV gene therapy appears toxic in monkeys and piglets

Posted on: 6 February 2018, source: Science
A gene therapy trial that recently led to dramatic benefits for babies born with a fatal neuromuscular condition has raised hopes for using a similar approach to treat other diseases. But a new animal study suggests that the high doses (2x10E14 genome copies per kilogram body weight) of gene-carrying viruses used in such treatments may not always be as safe as the human clinical trial indicated. In the new research, the disclosure of which briefly sent the stock prices of several gene therapy companies plummeting yesterday, researchers injected a handful of young monkeys and pigs with many copies of adeno-associated virus 9 (AAV9), a normally harmless virus that infects neurons and is increasingly being used to ferry therapeutic genes into cells to treat neuromuscular diseases. Within days, some of the animals developed severe liver and neuron damage.

Chinese scientists used Crispr gene editing on 86 human patients

Posted on: 25 January 2018, source: qz.com
China is taking the lead in the global race to perfect gene therapies. Scientists have genetically engineered the cells of at least 86 cancer and HIV patients in the country using Crispr-Cas9 technology since 2015, the Wall Street Journal reports (paywall). Although no formal scientific papers have been written about these experiments, doctors told journalists at the WSJ that some patients have improved. There have also been at least 15 deaths, although only about half of them were reportedly related to the gene therapy itself. These therapies, which involved taking the immune cells from hospital patients, editing the cells, and transfusing them back into the body, are the first to use Crispr-Cas9 in living humans.

Gene Therapy For Rare Forms Of Blindness Will Cost Nearly Half A Million Per Eye

Posted on: 4 January 2018, source: IFLScience
The pharmaceutical company behind a gene therapy for inherited blindness has announced the product’s price, and sticker shock has sent news outlets worldwide into a collective tizzy. Spark Therapeutics plans to charge $425,000 per eye for the therapy, called Luxturna, making it the most expensive drug ever marketed in the US. The landmark FDA approval of Luxturna in December 2017 represented the first approval of a gene therapy for an inherited condition, and only the third-ever approval of a gene therapy.

Gene therapy shows 'mind-blowing' success in treating haemophilia A

Posted on: 19 December 2017, source: BioNews
A new gene therapy for haemophilia A has exceeded expectations in a recent clinical trial. All seven patients who received the high-dose therapy to correct a faulty gene for a crucial blood clotting protein showed considerable improvement. 'This is huge,' Professor John Pasi, who led the trial at Barts and Queen Mary University of London told the BBC. He added that having 'the option to think about normalising levels' of the blood clotting protein factor VIII in patients with severe haemophilia was 'mind-blowing'. Gene therapy could be a potential 'cure' for haemophilia, suggested an editorial accompanying the study published in the New England Journal of Medicine.