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Explainer: what is gene therapy?

Posted on: 28 November 2013, source: Science Alert
Every now and again you might read about gene therapy and efforts to correct serious genetic diseases. But I’m betting that very few readers have had gene therapy, nor have they ever met anyone who has, nor will they ever meet that many. The reasons are simple – while these procedures are possible in theory, in practice, it has proved remarkably difficult to insert new genetic materials into human patients and ensure that the new genes are sustainably expressed. The difficulties encountered highlight the fact that we do not yet have enough fundamental knowledge about gene transfer and control. And what’s more, gene therapy is hugely expensive.

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Q&A: Gene Therapy the Fix for Faulty DNA

Posted on: 27 November 2013, source: ModVive.com
Inside (almost) all of our cells are 23 pairs of chromosomes, long strands of DNA that are packaged tightly together. Each chromosome houses hundreds of genes, each of which is the blueprint for at least one protein. Little messengers called mRNA make copies of the gene blueprints, edits them and carry them to “builders” called ribosomes. The ribosomes read the copy and translate it into long chains of amino acids that are then folded into unique shapes. These globs of amino acids are proteins, that travel throughout our entire body and are in some way vital for everything that happens inside of us. For example, helicase and chromatin are proteins essential to cell division, what allows us to grow and heal, and immunoglobulin, also called antibodies, protect us from infections.

What happens when something goes wrong with our DNA and thus our proteins?

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UK's first gene therapy trial for patients with chronic heart failure begins at GJNH

Posted on: 21 November 2013, source: News-Medical
The UK’s first gene therapy trial for advanced heart failure, CUPID 2, has officially begun at the Golden Jubilee National Hospital (GJNH); with the first candidate recently being administered with a dose of the MYDICAR treatment. The GJNH, home of the Scottish National Advanced Heart Failure Service (SNAHFS), announced in April 2013 that it would be participating in the international trial, helping gather a total of 200 patients from 50 institutions worldwide, to determine the effectiveness of the gene therapy treatment, MYDICAR, in advanced heart failure patients.

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Gene therapy company launched

Posted on: 1 November 2013, source: Boston Globe
Fidelity Biosciences, a venture capital firm that is a subsidiary of Fidelity Investments, and REGENX Biosciences, announced the formation of Dimension Therapeutics, a Cambridge gene therapy company focused on developing treatments for rare diseases such as hemophilia. Dimension has completed an undisclosed Series A financing that was led by Fidelity Biosciences.

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Several gene therapies are or will soon be in late-stage human trials

Posted on: 2 October 2013, source: NextBigFuture
Though many gene therapies have been tested in patients around the world in hopes of curing hereditary diseases, few governments have approved their sale, and none has been approved in the United States. That could change in coming years as several therapies enter advanced trials. Experts say it is likely to be a few years before a treatment is approved in the U.S. With its European approval in hand, UniQure may have good chance of also getting the first U.S. approval, but the company says it has not yet submitted an application to the FDA.

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Vaccine appears to clear HIV-like virus in monkeys

Posted on: 14 September 2013, source: Science Recorder
Researchers at Oregon Health and Science University (OHSU) have developed a candidate vaccine that appears to completely clear an AIDS-causing virus from the body. Developed at OHSU’s Vaccine and Gene Therapy Unit, the vaccine is now being tested through a non-human primate form of HIV, called simian immunodeficiency virus (SIV). It is hoped that an HIV-form of the vaccine will soon be tested on humans.

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Gene therapy repairs heart attack damage

Posted on: 29 August 2013, source: BioNews
Researchers have concocted a 'gene cocktail' that may help to turn scar tissue into functioning cardiac cells after a heart attack. Scar tissue often forms following a heart attack in the area that was most affected. The result is a decreased ability of the heart to beat properly and can eventually lead to heart failure. The scientists, from the Gladstone Institutes in San Francisco, USA, are working with cells called fibroblasts, which make up about half of all heart cells. They are aiming to reprogram the function of these fibroblasts from primarily providing structural support to beating heart cells by injecting a cocktail of genes.

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First Gene Therapy For Rett Syndrome, An Autism Spectrum Disorder

Posted on: 21 August 2013, source: Medical Daily
Scientists in Oregon have developed a new gene therapy for Rett Syndrome, the most disabling of the autism spectrum disorders. Field-tested in mice, it is the first therapy to reverse the course of the disorder in a fully symptomatic model, which represents an important step towards clinical application. Nearly one in 10,000 kids are born each year with Rett syndrome. Neurological and behavioral features, like loss of speech, immobility, and seizures, typically appear after six months of age. The most frequent cause is the mutation of an X chromosome gene called MECP2, which explains why girls are primarily affected by the condition.

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Rules for Gene-therapy Vectors

Posted on: 14 August 2013, source: Health News
Rice University researchers are making strides toward a set of rules to custom-design Lego-like viral capsid proteins for gene therapy. A new paper by Rice scientists Junghae Suh and Jonathan Silberg and their students details their use of computational and bioengineering methods to combine pieces of very different adeno-associated viruses (AAVs) to create new, benign viruses that can deliver DNA payloads to specific cells. The research appears this month in the American Chemical Society journal ACS Synthetic Biology.

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Wiskott-Aldrich Syndrome: Lentivirus Gene Therapy Helps Three Kids

Posted on: 14 July 2013, source: Science 2.0
An international team that developed a new gene therapy approach to treatment of Wiskott-Aldrich Syndrome, a fatal inherited form of immunodeficiency has reported results. Wiskott-Aldrich Syndrome is a disorder that weakens the body's immune system. It is caused by a mutation in a gene that encodes the protein WASP. The most often used therapy is a bone marrow or stem cell transplant from a matching donor, often a sibling or relative. It can be curative for some patients, mostly those who have a strongly matching donor.

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WADA Symposium Covers Broad Spectrum of Gene Doping, and Potential for Misuse of Cell Therapy in World of Sport

Posted on: 7 June 2013, source: WADA
Leading experts in gene therapy, scientists from the field of anti-doping, and sport ethicists joined WADA’s Gene Doping Panel at the Agency’s Fourth Gene and Cell Doping Symposium, held June 5-6 in Beijing to review recent achievements in the field and share the most promising approaches in doping detection under development. Organized with the support of the China Anti-Doping Agency (CHINADA) and Beijing Olympic City Development Association (BODA), the Symposium attracted more than 70 international participants. Sixteen invited speakers included international experts who either study the latest advances in the field of gene therapy and gene doping detection methods, or are involved in related WADA-funded research projects. The 2013 Symposium was the first to dedicate a formal session to cell therapy and doping.

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Public consultation on advanced therapy medicinal products: A summary of the responses

Posted on: 30 May 2013, source: European Commission
The European Commission should prepare a report on the application of Regulation (EC) No 1394/2007 on advanced therapy medicinal products. With a view to prepare this report, the Commission services launched a public consultation on 20 December 2012. Stakeholders were invited to provide their views on the requirements to obtain a marketing authorisation for an ATMP (including for combined ATMPs), on the application of the so-called hospital exemption, on the incentives provided for in the ATMP Regulation, as well as on the scope thereof and its adaptation to technical progress. The number of contributions received was 63.

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Scientists reveal inside story of European regulatory review and approval of Glybera

Posted on: 23 May 2013, source: News Medical
The scientists who led the team that developed Glybera, the first gene therapy drug approved for use in the Western world, provide a fascinating first-person account of their pioneering work in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. In addition, an in-depth Review revealing the inside story of the European regulatory review and approval of Glybera—chock full of twists and turns, politics, and intrigue— and critiquing the groundbreaking drug's path to the marketplace appears in Human Gene Therapy Clinical Development. Both articles are available free on the Human Gene Therapy website.

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EMA Consultation: Draft guideline on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy

Posted on: 13 May 2013, source: EMA
The European Medicines Agency has recently published a draft guideline on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy. Recent advances in basic and clinical research have opened new perspectives for future therapeutic options in Duchenne and Becker muscular dystrophy (DBMD). As most of the cases of Duchenne muscular dystrophy (DMD) have an onset in early childhood, while the onset of Becker muscular dystrophy (BMD) covers a broader age spectrum, specific difficulties have been identified that pertain to diagnostic criteria, age- and stage related clinical relevance and different safety aspects.

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Netherlands Gene Therapy Office launches website

Posted on: 12 May 2013, source: Gene Therapy Office
The aim of the governmental Gene Therapy Office is to streamline the licensing and permit granting procedures for clinical gene therapy studies in The Netherlands and to provide more insight into the procedures for investigators. The Gene Therapy Office is primarily a service for professionals conducting clinical gene therapy research involving human subjects. The official website is www.loketgentherapie.nl.

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T-Cell Therapy for ALL Shows Benefit

Posted on: 28 March 2013, source: MedPageToday
Researchers reported encouraging but preliminary results of gene-based immune therapy in two children with relapsed and refractory acute lymphoblastic leukemia (ALL). Both children achieved a remission after they were given T cells modified to attack B cells bearing the surface protein CD19, according to Stephan A. Grupp, MD, PhD, of the Children's Hospital of Philadelphia, and colleagues.

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Lentigen wins US Orphan Drug Designation for glioblastoma

Posted on: 28 February 2013, source: Lentigen
Lentigen Corporation, a biotechnology company specializing in the development and manufacture of lentiviral gene delivery products, announced today that that the U.S. Food and Drug Administration (FDA) has granted orphan drug status to P140K methylguanine methyltransferase (MGMT) transduced human CD34 cells (product name: LG631-CD34) for bone marrow protection in the treatment of glioblastoma multiforme. Orphan drug designation qualifies Lentigen for seven years of market exclusivity following marketing approval by the FDA and provides other development-related incentives.

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Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases

Posted on: 5 February 2013, source: Molecular Therapy
In the latest issue of the journal Molecular Therapy, Professor Jacques P. Tremblay (president of the Association of Gene Therapy of Quebec and researcher in the Research center of the Centre Hospitalier Universitaire (CHU) of Quebec) launches a call - with 50 other world experts in gene therapy - for the creation of an International Gene Therapy Consortium for Monogenic Diseases. The bases of this consortium will be established during a workshop, which will be held during the congress of the American Society of Gene and Cell Therapy (ASGCT) in Salt Lake City in May 2013.

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Another Bump in the Road to Gene Therapy?

Posted on: 1 February 2013, source: PlosBlogs
By Ricki Lewis - I am astonished, once again, by the complexity and unpredictability of science. Last week, a paper in the Proceedings of the National Academy of Science (PNAS) reported that gene therapy to treat a form of blindness called Leber congenital amaurosis type 2 (LCA2) doesn’t stop degeneration of the rods and cones – the photoreceptor cells that provide vision. Gene therapy sends genetic instructions for a protein called RPE65 into a layer of cells that supports the rods and cones – the retinal pigment epithelium, or RPE. The protein is essential for the eye to use vitamin A. And the gene therapy works, so far.

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New Gene Therapy Extends Lives Of Children With Rare Brain Disorder

Posted on: 24 January 2013, source: Sinq
Chalk up another victory for gene therapy. A recent clinical trial has shown that gene therapy can be used to extend the lives of children with a rare brain disorder that typically proves fatal in the first few years of life. The oldest child to benefit from this reversal of fortune has reached an amazing 18 years of age.

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Positive gene therapy results in large mammals of Duchenne muscular dystrophy

Posted on: 17 January 2013, source: NewsMedical
Usually, results from a new study help scientists inch their way toward an answer whether they are battling a health problem or are on the verge of a technological breakthrough. Once in a while, those results give them a giant leap forward. In a preliminary study in a canine model of Duchenne muscular dystrophy (DMD), University of Missouri scientists showed exactly such a leap using gene therapy to treat muscular dystrophy. The results of the study will be published in the journal Molecular Therapy on Jan. 15, 2013.

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MIT researchers crack cheap, precise gene therapy

Posted on: 11 January 2013, source: ExtremeTech
Disease is on the run! An incredible advance in the realm of gene therapy has been made by top researchers in the US and Sweden. A single enzyme from a bacteria’s immune system can effectively be used to locate specific sections of DNA in a living cell. From there, entire genes can be neutralized or replaced with superior sections of DNA. Clearly, we are on the brink of gene therapy becoming a common and highly effective treatment for terrible diseases like AIDS and cancer.

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Shape matters in DNA nanoparticle therapy

Posted on: 14 December 2012, source: National Cancer Institute
Researchers from The Johns Hopkins University and Northwestern University have discovered how to control the shape of nanoparticles that move DNA through the body and have shown that the shapes of these carriers may make a big difference in how well they work in treating cancer and other diseases. The results of this study, which were published in the journal Advanced Materials ("Plasmid-Templated Shape Control of Condensed DNA–Block Copolymer Nanoparticles"), are also noteworthy because this gene therapy technique does not use a virus to carry DNA into cells. “These nanoparticles could become a safer and more effective delivery vehicle for gene therapy targeting cancer and other illnesses than can be treated with gene medicine,” said Hai-Quan Mao, of Johns Hopkins, who, together with Erik Luijten of Northwestern, led this research.

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The CliniBook: clinical gene transfer state-of-the-art

Posted on: 23 November 2012, source: CliniGene
This book provides a reference for state-of-the-art gene transfer technology as of 2012 and the different aspects of its clinical translation with a focus on European-based initiatives. As examples of successful outcomes, recent clinical trials are presented together with ethical, safety and legal issues, which are discussed. This book is ideal for researchers, clinical investigarors, post-graduate fellows, undergraduate scholars, regulators, patients' advocacy groups and policy-makers, looking for state-of-the-art information as well as emerging prospects - including gene targeting and homologous recombination - for gene transfer intended for clinical translation. List of authors and table of contents.

Gene Therapy Net members can order now for a reduced price of 80 € (public price: 95 €).

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uniQure's Glybera® First Gene Therapy Approved by European Commission

Posted on: 2 November 2012, source: uniQure
uniQure announced today it has received approval from the European Commission for the gene therapy Glybera® (alipogene tiparvovec), a treatment for patients with lipoprotein lipase deficiency (LPLD, also called familial hyperchylomicronemia) suffering from recurring acute pancreatitis. Patients with LPLD, a very rare, inherited disease, are unable to metabolize the fat particles carried in their blood, which leads to inflammation of the pancreas (pancreatitis), an extremely serious, painful, and potentially lethal condition. The approval makes Glybera the first gene therapy approved by regulatory authorities in the Western world.

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DNA folded into shapes offers alternative gene delivery vehicle

Posted on: 18 October 2012, source: Nanowerk News
Researchers from Johns Hopkins and Northwestern universities have discovered how to control the shape of nanoparticles that move DNA through the body and have shown that the shapes of these carriers may make a big difference in how well they work in treating cancer and other diseases. This study, published in the Oct. 12 online edition of Advanced Materials is also noteworthy because this gene therapy technique does not use a virus to carry DNA into cells. Some gene therapy efforts that rely on viruses have posed health risks.

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EMA Streamlines Working Parties

Posted on: 25 September 2012, source: PharmTech
The European Medicines Agency (EMA) has abolished its Cell-based Product Working Party (CPWP) and Gene Therapy Working Party (GTWP), with the aim of improving efficiencies and optimizing the use of available expertise. Instead, EMA’s Committee for Advanced Therapies (CAT) will be assuming the lead role in all aspects concerning the development of advanced-therapy medicines in Europe, including developing guidelines, organizing workshops and establishing ad-hoc draft groups as and when required to develop specific guidance documents. Guidance documents already being developed by CPWP or GTWP will be taken over by the new drafting groups

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Researchers Improve Gene Therapy Technique for Children with Immune Disorder

Posted on: 12 September 2012, source: Healthcare Globa
Researchers enhance gene therapy approach for children with adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID) using chemotherapy as conditioning regimen. For the first time, investigators test two different viral vectors to transport normal genes into the young patients' bone marrow stem cells and two different treatment plans in preparation for receiving gene therapy and compare outcomes.

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Scientists restore sense of smell in mice through gene therapy

Posted on: 3 September 2012, source: News-Medical
Scientists have restored the sense of smell in mice through gene therapy for the first time -- a hopeful sign for people who can't smell anything from birth or lose it due to disease. The achievement in curing congenital anosmia -- the medical term for lifelong inability to detect odors -- may also aid research on other conditions that also stem from problems with the cilia. Those tiny hair-shaped structures on the surfaces of cells throughout the body are involved in many diseases, from the kidneys to the eyes. The new findings, published online in Nature Medicine, come from a team at the University of Michigan Medical School and their colleagues at several other institutions

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The cheat gene: Could the next step in sporting fraud come from manipulating DNA?

Posted on: 22 August 2012, source: The Independent
The London Olympics may turn out to be one of the cleanest in history in terms of banned substances – but behind the scenes, scientists fear the next big challenge to fair play in sport: gene doping. Over the past decade, scientific advances in the understanding of how genes control muscle activity have alarmed experts within the World Anti-Doping Agency (Wada) who believe that using genes, rather than drugs, will be the next way illicitly to boost athletic performance without fear of detection.

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