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.
Paying for gene therapy: Are annuities the next big thing?
Posted on: 21 February 2015, source: St. Louis News
As U.S. drugmakers face growing resistance to the high price of cutting-edge treatments, a handful of companies is working on a new payment model that rewards them for the long-term performance of their medicines. The effort, industry executives say, is being led by firms developing so-called gene therapies, which aim to cure inherited diseases like hemophilia by “fixing” the single faulty gene responsible for the disorder. They include BioMarin Pharmaceutical Inc in San Rafael, Calif., and Sangamo BioSciences Inc in Richmond, Calif.
If these new hemophilia drugs and others like them succeed, a one-time infusion could replace the need for frequent, life-long injections of blood clotting proteins that can cost up to $300,000 a year for a single patient. These existing treatments, including Pfizer Inc.’s Xyntha and Baxter International’s Advate, are expected to command annual sales of more than $11 billion by 2016.
As U.S. drugmakers face growing resistance to the high price of cutting-edge treatments, a handful of companies is working on a new payment model that rewards them for the long-term performance of their medicines. The effort, industry executives say, is being led by firms developing so-called gene therapies, which aim to cure inherited diseases like hemophilia by “fixing” the single faulty gene responsible for the disorder. They include BioMarin Pharmaceutical Inc in San Rafael, Calif., and Sangamo BioSciences Inc in Richmond, Calif.
If these new hemophilia drugs and others like them succeed, a one-time infusion could replace the need for frequent, life-long injections of blood clotting proteins that can cost up to $300,000 a year for a single patient. These existing treatments, including Pfizer Inc.’s Xyntha and Baxter International’s Advate, are expected to command annual sales of more than $11 billion by 2016.
Innovative nanoparticle gene therapy system eliminates cancerous brain cells
Posted on: 5 February 2015, source: Johns Hopkins
Despite decades of surgery, chemotherapy and radiation therapy treaments for glioma, a cure for this life-threatening brain cancer has remained elusive. In a study published on the website of the journal ACS Nano, BME Associate Professor Jordan Green and other Johns Hopkins researchers have successfully used compound-filled biodegradable nanoparticles to effectively kill brain cancer cells — and extend survival in rats.
The biodegradable nanoparticles filled with a DNA-encoded enzyme, herpes simplex virus type 1 thymidine kinase (HSVtk), proved to be potent in killing brain cancer cells. When researchers combined this with the compound ganciclovir, the loaded nanoparticles were 100 percent effective at killing glioma cells grown in laboratory dishes.
Despite decades of surgery, chemotherapy and radiation therapy treaments for glioma, a cure for this life-threatening brain cancer has remained elusive. In a study published on the website of the journal ACS Nano, BME Associate Professor Jordan Green and other Johns Hopkins researchers have successfully used compound-filled biodegradable nanoparticles to effectively kill brain cancer cells — and extend survival in rats.
The biodegradable nanoparticles filled with a DNA-encoded enzyme, herpes simplex virus type 1 thymidine kinase (HSVtk), proved to be potent in killing brain cancer cells. When researchers combined this with the compound ganciclovir, the loaded nanoparticles were 100 percent effective at killing glioma cells grown in laboratory dishes.
U.K. Parliament approves controversial three-parent mitochondrial gene therapy
Posted on: 4 February 2015, source: Sciencemag
The United Kingdom’s House of Commons voted overwhelmingly today to allow British researchers to pursue a new fertility treatment that could prevent certain kinds of genetic diseases. The technique, called mitochondrial DNA replacement therapy, could allow women who carry disease-causing mutations in their mitochondrial genes to give birth to genetically related children free of mitochondrial disease.
The measure, which passed 382 to 128, has been controversial, especially because it would alter the DNA of an embryo in a way that could be passed on to future generations. Some scientists and nongovernmental organizations have argued that not enough is known about possible side effects of the technique to go forward in human patients. “We believe the House of Commons has made a serious mistake, which we hope does not have dire consequences,” said Marcy Darnovsky, executive director of the Center for Genetics and Society in Berkeley, California, in a statement.
The United Kingdom’s House of Commons voted overwhelmingly today to allow British researchers to pursue a new fertility treatment that could prevent certain kinds of genetic diseases. The technique, called mitochondrial DNA replacement therapy, could allow women who carry disease-causing mutations in their mitochondrial genes to give birth to genetically related children free of mitochondrial disease.
The measure, which passed 382 to 128, has been controversial, especially because it would alter the DNA of an embryo in a way that could be passed on to future generations. Some scientists and nongovernmental organizations have argued that not enough is known about possible side effects of the technique to go forward in human patients. “We believe the House of Commons has made a serious mistake, which we hope does not have dire consequences,” said Marcy Darnovsky, executive director of the Center for Genetics and Society in Berkeley, California, in a statement.
Controlled Gene Therapy May be Possible with New Method
Posted on: 11 January 2015, source: Bionews Texas
Gene therapy works by introducing genetic material (either DNA or RNA) into cells with a viral vector. This may be to replace a defective gene, but can also be to increase levels of a beneficial molecule that can compensate for the disease state. Most people have negative reactions when they think about viruses, but viral vectors are useful for injecting genetic material into cells. In research, and ultimately in the clinic, cells can then be controlled to churn out desired molecules for treating diseases. The use of viral vectors has held promise for gene therapy for many years, with the first gene therapy approved in the European Union in 2012. Despite gene therapy’s promise, researchers are still trying to overcome the limitations associated with this technology. Controlling where and how much of the gene is delivered to cells remains a formidable challenge. A new study, published in the journal Nucleic Acids Research in December 2014, may hold promise for enabling controlled gene therapy.
Gene therapy works by introducing genetic material (either DNA or RNA) into cells with a viral vector. This may be to replace a defective gene, but can also be to increase levels of a beneficial molecule that can compensate for the disease state. Most people have negative reactions when they think about viruses, but viral vectors are useful for injecting genetic material into cells. In research, and ultimately in the clinic, cells can then be controlled to churn out desired molecules for treating diseases. The use of viral vectors has held promise for gene therapy for many years, with the first gene therapy approved in the European Union in 2012. Despite gene therapy’s promise, researchers are still trying to overcome the limitations associated with this technology. Controlling where and how much of the gene is delivered to cells remains a formidable challenge. A new study, published in the journal Nucleic Acids Research in December 2014, may hold promise for enabling controlled gene therapy.
Gene Therapy 3.0: Rise and fall and rise again of gene therapy–For real this time?
Posted on: 18 December 2014, source: Genetic Literacy Project
Gene therapy is back in the news, and in a big way as regular readers of the GLP would know. Studies involving the use of gene therapy are showing promising results for the cure of blood disorders, ‘bubble boy’ disease and HIV among others. Industry interest has also picked up and as positive results from clinical trials roll in, the market suddenly appears bullish on the future. But are we seeing enough to suggest that the technology is promising enough to make a major contribution to public health? Why does gene therapy have to be ‘back’ in the first place?
We offer a quick recap here, but for a more detailed read on the rise and fall (and rise again) of gene therapy, read this excellent narrative by Carl Zimmer in Wired or this comprehensive feature by Laura Cassiday in Nature. Things looked bright for gene therapy in the 90s when it promised to be a revolution that would let us move from just treating genetic diseases to curing them permanently. The big question surrounding gene therapy had always been how to effectively deliver the correct form of the gene into cells.
Gene therapy is back in the news, and in a big way as regular readers of the GLP would know. Studies involving the use of gene therapy are showing promising results for the cure of blood disorders, ‘bubble boy’ disease and HIV among others. Industry interest has also picked up and as positive results from clinical trials roll in, the market suddenly appears bullish on the future. But are we seeing enough to suggest that the technology is promising enough to make a major contribution to public health? Why does gene therapy have to be ‘back’ in the first place?
We offer a quick recap here, but for a more detailed read on the rise and fall (and rise again) of gene therapy, read this excellent narrative by Carl Zimmer in Wired or this comprehensive feature by Laura Cassiday in Nature. Things looked bright for gene therapy in the 90s when it promised to be a revolution that would let us move from just treating genetic diseases to curing them permanently. The big question surrounding gene therapy had always been how to effectively deliver the correct form of the gene into cells.
Regulatory landscape of advanced-therapy medicinal products
Posted on: 14 December 2014, source: lexology.com
Advanced therapy medicinal products (ATMPs) are a category of innovative products comprising gene-therapy medicinal products (GTMPs),somatic cell-therapy medicinal products (sCTMP) and tissue-engineered products (TEPs). The main therapeutic areas are oncology and regenerative medicine, particularly in the field of cardiovascular conditions and haematology. Yet despite the harmonising EU legislation which has been in place since 2008, few products have reached the market in commercial form. In 2014 the European Commission issued a report on the implementation of ATMP legislation to date. On June 30 2014 the European Medicines Agency Committee for Advanced Therapies announced a public consultation on its revised guidance for ATMP classification. The consultation ended on October 31 2014.
Advanced therapy medicinal products (ATMPs) are a category of innovative products comprising gene-therapy medicinal products (GTMPs),somatic cell-therapy medicinal products (sCTMP) and tissue-engineered products (TEPs). The main therapeutic areas are oncology and regenerative medicine, particularly in the field of cardiovascular conditions and haematology. Yet despite the harmonising EU legislation which has been in place since 2008, few products have reached the market in commercial form. In 2014 the European Commission issued a report on the implementation of ATMP legislation to date. On June 30 2014 the European Medicines Agency Committee for Advanced Therapies announced a public consultation on its revised guidance for ATMP classification. The consultation ended on October 31 2014.
Correcting the genetic error in sickle-cell disease might be as simple as amending text
Posted on: 15 November 2014, source: Nature
Tiny changes in DNA can have huge consequences. For years, scientists have been trying to 'fix' these mutations in the hope of treating and potentially curing some of humanity's most devastating genetic diseases. After some tragic early setbacks (see Nature 420, 116–118; 2002), techniques that allow precise genetic manipulation have created a surge of research.
Although most existing treatments for genetic diseases typically only target symptoms, genetic manipulation or 'gene therapy' goes after the cause itself. The approach involves either inserting a functional gene into DNA or editing a faulty one that is already there, so the conditions most likely to prove curable are those caused by a single mutation. Sickle-cell disease is a perfect candidate: it is caused by a change in just one amino acid at a specific site in the β-globin gene. This results in the production of abnormal haemoglobin proteins that cause the red blood cells that house them to twist and become sickle shaped. The distorted cells get sticky, adhere to each other and block blood vessels, preventing oxygenated blood from flowing through
Tiny changes in DNA can have huge consequences. For years, scientists have been trying to 'fix' these mutations in the hope of treating and potentially curing some of humanity's most devastating genetic diseases. After some tragic early setbacks (see Nature 420, 116–118; 2002), techniques that allow precise genetic manipulation have created a surge of research.
Although most existing treatments for genetic diseases typically only target symptoms, genetic manipulation or 'gene therapy' goes after the cause itself. The approach involves either inserting a functional gene into DNA or editing a faulty one that is already there, so the conditions most likely to prove curable are those caused by a single mutation. Sickle-cell disease is a perfect candidate: it is caused by a change in just one amino acid at a specific site in the β-globin gene. This results in the production of abnormal haemoglobin proteins that cause the red blood cells that house them to twist and become sickle shaped. The distorted cells get sticky, adhere to each other and block blood vessels, preventing oxygenated blood from flowing through
Popular lectures on gene therapy
Posted on: 6 November 2014, source: ieet.org
Maria Konovalenko and team put together a list of popular science video lectures on gene therapy – one of the most promising molecular medicine directions. What makes this approach different is that nucleic acid molecules, DNA and RNA, are used as therapeutic agents.
Maria Konovalenko and team put together a list of popular science video lectures on gene therapy – one of the most promising molecular medicine directions. What makes this approach different is that nucleic acid molecules, DNA and RNA, are used as therapeutic agents.
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Recent early view article in Journal of Gene Medicine:
Recent early view article in Journal of Gene Medicine: