Posted on: 16 February 2012, source: ACGT Foundation
The Alliance for Cancer Gene Therapy, Inc. (ACGT) funds research aimed at furthering the development of gene therapy approaches to the treatment of cancer. To this end, ACGT offers awards in Clinical Translation of Gene Therapy for Cancer, to qualified scientists at the assistant professor level and above. This award is specifically for those conducting Gene Therapy Research for Pancreatic Cancer and is entitled, 2012 Investigator’s Award in Clinical Translation of Gene Therapy for Pancreatic Cancer. Abstract Deadline: March 7, 2012

Posted on: 3 February 2012, source: NVGCT
Since gene and cell therapy are closely related and actually intertwined scientific disciplines aiming at expanding and innovating the therapeutic arsenal for many so far incurable diseases, the Netherlands Society of Gene and Cell Therapy (NVGCT) now includes cell therapy in its name. Its new web site (www.nvgct.nl), launched February 1, 2012, aims at fostering further development and communicating progress.

Posted on: 30 January 2012, source: PRNewswire
Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, was informed on Friday January 27, 2012 after business hours that the European Commission's Standing Committee of the European Parliament discussed on Monday January 22, 2012 the implementation decision not to grant marketing authorization for Glybera (alipogene tiparvovec) as recommended by the Committee for Human Medicinal Products (CHMP). After the discussion no clear position in favor or against granting a marketing authorization for Glybera was reached. Instead the Standing Committee considered it necessary to request additional information to the CHMP in the European Medicines Agency (EMA). A formal vote by the Standing Committee will be made on review of the additional information. It is currently unclear when a final decision will be reached.

Posted on: 21 December 2011, source: Human Stem Cell Institute (HSCI), Dr. Isaev
The Human Stem Cells Institute (“HSCI”, MICEX: ISKJ), Russia’s biotech company, announced today that it has received state approval for Neovasculgen® – the first Russian gene therapy drug to treat Peripheral Arterial Disease. The approval was received by HSCI on December 7, 2011, with the decision by the Russian Ministry of Healthcare and Social Development to include Neovasculgen® in the State Registry of Medications dating back to September 28, 2011. The decision was based on findings on the drug’s efficacy and safety reached during the pre-clinical and clinical trials carried out by HSCI. The Phase IIB/III clinical trials for Neovasculgen® were completed in Q2 2011, and in July HSCI submitted the relevant documentation to the Ministry for approval.

Posted on: 12 December 2011, source: Reuters
A single treatment with gene therapy, an experimental technique for fixing faulty genes, has been shown to boost output of a vital blood clotting factor, possibly offering a long-term solution for people with hemophilia B. Researchers said the same technology was also being studied as a treatment for hemophilia A, the far more common type of the inherited bleeding disorder.

Posted on: 23 November 2011, source: CLinigene
On the CliniGene website you can find a 20 minutes video on Gene Therapy called: Gene Therapy a new tool to cure human diseases. This video is available directly at the following address: https://www.youtube.com/watch?v=U3RygvuSrok. This video has been designed by Fatima Bosch, Carles Roca, Xavier Anguela and Albert Ruzo and is property of the Center for Animal Biotechnology and Gene Therapy of the Universitat Autónoma de Barcelona (UAB). The design of this video has been supported by CliniGene.

Posted on: 25 October 2011, source: AMT
Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today provided an update on operations and disclosed the outcome of a strategic review of its gene therapy product pipeline conducted by management and AMT's Board of Directors that enables the company to remain at the forefront of gene therapy development. The CHMP's recent opinion, while not approving Glybera®, did not identify any safety risks with AMT's adeno-associated virus vector technology. This will allow AMT to leverage its expertise and strength in building other portfolio products. The selection of specific pipeline products is aimed at securing partnering agreements that will provide solid financial foundations to take the company forward. Existing shareholders continue to see promise in the technology and are in discussions with AMT to provide additional equity.

Posted on: 21 October 2011, source: PRNewswire
Amsterdam Molecular Therapeutics (AMT), a leader in the field of human gene therapy, today announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has maintained its earlier opinion that Glybera (alipogene tiparvovec) is not approvable at this time. The decision was made contrary to the positive recommendation of CHMP Rapporteurs, the Scientific Advisory Group (SAG), an expert panel specifically selected to evaluate clinical results and the science of the product, and the Committee for Advanced Therapies (CAT), which provides guidance on advanced therapeutics such as gene and cell therapy.

Posted on: 13 October 2011, source: BBC news
Two of the holy grails of medicine - stem cell technology and precision gene therapy - have been united for the first time in humans, say scientists. It means patients with a genetic disease could, one day, be treated with their own cells. A study in Nature corrected a mutation in stem cells made from a patient with a liver disease. Researchers said this was a "critical step" towards devising treatments, but safety tests were still needed.

Posted on: 7 October 2011, source: Science
It has taken many years, but researchers may have reached a prized goal in gene therapy: lowering the risk of uncontrolled bleeding in patients with hemophilia. At a meeting last week, researchers reported that six patients who received a virus engineered to carry a gene for a blood-clotting protein called factor IX needed fewer transfusions of the protein for as long as 18 months; some didn't require any transfusions. One patient developed an immune response to the viral vector, but this side effect was successfully treated with drugs.

Posted on: 19 September 2011, source: Reuters
An early stage trial of Sangamo BioSciences Inc's HIV treatment found that the gene therapy reduced levels of the virus and even eliminated it in one patient with a naturally occurring gene mutation. The very small Phase 1 trial tested the SB-728-T gene therapy, which is designed to disrupt the CCR5 gene used by HIV to infect cells of the immune system. If shown to be safe and effective, the treatment could end the need for the antiretroviral drugs now used to keep the virus that causes AIDS in check by suppressing viral replication in the blood.

Posted on: 25 August 2011, source: Gizmodo Australia
It’s been a big month for gene therapy: first a breakthrough for leukaemia last week, now today scientists announced they’ve successfully treated kids with “bubble boy” disease. We’ve heard this before — back in 2000 gene therapy successfully treated the same immune disease, but by 2007 five out of the 10 boys had developed leukaemia (ironically, the disease successfully treated in the study announced last week), and one died. This time, the researchers smartly waited NINE YEARS to declare success with 14 out of 16 boys. It’s hard to argue with nearly a decade of good health.

Posted on: 11 August 2011, source: ABC News
Scientists for the first time have used gene therapy to successfully destroy cancer tumours in patients with advanced disease - a goal that has taken 20 years to achieve. Researchers at the University of Pennsylvania engineered patients' own pathogen-fighting T-cells to target a molecule found on the surface of leukaemia cells.
The altered T-cells were grown outside of the body and infused back into patients suffering from late-stage chronic lymphocytic leukaemia (CLL), which affects the blood and bone marrow and is the most common form of leukaemia.

Posted on: 3 August 2011, source: James Shapiro

Evolution: A View from the 20th Century describes the many ways that cells reorganize their own genomes in the course of evolution. This genome restructuring involves processes such as symbiogenesis, horizontal DNA transfer, and natural genetic engineering within the cell. These natural processes, as homologous recombination, transposition and site-specific recombination have already done, can provide models for artificial genetic engineering. Hopefully, increased focus on the evolutionary capabilities of living cells will expand the range of technologies available for gene therapy.

Posted on: 12 July 2011, source: ESGCT
Please consider participating in the following survey, which is being conducted by gene therapy and ethics researchers from the University of Sydney. This research aims to investigate how gene therapy researchers take account of considerations of risk when making decisions about the design and conduct of gene therapy clinical trials. The survey is completely anonymous and takes approximately 10-15 minutes to complete.

Posted on: 24 June 2011, source: PRNewswire
Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced that it has received an opinion on its Marketing Authorisation Application (MAA) for Glybera® (alipogene tiparvovec) as a potential therapy for Lipoprotein Lipase Deficiency ("LPLD"). Following a recent meeting with the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP), AMT has been notified that, at this time, Glybera is not approvable.
Subsequent to a review of the CHMP's letter, AMT believes that Glybera can receive a positive opinion subject to generating additional data from existing patients. AMT has therefore decided to ask for a re-examination of the clinical data package.

Posted on: 20 June 2011, source: ScienceBlog
University of Leeds researchers, funded by Cancer Research UK, have used a library of DNA to create a vaccine that could be used to treat cancer, according to a study published in Nature Medicine. Before now, ‘gene therapy’ vaccines have often delivered just one gene to stimulate the immune system. It produces a protein, called an antigen, which activates the immune system to destroy cancer cells. It has been difficult to develop successful cancer vaccines because each tumour has specific proteins and identifying the right antigens has been a huge challenge

Posted on: 5 June 2011, source: Children's Hospital Boston's Vector blog
Until this month, Agustín Cáceres’s baptism was the only time his family could come close to him. Everyone had to wear masks, gloves and gowns.
After that, he went into isolation, along with his mother Marcela, who came out only for meals. His father Alberto, and his four-year-old brother Jeremías, kept to a separate bedroom. Jeremías had to stop attending nursery school, for fear he’d bring home an infection his baby brother might catch. When Agustín’s relatives came to help out, they had to change their clothes and wash their hands, and couldn’t enter Agustín’s room.

Posted on: 12 May 2011, source: Medical News Today
A vaccine has been developed that protects non-human primates (monkeys) from SIV (Simian Immunodeficiency Virus), the monkey equivalent of HIV. Researchers reported in the journal Nature that this breakthrough could eventually lead to a vaccine for humans, protecting them from HIV. Louis Picker, M.D., from the Vaccine and Gene Therapy Institute (VGTI) in Oregon, and team produced a vaccine that programs monkeys' immune systems to respond more rapidly to the presence of SIV. Researchers from the International AIDS Vaccine Initiative and the National Cancer Institute-Fredick were also involved in this research.

Posted on: 1 May 2011, source: Gene Therapy Net
More than 60% of the people are of the opinion that gene therapy will be a vital part of future health care. Only 3% think that there is no future for gene therapy as it causes more problems than produce solutions. These are the results of an internet poll among the visitors of Gene Therapy Net.

Posted on: 22 April 2011, source: Children's Hospital Boston's Vector blog
Gene therapy, still experimental but beginning to enter the clinic, attempts to utilize advanced molecular methods to treat and even reverse genetic diseases. The field started in earnest about 25 years ago and has had many setbacks along the way to its recent earliest successes.
International collaboration has been critical. Children’s Hospital Boston is one of the founding members of the Transatlantic Gene Therapy Consortium (TAGTC), a new collaboration that seeks to facilitate a more rapid advancement of this technology for treating human diseases. It was initiated shortly after the first trials of gene therapy for X-linked Severe Combined Immunodeficiency (X-SCID) (in both Paris and London) reported leukemia as a serious side effect. The TAGTC was formed to address this setback, developing safer gene therapy reagents, sharing the costs of their development, and then implementing new gene therapy trials for rare diseases across multiple international sites.

Posted on: 12 April 2011, source: ScienceDaily
In the first clinical trial of gene therapy for treatment of intractable pain, researchers from the University of Michigan Department of Neurology observed that the treatment appears to provide substantial pain relief. In a study published online in the Annals of Neurology last week, the researchers showed that the novel agent NP2 is safe and well-tolerated. In addition, measures of pain in the treated patients suggested that NP2 may provide a substantial analgesic effect.

Posted on: 24 March 2011, source: EMA
Public online register gives access to information on clinical trials. The EU Clinical Trials Register (https://www.clinicaltrialsregister.eu) was launched today by the European Medicines Agency (EMA). The online register gives for the first time public access to information on interventional clinical trials for medicines authorised in the 27 EU Member States and Iceland, Liechtenstein and Norway. The database also allows the public to search for information on clinical trials authorised to be carried out outside the EU if these trials are part of a paediatric investigation plan. See also Clinical Trials Databases.

Posted on: 17 March 2011, source: Detroit Free Press
A groundbreaking study headquartered in Detroit has found that half the Parkinson's patients treated with gene therapy (NLX-P101) had lasting, "clinically meaningful improvements" without serious side effects, the Henry Ford Health System announced Tuesday.
The study is the first time gene therapy has proven successful in Parkinson's patients and could substantially improve the lives of one million Americans with the degenerative disorder, said Dr. Peter LeWitt, director of movement disorders and lead author of the study.

Posted on: 14 March 2011, source: NVGT
The Dutch Society of Gene Therapy (NVGT) has officially changed its name last Friday to the Netherlands Society of Gene and Cell Therapy (NVGCT), a change intended to better reflect the intimate relationship between the two fields.
The NVGCT is dedicated to promoting basic and clinical research in gene therapy and cell therapy within the Netherlands. By connecting scientists, clinicians, patients, public and government we wish to contribute to the development of gene and cell therapy for various diseases. The society aims to facilitate the exchange of information and technology, improve communication towards the public and provide education within the professional community.

Posted on: 1 March 2011, source: Aidsmap
Gene therapy that interferes with co-receptors on the surface of T-cells can protect these cells from HIV infection, representing a potential first step toward achieving a "functional cure," researchers reported at the 18th Conference on Retroviruses and Opportunistic Infections (CROI), taking place this week in Boston.
HIV uses two different surface co-receptors, CCR5 and CXCR4, to enter CD4 T-cells. If the co-receptors are blocked or disrupted, the virus is unable to enter cells. Two presentations on Monday looked at using gene therapy to create cells that lack these receptor proteins and therefore are protected from infection.

Posted on: 27 January 2011, source: ScienceDaily
An innovative genetic strategy for rendering T-cells resistant to HIV infection without affecting their normal growth and activity is described in a paper published in Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc.
A team of researchers from Japan, Korea, and the U.S. developed an anti-HIV gene therapy method in which a bacterial gene called mazF is transferred into CD4+ T-cells. The MazF protein is an enzyme (an mRNA interferase) that destroys gene transcripts, preventing protein synthesis. The design of this mazF gene therapy vector ensures that synthesis of the MazF protein is triggered by HIV infection. When HIV infects treated T lymphocytes, MazF is induced, blocking HIV replication and, essentially, making the T-cells HIV resistant.

Posted on: 29 November 2010, source: themedguru.com
A latest research by scientists at the Gladstone Institute of Neurological Disease (GIND) in San Francisco has revealed that a new gene therapy technique might hold the key to Alzheimer's cure. The study found that increasing the levels of EphB2, a protein responsible for regulation of neural communication in the brain, alleviated symptoms of long-term memory loss in mice.
Cognitive processes such as learning and memory retention require effective communication between brain cells called neurons. This communication involves release of chemicals that stimulate cell surface receptors present on the nerve cells. The process, called neurotransmission, is impaired by amyloid plaques, which build-up to abnormally high levels in the brains of Alzheimer's patients and are believed to be the root cause the disease.

Posted on: 16 November 2010, source: Irv Arons
Irv Arons recently completed a comprehensive treatise on the use of gene therapy for treating retinitis pigmentosa (RP) and the dry form of age-related macular degeneration (AMD). The report introduces RetroSense Therapeutics and its efforts to develop a gene therapy treatment to restore vision in those suffering from RP (first) and dry AMD (second). Included are sections on alternative therapies for both RP and AMD, including retinal implants (for RP), stem cells, and sustained release drugs and a few other options. In addition, there is a short writeup on other applications of gene therapy in ophthalmology.

Posted on: 31 October 2010, source: NIH RAC
In past safety symposia, the NIH Recombinant DNA Advisory Committee (RAC) reviewed clinical and molecular data concerning leukemias caused by insertional mutagenesis at or near oncogenes in two trials for X-linked Severe Combined Immunodeficiency Disease (X-SCID) involving transduction of CD34+ hematopoetic stem cells by retroviral vectors. Since these discussions, investigators have reported advances in the field, including clinical benefits in trials studying other clinical indications (e.g., adenosine deaminase deficient-SCID, adrenoleukodystrophy). In addition, research has focused on alternative vectors, either lentiviral vectors or modified retroviral vectors designed to decrease the risk of enhancer-mediated insertional mutagenesis.
Given these developments, as well as recent reports of myelodysplasia in a trial for chronic granulomatous disease and the finding of a relative clonal population of cells in a trial for thalassemia, the RAC and the European Network for the Advancement of Clinical Gene Transfer and Therapy (CliniGene) are holding this symposium (December 9-10, 2010, Bethesda).