eXTReMe Tracker
Gene Therapy Net RSS feed Follow Gene Therapy Net on Twitter LinkedIn - Gene Therapy Net discussion group Facebook - Gene Therapy Net

International Consortium

Related Links

Gene-Therapy Company Crispr Drops as FDA Puts Trial on Hold

Posted on: 10 June 2018, source: Bloomberg
Crispr Therapeutics AG plunged after the gene-editing company said a planned trial of its treatment to help people with sickle-cell disease was being put on hold by U.S. regulators. Crispr is developing the therapy with Vertex Pharmaceuticals Inc., and the trial had yet to begin. The companies said in a statement Wednesday that the Food and Drug Administration placed a halt on the experiment until the agency could get more information from the companies.

Register to read more ...

A Harvard startup is using gene therapy to reverse ageing in dogs, humans are next

Posted on: 14 May 2018, source: Moneycontrol.com
In order to achieve the objective to “have the body and mind of a 22-year-old but the experience of a 130-year-old,” a Harvard professor backed startup is planning to rejuvenate dogs using gene therapy. And, if results are positive, the same approach could be used in humans. Rejuvenate Bio, co-founded by George Church of Harvard Medical School, has already carried out age-defying tests on beagles and claims it will make animals “younger" by adding new DNA instructions to their bodies.

Register to read more ...

Latest Up and Coming Gene Therapies

Posted on: 8 May 2018, source: GEN
Gene therapy’s numbers look promising. In a year full of biopharma mergers and acquisitions, one of the biggest deals has been Novartis’ planned $8.7 billion purchase of AveXis, whose lead candidate AVXS-101 has advanced into a pivotal trial following positive Phase I data published in November in The New England Journal of Medicine. Additional figures furnished to GEN by Informa show the extent of gene-therapy development: Informa Pharma Intelligence’s Trialtrove database records 729 gene therapies as having been developed, of which nearly two-thirds (461) were preclinical. Those therapies have been assessed in 1,855 clinical trials, most in early phases: 657 in Phase I, 509 in Phase I/II, and 455 in Phase II. As for later development, 89 have reached Phase III, 32 are in Phase II/III, and 28 in Phase IV.

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...

FDA warns against ‘do it yourself’ gene therapy kits

Posted on: 28 November 2017, source: Bizjournals
The U.S. Food and Drug Administration has issued a stern warning against the use of “do it yourself” gene therapy kits, pushing back against a nascent “biohacker” movement that seeks to make experimental medicines and technologies available to the masses, often by circumventing regulators. A statement posted on the FDA’s website on Tuesday cautions that it is illegal to sell gene therapy products and kits intended for self-administration. Gene therapy involves inserting new, healthy genes into cells to cure diseases that are caused by faulty genes. The FDA has classified gene editing — cutting out and replacing parts of faulty genes — as a form of gene therapy. A host of local biotechs, including Cambridge-based Editas Medicine (Nasdaq: EDIT), are seeking to develop such treatments.

Register to read more ...

Alarmed by Recent Events, Scientists Speak Out Against DIY Gene Therapy

Posted on: 13 December 2017, source: Gizmodo
It seems hard to fathom that scientific progress has advanced to a point where it’s suddenly possible for people to undertake something as complicated as gene therapy outside the safety and confines of a well-funded lab, but it has. In just the past few months we’ve seen not one, but two examples of people pursuing unregulated gene therapy. That’s right, people are genetically modifying their bodies in DIY labs and friend’s apartments. Now, the largest organization of scientists who work in gene and cell therapy have come out against DIY gene therapies. A statement from the American Society of Gene and Cell Therapy comes on the heels of a stern warning against DIY gene therapy from the US Food and Drug Administration. For years, when it came to biohacking, regulators and establishment science have mainly looked the other way. That seems to be changing.

Register to read more ...

First gene-editing attempted in human body to cure disease

Posted on: 21 November 2017, source: The Telegraph
Doctors have edited a patient's genes in a world-first attempt to alter DNA from within the human body. Medics in California administered the experimental intravenous treatment, which provides the body with instructions to change DNA, on Brian Madeux, who suffers from Hunter's syndrome. People with Hunter's syndrome are unable to break down long sugary molecules called mucopolysaccharides. This leads to the molecules building up and causing brain and organ damage, and in severe cases it can be fatal. Sufferers require regular enzyme replacement therapy to break down the molecules, but Mr Madeux said he opted to take part in a trial as he is "in pain every second of the day". The 44-year-old from Arizona underwent the treatment at UCSF Benioff Children's Hospital in Oakland, California on Monday.

Register to read more ...

Gene therapy helps a boy get new skin

Posted on: 21 November 2017, source: Biothin.Asia
A medical team at the Ruhr-Universität Bochum’s burn unit and the Center for Regenerative Medicine at the University of Modena (Italy) were the first ever to successfully treat a child suffering from extensive skin damage using transplants derived from genetically modified stem cells. The boy is a so-called butterfly child: he suffers from epidermolysis bullosa, a genetic skin disease that had destroyed approximately 80 percent of his epidermis. After all established therapies had failed, the medical team from Bochum decided to try an experimental approach: they transplanted skin derived from genetically modified stem cells onto the wound surfaces. Thanks to the successful therapy, the boy is now – two years after the treatment – able to participate in his family’s life and social life. The scientists published their report in “Nature”.

Register to read more ...

Gene Therapy in Recurrent Glioma Shows Promise in Early Trial

Posted on: 7 November 2017, source: CancerNetwork
More than 25% of patients with recurrent, high-grade glioma treated with a gene therapy combination were alive more than 3 years after treatment, according to data from a subset of patients in a phase I clinical trial presented at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics, held October 26–30 in Philadelphia. The combination treatment involved the retroviral vector Toca 511 and the prodrug of the chemotherapy 5-fluorouracil (Toca FC). Toca 511 is a replicating virus that only infects actively dividing tumor cells. Once inside the cancer cell, the virus delivers a gene for an enzyme, cytosine deaminase (CD). As the virus replicates and spreads to other cancer cells, it programs them to make CD. Next, patients receive a pill, Toca FC, which is an inert compound. Once inside the cancer cell, CD converts Toca FC into the anticancer drug 5-fluorouracil, which kills the cancer cell. 5-fluorouracil also kills certain immune suppressive myeloid cells, boosting the patient’s immune system to recognize and attack the cancer cells.

Register to read more ...

FDA approves a game-changing treatment for blood cancer

Posted on: 20 October 2017, source: StatNews
The Food and Drug Administration on Wednesday approved a promising new treatment for a particularly deadly form of cancer, bringing hope to desperate patients while rekindling a global conversation about the escalating cost of new therapies. The treatment, made by Gilead Sciences, is made by extracting patients’ white blood cells and re-engineering them to home in on tumors. Called a CAR-T, the one-time treatment has shown unprecedented results for patients with dire diagnoses. Gilead’s treatment, to be sold as Yescarta, is the second CAR-T to win FDA approval but the first cleared for use in adults. A similar therapy from Novartis, approved in August, is used for children with an aggressive form of blood cancer.

Register to read more ...

FDA experts offer a unanimous endorsement for pioneering gene therapy for blindness

Posted on: 13 October 2017, source: ScienceMag
A pioneering AAV gene therapy from Spark Therapeutics took a giant stride toward an FDA approval yesterday as an outside panel of experts offered their support for getting this game-changing treatment into the market after looking over the data and hearing from some of the severely sight-impaired patients whose lives had been transformed by this therapy. The vote was 16 to 0 favoring the benefit-risk profile of the drug, backing an OK for voretigene neparvovec by the agency’s Cellular, Tissue and Gene Therapies Advisory Committee and providing a compelling reason for the U.S. Food and Drug Administration to follow through with an historic first U.S. approval of a vector-delivered gene therapy.

Register to read more ...

DNA surgery on embryos removes disease

Posted on: 5 October 2017, source: BBC News
Precise "chemical surgery" has been performed on human embryos to remove disease in a world first, Chinese researchers have told the BBC. The team at Sun Yat-sen University used a technique called base editing to correct a single error out of the three billion "letters" of our genetic code. They altered lab-made embryos to remove the disease beta-thalassemia. The embryos were not implanted. The team says the approach may one day treat a range of inherited diseases.

Register to read more ...

Gene therapy reverses multiple sclerosis in mice

Posted on: 25 September 2017, source: BioNews
A novel gene therapy can reverse the symptoms and progression of disease in a mouse model of multiple sclerosis (MS). The neurodegenerative illness is caused by the body's own immune system degrading and destroying nerve cells. US researchers have used a gene therapy approach in the livers of affected mice to produce more regulatory T-cells and reduce this autoimmune response. 'Using a clinically tested gene therapy platform, we are able to induce very specific regulatory cells that target the self-reactive cells that are responsible for causing multiple sclerosis,' said Dr Brad Hoffman at the University of Florida, College of Medicine in Gainesville, who led the study. 'Most current therapies for autoimmune diseases such as multiple sclerosis are based on general immune suppression, which has various side effects or complications.'

Register to read more ...

Gene therapy for cancer halted after patient death

Posted on: 13 September 2017, source: BioNews
Two trials assessing gene therapy for blood cancer have been put on hold by the US Food and Drug Administration (FDA), following a patient fatality. The therapy, known as 'off-the-shelf' CAR-T immunotherapy, used genetically modified immune system T cells to target cancer, yet unlike other trials the cells were taken from a healthy donor instead of the patient. It was hoped this therapy would prove easier and less expensive to produce.

Register to read more ...

FDA makes the first gene therapy Kymriah available for treatment of pediatric ALL

Posted on: 31 August 2017, source: News Medical Life Sciences
The U.S. Food and Drug Administration (FDA) has issued a press release making the first even gene therapy available for treatment of children with a form of acute lymphoblastic leukemia (ALL). The drug Kymriah (tisagenlecleucel) is the first of its kind that uses gene therapy to treat patients with this condition. The release was updated yesterday identifying the designantions that were granted to Kymriah.

Register to read more ...

Gene Therapy Corrects Factor VIII Levels in Hemophilia A

Posted on: 7 August 2017, source: Medscape
Patients with hemophilia A who received a gene for coagulation factor VIII (FVIII) packaged in a viral vector had sustained levels of FVIII during 1 year of observation, eliminating spontaneous bleeds[2]. Most of them did not need FVIII infusions even in the case of major trauma or surgery. In a late-breaking abstract session here at the International Society on Thrombosis and Hemostasis 2017 Congress, Dr John Pasi (Barts and the London School of Medicine and Dentistry, Queen Mary University of London, UK) said six out of the seven patients in one dose cohort have achieved normal factor VIII levels.

Register to read more ...

First gene therapy — 'a true living drug' — on the cusp of FDA approval

Posted on: 14 July 2017, source: Chicago Tribune
Food and Drug Administration advisers on Wednesday enthusiastically endorsed a first-of-its-kind cancer treatment that uses patients' revved-up immune cells to fight the disease, concluding that the therapy's benefits for desperately ill children far outweigh its potentially dangerous side effects. The unanimous recommendation from the Oncologic Drugs Advisory Committee means the treatment could be approved by the FDA by the end of September, forging a new path in the immunotherapy frontier.

Register to read more ...

South Korea approves first gene therapy drug

Posted on: 12 July 2017, source: Yonhap News
South Korea's health authorities said Wednesday that they have approved the use of the country's first gene therapy drug to help patients with degenerative joint disease. The drug, Invossa, manufactured by local drug firm Kolon Life Science Inc. has obtained the green light from the Ministry of Food and Drug Safety for sales in the domestic market. Invossa is the world's first cell-medicated gene therapy for osteoarthritis, a disorder involving movable joints. The company said the drug can be injected into the joint cavity of a patient. The drug has proven to reduce osteoarthritis pain and improve cartilage through clinical trials, said Kolon Life Science, expecting to help an estimated five million patients in the country.

Register to read more ...

Gene Therapy & Mesothelioma

Posted on: 18 June 2017, source: mesothelioma.com
Technological innovations are changing the world we live in every day, including the way society views diseases that have been around for hundreds of years. Cancer continues to be a leading cause of death around the world, but day by day we, as a society, are getting closer to finding a cure. In 2016, former President Barack Obama asked former Vice President Joe Biden to head the Cancer Moonshot Initiative (CMI), whose overarching goal would be to find a cure for cancer by the year 2020. In the time that’s passed, Biden has formed a committee of governmental organizations and a Blue Ribbon Panel that provided recommendations of how to achieve the lofty goals, and set to bringing this moonshot into reality.

Register to read more ...

Altered virus may expand patient recruitment in human gene therapy trials

Posted on: 14 June 2017, source: Science Daily
For many patients, participating in gene therapy clinical trials isn't an option because their immune system recognizes and fights the helpful virus used for treatment. Now, University of Florida Health and University of North Carolina researchers have found a solution that may allow it to evade the body's normal immune response.

Register to read more ...

Allergy Breakthrough with Gene Therapy

Posted on: 8 June 2017, source: World Health
It might soon be possible for a single treatment to provide life-long protection against harsh allergies including asthma. An immunology research team at The University of Queensland led by Professor Ray Steptoe has figured out how to disable the immune response that triggers allergic reactions. The research team operates out of the university's Diamantina Institute. Professor Steptoe's lab is situated at the Translational Research Institute. The research was funded by the National Health and Medical Research Council and the Asthma Foundation. The research team's findings are published in JCI Insight.

Register to read more ...

European Child Receives Strimvelis Gene-Therapy Treatment

Posted on: 3 May 2017, source: Rare Disease Report
Before March, only 1 patient had ever been treated via commercial gene therapy. GlaxoSmithKline (GSK) doubled that total with their announcement this week. In 2015, a European patient with familial chylomicromenia received Glybera to restore their LPL enzyme levels. On Tuesday, GSK announced that it treated its first patient in March, almost a full calendar year after their drug, Strimvelis, was approved for sale. Strimvelis is assumed to provide an outright cure for Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency (ADA-SCID), a rare disorder caused by the absence of the essential protein adenosine deaminase (ADA). ADA is required to produce lymphocytes. It leaves babies without a fully-functioning immune system and extremely vulnerable to infections.

Register to read more ...

uniQure Announces It Will Not Seek Marketing Authorization Renewal for Glybera in Europe

Posted on: 20 April 2017, source: UniQure
uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will not pursue the renewal of the Glybera® (alipogene tiparvovec) marketing authorization in Europe when it is scheduled to expire on October 25, 2017. "The decision to not pursue marketing authorization renewal of Glybera in Europe involved a thoughtful and careful evaluation of patient needs and the clinical use of the therapy, and is not related to any risk-benefit concern," stated Matthew Kapusta, chief executive officer of uniQure. "Glybera's usage has been extremely limited and we do not envision patient demand increasing materially in the years ahead."

Register to read more ...

Interview with Daniel Leonard, Director, Global Patient Advocacy, UniQure

Posted on: 11 April 2017, source: Gene Therapy for Rare Disorders 2017
Daniel Leonard is the Director of Global Patient Advocacy at uniQure, a pioneering company in gene therapy. Prior to joining uniQure, Dan spent 16 years at Genzyme, a company that was known as a leader in patient centricity. In his current role Dan works primarily with hemophilia organizations in the US and abroad. In this role Dan serves as an ambassador to the patient community, but also as an internal advocate for patients’ interests. Daniel recently sat down with David Snowdon, Program Director of Gene Therapy for Rare Disorders to discuss his work in this space and how he sees the field evolving. Full interview.

Register to read more ...