Bluebird wins orphan drug designation for ALD treatment
Cambridge-based genetic disorder drug maker bluebird bio said today it has received an orphan drug designation from both the U.S. Food and Drug Administration and the European Medicines Agency for its investigational gene therapy designed to treat adrenoleukodystrophy. Known as ALD, adrenoleukodystrophy is a rare, inherited disorder that leads to progressive brain damage, failure of the adrenal glands and ultimately death. The company said it plans to initiate a Phase 2/3 clinical study in childhood cerebral ALD in both the U.S. and Europe next year.
Gene Therapy Society Looking to NIH to Revisit Role of the Recombinant DNA Advisory Committee
The principal U.S. professional society representing gene therapists is awaiting word from NIH about whether or not there will be re-examination of the role of its Recombinant DNA Advisory Committee (RAC). They aim to limit its longstanding role in clinical trials while continuing its service as a forum for discussing and educating professionals on policy issues. The American Society of Gene & Cell Therapy (ASGCT) touched off the debate earlier this year, when its president and her predecessor conveyed the society’s view that RAC should focus on broader issues rather than review the clinical protocols of individual researchers. They sent a letter to Amy Patterson, M.D., director of NIH’s Office of Biotechnology Activities (OBA), and issued a statement seven days later at a RAC meeting.
Targeting DNA: After 20 years of high-profile failure, gene therapy is finally well on its way to clinical approval
The concept is simple: if a mutated gene is causing a problem, replace or supplement it with a new, accurate copy. In theory, such a strategy could not just treat, but cure countless human genetic diseases. In practice, however, developing safe and effective gene therapies has not been easy. Even when identifying a disorder’s genetic basis is fairly straightforward, finding the appropriate delivery vector to target the diseased tissues in the body, while avoiding unintended consequences, has challenged would-be gene therapists for more than 20 years. But more and more researchers are convinced that the technique is on the brink of becoming a common medical practice.
RetroSense Therapeutics Completes pre-IND Meeting for RST-001
RetroSense Therapeutics, a gene therapy company dedicated to vision restoration, announced completion of a pre-IND meeting with the Center for Biological Evaluation and Research (CBER) division of the FDA that took place on May 22nd 2012 regarding RetroSense’s lead biologic, RST-001 for vision restoration in retinal degenerative conditions. The purpose of the meeting was to obtain CBER’s guidance for the clinical path to a Biologics License Application (BLA) for RST-001 in the US, and clarity on the steps required for Investigational New Drug (IND) submission. The discussions included manufacturing criteria, the scope and design of the preclinical studies, and the scope and design of Phase I and IIa clinical trials.
Gene Therapy in Ophthalmology Clinical Trial Details: An Attempt to Track the Number of Patients Treated to Date
In attempting to determine how many patients had been treated with gene therapy for eye disorders, Irv Arons quickly found that no one was keeping track – at least no one that he could find. So, Irv Arons decided to try and get this data. He has now found reliable data for more than two-thirds of the 15 clinical trials underway and presents this information in his new table.
Gene Therapy Safe in Decade-Long HIV Study That May Widen Use
HIV patients given gene therapy more than a decade ago are healthy and the altered DNA they received remains stable in their bodies, according to a study that scientists say proves the treatment may safely be tested as a way to attack other illnesses. All except two of 43 people treated with genetically- altered versions of their own infection-fighting T cells were healthy as many as 11 years later, according to the research reported today in the journal Science Translational Medicine.
Top Cancer Scientists Converge to Celebrate ACGT's 10th Anniversary and New Frontiers in Treating Cancer
More than 30 of the nation's top cancer research scientists are gathering in Greenwich, Conn., this week to compare notes, collaborate, and celebrate ACGT's 10 years of innovative cancer research funding. ACGT was the funding arm of the breakthrough Leukemia clinical trial study at the University of Pennsylvania last summer that obliterated the cancer in patients.
Brain gene therapy is beating my Parkinson's
Doctors in Britain say a radical new treatment for Parkinson's could improve the lives of millions who suffer from the disease. Sheila Roy says she can finally enjoy life at her countryside home in Cambridge, England. She has lived with Parkinson's disease for 17 years, but a team of Oxford scientists developed a treatment that changed her life. “I can see a glimmer of a person I use to be now, which is really exciting,” she said. Roy was one of only 15 people in the world to take part in a gene therapy experiment that was previously tested on lab animals. Scientists create and inject a virus into the brain. It jumpstarts the production of dopamine, a chemical that fights Parkinson's. Without dopamine, patients suffer tremors and uncontrolled movements.
Potential gene therapy for patients with rare disease
Australian scientists have discovered that a biological phenomenon known as ‘somatic reversion’, when an abnormal gene spontaneously becomes normal again, explains why some patients with a rare genetic disorder live much longer than they should.
Study shows developing a gene therapy for cancer will be harder than scientists thought
Scientists working to customize cancer treatment based on each person's genes are finding new evidence of just how tough this task will be. In a single tumor, there are big differences as to which genes are active or mutated. Nor are the genetics of a single tumor and where it has spread consistent. This means that the single biopsies that doctors rely on to choose drugs are probably not giving a true view of the cancer's biology.
CHMP asks AMT to provide additional information on Glybera
Amsterdam Molecular Therapeutics (AMT), a leader in the field of human gene therapy, has received a request to provide additional information to the Committee for Human Medicinal Products (CHMP) on Glybera (alipogene tiparvovec) by March 8, 2012. The CHMP request follows the announcement on January 30, 2012 that the European Commission Standing Committee was requesting additional information from the CHMP. The CHMP has indicated that a final decision on Glybera could be made by May 2012.
ACGT 2012 Investigator's Award in Clinical Translation of Gene Therapy for Pancreatic Cancer,
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
NVGCT launches new website
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.
Amsterdam Molecular Therapeutics Announces European Commission Delays Decision on Glybera® Marketing Authorisation And Requests Further Information From the CHMP
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.
HSCI Receives Approval To Market Neovasculgen – The First Russian Gene Therapy Drug For Treatment Of Peripheral Arterial Disease
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.
Gene therapy proves effective for hemophilia B
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.
Gene Therapy Video on the CliniGene Website
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: http://www.clinigene.eu/video-intro-gene-therapy.html. 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.
AMT Suspends Glybera and Plans 50% Job Cuts in Restructuring Moves
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.
Amsterdam Molecular Therapeutics Receives Opinion on Re-examination of Glybera® Marketing Authorisation Application
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.
Gene therapy and stem cells unite
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.
Gene Therapists Celebrate a Decade of Progress
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.
Sangamo gene therapy shows promise in reducing HIV
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.
Bubble Boys Treated With Gene Therapy Still Healthy After 9 Years
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.
Genetically modified cells destroy leukaemia tumours
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.
Evolution: A View from the 20th Century
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.
Survey on Risk Assessment in Clinical Trials
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.
Amsterdam Molecular Therapeutics Receives Opinion on Glybera Marketing Authorisation Application
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.
New approach to cancer vaccines proves successful in early studies
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
A new start for gene therapy for “bubble boy” disease: First U.S.-treated patient doing well
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.
Monkey Vaccine Has Promise For Eventual Human HIV Vaccine
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.