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‘Dead as Doornail’ Gene Therapy Revival Spurs Genzyme

Posted on: 23 April 2010, source:
Five years ago, doctors drilled a hole into Jean Erickson’s skull, pushed in a needle and injected a cold virus modified to help transport a gene into her brain. The gene’s job was to create a mini-factory within her brain cells to pump out dopamine, a chemical that regulates physical movement. Since then, Erickson has lived more comfortably with Parkinson’s disease, a disorder that robs patients of mobility. “I stopped freezing up,” said Erickson, 71, a former pre- school director who lives in Castro Valley, California.

New Gene Therapy Breakthrough in Humans is Result of Research Pioneered at OHSU's Primate Center

Posted on: 15 April 2010, source:
A promising gene therapy method first developed through animal studies at Oregon Health & Science University has now been successfully tested using human cells. Today, the scientific journal Nature published results of a study conducted at Newcastle University in the United Kingdom where a nucleus from one embryo was transferred to another.
It is believed that by using this method of gene transfer, genetic diseases carried in cell mitochondria might be prevented, and maternally-based diseases that are frequently passed from generation to generation could be halted.

UF-led effort creates virus reference standard that can improve patient safety in clinical trials

Posted on: 13 April 2010, source: University of Florida news
The use of viruses as vehicles for delivering genes to replace malfunctioning or missing ones holds promise for treating many disorders. Adeno-associated viruses are one type of vector being used increasingly in human gene therapy clinical trials and laboratory studies leading up to those trials. But differences in the way researchers determine the administered doses have made it difficult to accurately compare results from various studies.

Nanotech robots deliver gene therapy through blood

Posted on: 22 March 2010, source: Reuters
U.S. researchers have developed tiny nanoparticle robots that can travel through a patient's blood and into tumors where they deliver a therapy that turns off an important cancer gene. The finding, reported in the journal Nature on Sunday, offers early proof that a new treatment approach called RNA interference or RNAi might work in people.

Scientists Still Hopeful About Gene Therapy's Promise

Posted on: 8 March 2010, source: NPR
Twenty-five years ago, it seemed as if gene therapy was on the verge of revolutionizing medicine. But that revolution never occurred, and scientists realized that they had been overly optimistic about how quickly they could develop such therapies. Now, however, there are signs that the field of gene therapy is making definite progress, even if the revolution is still on hold. The concept of gene therapy is simple. Some diseases are caused by damage to a single gene — for example, cystic fibrosis and hemophilia. Give patients the healthy gene, and in theory, the disease is cured.

Gene Therapy a Step Closer to Restoring Eyesight to Some Blind Patients

Posted on: 7 March 2010, source: Voice of America
Researchers have moved a step closer toward fully restoring the eyesight of people with a rare genetic disorder. A new study shows the treatment is safe and effective, and could pave the way for helping cure more common causes of blindness. Leber's congenital amaurosis is an extremely rare condition that causes blindness in approximately 4,000 people in the United States.

Adeno-Associated Virus Type 2 Reference Standard Material is Now Available

Posted on: 30 January 2010, source: ASGCT
The AAV2 vector reference standard material (an AAV2-GFP viral vector) is now available from ATCC ( The AAV2 RSM is intended for use in calibrating internal (laboratory-specific) reference materials and assays for recombinant AAV viral gene transfer products, with the purpose of making data from different pre-clinical and clinical studies more comparable.

Amsterdam Molecular Therapeutics: EMA starts formal review of Glybera dossier

Posted on: 25 January 2010, source:
Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, has reached another important milestone in the official marketing authorisation process for its lead product Glybera, AMT's proprietary product for lipoprotein lipase deficiency (LPLD). The submission of the Glybera Marketing Authorisation Application (MAA), announced earlier, has cleared the validation stage with The European Medicines Agency (EMA, formerly known as EMEA). The EMA will now commence its formal review of Glybera.

The EMA has published a Concept paper on the revision of the note for guidance on the quality, pre-clinical and clinical aspects of gene transfer medicinal products

Posted on: 24 January 2010, source: The European Medicines Agency
The European Medicines Agency has published a Concept paper on the revision of the note for guidance on the quality, pre-clinical and clinical aspects of gene transfer medicinal products (EMA/CHMP/GTWP/BWP/234523/2009). This Concept Paper proposes a revision of the Note for Guidance on the Quality, Preclinical and Clinical Aspects of Gene Transfer Medicinal Products (CPMP/BWP/3088/99) that came into effect in 2001. This revision will address the issues identified from clinical experience and provision of Scientific Advice on gene therapy medicinal products and will lay down detailed and updated requirements for the quality, non-clinical and clinical aspects of gene therapy medicinal products.

Molecular Security System That Protects Cells from Potentially Harmful DNA Discovered

Posted on: 14 January 2010, source: ScienceDaily
Researchers at the University of Minnesota have discovered a molecular security system in human cells that deactivates and degrades foreign DNA. This discovery could open the door to major improvements in genetic engineering and gene therapy technologies. Led by Reuben Harris, associate professor of biochemistry, molecular biology and biophysics in the College of Biological Sciences, the report's findings will be published online by Nature Structural and Molecular Biology on Jan. 10.

Oxford BioMedica Receives Orphan Designation for Gene Therapy Treatment for Stargardt Disease

Posted on: 15 December 2009, source: Proactive Investors UK
Gene therapy developers, Oxford BioMedica (LSE: OXB) announced its StarGen therapy has received orphan treatment designation from the European Medicines Agency (EMEA). As a result the treatment will have ten years of marketing exclusivity and reduced regulatory fees. StarGen’s clinical development is expected to start in 2010, in collaboration with Sanofi-Aventis (NYSE: SNY). Stargardt Disease is a rare disease that causes cells on the macula area of the retina to stop working, which leads to problems with central vision. At first it makes vision unclear, distorted or blurred. After a longer period, Stargardt's can cause a ‘blank patch’ in the centre vision. There are currently no treatments available for Stargardt disease.

Gene Therapy Halts Brain Disease in Two Boys

Posted on: 8 November 2009, source: ScienceNOW
Researchers have used a modified AIDS virus to halt a devastating brain disease in two young boys. The treatment, in which the virus delivered a therapeutic gene, marks the first time gene therapy has been successfully used against X-linked adrenoleukodystrophy (ALD)--a disorder that is always fatal if untreated. With this proof of principle, scientists hope versions of the AIDS virus engineered to carry different genes can now be applied to a variety of other diseases. ALD is caused by a defect in an X chromosome gene that produces a protein called ALD. Cells need this transporter protein to break down certain fats; without it, the fats build up and damage the myelin sheathing that protects nerves. In X-linked ALD, which strikes mainly boys, patients develop neurological symptoms such as seizures and loss of vision around age 6 to 8, and within months they become paralyzed, deaf, and eventually die.

Gene Therapy Repairs Injured Human Donor Lungs For The First Time

Posted on: 2 November 2009, source: ScienceDaily
For the first time, scientists in the McEwen Centre for Regenerative Medicine, University Health Network have successfully used gene therapy to repair injured human donor lungs, making them potentially suitable for transplantation into patients. This technique could significantly expand the number of donor lungs by using organs that are currently discarded, and improve outcomes after transplantation.
In their pioneering work, a team of researchers led by Dr. Shaf Keshavjee, Senior Scientist at the McEwen Centre for Regenerative Medicine, University Health Network and Director of the Lung Transplant Program, University Health Network developed a technique of ex vivo gene delivery to donor lungs, before they are implanted into a recipient's body. The technique was shown to be simple and effective in improving lung function.

One Shot Of Gene Therapy, And Children With Congenital Blindness Can Now See

Posted on: 30 October 2009, source: ScienceDaily
Born with a retinal disease that made him legally blind, and would eventually leave him totally sightless, the nine-year-old boy used to sit in the back of the classroom, relying on the large print on an electronic screen and assisted by teacher aides. Now, after a single injection of genes that produce light-sensitive pigments in the back of his eye, he sits in front with classmates and participates in class without extra help. In the playground, he joins his classmates in playing his first game of softball.

AMT Receives EMEA Orphan Drug Designation for Duchenne Muscular Dystrophy

Posted on: 13 October 2009, source:
Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today that the European Medicines Agency (EMEA) has granted Orphan Drug Designation to AMT's gene therapy product AMT-080 for the treatment of Duchenne muscular dystrophy.
Orphan Drug Designation for Duchenne muscular dystrophy (DMD) entitles AMT to ten year market exclusivity in Europe following marketing approval for AMT-080 if this product candidate is the first new drug with a major medical benefit receiving marketing approval for the European Union. The designation also provides for special benefits, including research support, eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees during development or at the time of application for marketing approval.

Scientists Use Gene Therapy To Cure Colorblind Monkeys

Posted on: 17 September 2009, source: The Washington Post
Scientists say they have used gene therapy to enable colorblind monkeys to see red and green, possibly opening the door to curing colorblindness in people. Jay Neitz of the University of Washington in Seattle and his colleagues injected gene-carrying viruses into the retinas of two male squirrel monkeys, which are naturally colorblind. The gene carried instructions for the production of a protein known as opsin, which makes pigments that are sensitive to the colors red and green. About five weeks after the treatment, the monkeys -- named Dalton and Sam -- began to develop the ability to see those colors, according to the results of detailed testing reported this week in the journal Nature.

The Second Coming of Gene Therapy

Posted on: 3 September 2009, source: Discover magazine
For years, gene therapy produced tons of hype but no results. Recently, though, new approaches have yielded its first successes: breakthrough treatments for blindness, cancer, and the deadly bubble boy disease.
“For the first two years of her life, my daughter, Katlyn, was knocking on heaven’s door every day,” says Daisy Demerchant, a 26-year-old mom living in Centreville, New Brunswick, just north of Maine. “Two months after she was born she started getting sick, and she never got better.” At six months Katlyn was diagnosed with “bubble boy” disease, formally known as severe combined immunodeficiency (SCID), which robs the immune system of the ability to fight infection. There are many causes of this disorder; in Katlyn’s case it was lack of the enzyme adeno­sine deaminase, or ADA, which rids the body of a natural toxin called deoxyadenosine. When the toxin builds up, it destroys T and B lymphocytes, the body’s infection-fighting immune cells. As a result, Katlyn’s immune cells were dying.

Scientists Construct 'Off Switch' For Parkinson Therapy

Posted on: 29 August 2009, source: ScienceDaily
A common antibiotic can function as an "off switch" for a gene therapy being developed for Parkinson's disease, according to University of Florida researchers writing online in advance of September's Molecular Therapy. The discovery in rats answers an important question — how can new, therapeutic genes that have been irrevocably delivered to the human brain to treat Parkinson's be controlled if the genes unexpectedly start causing problems? Meanwhile, in a review of Parkinson treatments, the researchers say that prior experimental attempts using growth factors — naturally occurring substances that cause cells to grow and divide — to rescue dying brain cells may have failed because they occurred too late in the course of the disease. Together, the findings suggest that gene therapy to enable the brain to retain its ability to produce dopamine, a neurotransmitter that falls in critically short supply in Parkinson's patients, could be safely attempted during earlier stages of the disease with an added likelihood of success.

Study Clears Gene Therapy in Death of Arthritis Patient

Posted on: 10 July 2009, source: MedPage Today
The death of a young patient participating in a gene-therapy trial for a rheumatoid arthritis drug was most likely the result of another arthritis drug, adalimumab (Humira), and not the novel gene therapy agent, a new study found. "The death of this young patient who was receiving multiple forms of TNF inhibitors highlights the risk of opportunistic infections in patients receiving such agents, and the importance of having a well-designed monitoring plan when a patient in a study becomes ill," Karen M. Frank, MD, of the University of Chicago Medical Center, and colleagues wrote in the July 9 issue of New England Journal of Medicine.
The patient, a 35-year-old woman with a 15-year history of rheumatoid arthritis, first presented three days after receiving a second injection on July 2, 2007, of the active gene-therapy agent, tgAAC94, a tumor necrosis factor alpha (TNF-α) antagonist. She was suffering from a range of symptoms that including fever, chills, abdominal pain, and vomiting.

Clonal Population of Cells Detected in a Clinical Human Gene Transfer Trial Using Lentiviral Vector

Posted on: 23 June 2009, source: Recombinant DNA Advisory Committee
The National Institutes of Health Office of Biotechnology Activities (OBA) has been informed that a "relative clonal dominance" was detected during follow-up of a subject who is participating in a French human gene transfer trial being conducted for individuals with β-Thalassemia Major and Sickle Cell Anemia. The clinical trial, sponsored by Genetix France, used hematopoietic stem cells transduced by a self inactivating (SIN) HIV-1 lentiviral vector containing the gene for β-globin under the control of the β-globin promoter. The subject received the gene modified cells in June 2007.
This clonal dominance appears to result from the integration of the vector in the gene encoding for the HMGA2 protein, which is associated with both benign and malignant tumors. The clone however has been stable for five months and the subject remains in good health. In fact, although the subject required almost monthly blood transfusions during the 11 months prior to the gene transfer intervention, the subject has not since required a blood transfusion.

ASGT Embraces Cell Therapy with Name Change

Posted on: 29 May 2009, source: ASGCT
The American Society of Gene Therapy (ASGT) will officially change its name Saturday to the American Society of Gene & Cell Therapy (ASGCT), a change intended to better reflect the intimate relationship between the two fields. “This new name more accurately reflects who we are and what we do,” said David M. Bodine, PhD, ASGT president, who led the initiative to add cell therapy to the name. “Gene therapy and cell therapy cannot be separated; each is an integral part of the other.” Added Ken Cornetta, MD, incoming ASGCT president: “Gene therapists are cell therapists. We have the same goal, which is improved patient care.” The change goes into effect at 7:45 a.m. Saturday, May 30, during the business meeting at the ASGT 12th Annual Meeting.

Moving Gene Therapy Forward With Mobile DNA

Posted on: 11 May 2009, source: ScienceDaily
Gene therapy is the introduction of genetic material into a patient's cells resulting in a cure or a therapeutic effect. In recent years, it has been shown that gene therapy is a promising technology to treat or even cure several fatal diseases for which there is no attractive alternative therapy. Gene therapy can be used for hereditary diseases, but also for other diseases that affect heart, brain and even for cancer. Indeed, recent results suggest that gene therapy can be beneficial for patients suffering from aggressive brain cancer that would otherwise be lethal.

Analysis: Gene therapy has immense potential

Posted on: 14 April 2009, source: Times Online
After almost two decades, gene therapies have recently started to deliver on their immense promise. Gene therapies have been a part of medicine for almost two decades. Only recently have they started to deliver on their immense promise after a string of setbacks raised doubts about the safety of manipulating human DNA, centre, to treat disease.
The idea behind gene therapy is that by inserting new copies of a particular gene into human cells, it should be possible to correct defects, or to enhance beneficial biological processes. This can be done with viruses, which introduce their own genetic material into the cells they infect, and can be engineered to carry a human gene.

Gene Therapy Gets Closer to a 'Cure'

Posted on: 3 March 2009, source:
Two decades ago, medicine seemed on the cusp of a revolution. Doctors would soon treat diseases at their very roots, inserting "good" genes to replace patients' faulty ones. Gene therapy was a seductively straightforward idea that offered promise for treating everything from cancer to sickle cell disease.
But only now, after overcoming unexpected scientific obstacles and the high-profile death of a teenage patient, is gene therapy racking up some clear-cut successes. Promising studies are sending ripples of excitement through the field. Some researchers are daring to use the word cure.

Ark Therapeutics Files Cerepro® Application for European Marketing Authorisation

Posted on: 8 January 2009, source: Biospace
Ark Therapeutics announced that the Marketing Authorisation Application (MAA) for Cerepro®, Ark’s novel gene-based therapy for operable malignant glioma (brain cancer) which was recently filed with the EMEA, has cleared the validation stage. The Cerepro® MAA application now commences formal review via the centralised procedure which is the standard route for all advanced therapies.
Cerepro® was originally filed for marketing approval in late 2005 and was reviewed by the EMEA with reliance on Phase II data. The review established that the technical chemistry and manufacturing controls (CMC), preclinical and environmental sections appeared acceptable, but more clinical data were needed to confirm the Phase II findings and to demonstrate the reproducibility of the results in a larger multi-centre Phase III trial.

Proposed ASGT name change to the American Society of Gene and Cell Therapy (ASGCT)

Posted on: 27 November 2008, source: ASGT website / Molecular Therapy
A proposal to change the name of the American Society of Gene Therapy (ASGT) to the American Society of Gene and Cell Therapy (ASGCT) has recently been adopted by the 2008 Membership Committee and forwarded to the Board of Directors of ASGT. The Board has unanimously approved the proposal, which is now being forwarded to the general membership of the Society for a final vote in April of 2009. The Membership Committee’s rationale in making this forward-looking proposal is that the concept of gene therapy includes gene-modified cell therapy, and an inclusive name will empower the Society to expand its membership base and help foster further collaborative research to advance the use of genes and cells as medicine to treat disease.

Gene Therapy For Blindness Improves Vision, Safety Study Indicates

Posted on: 8 September 2008, source: ScienceDaily
All three people who received gene therapy at the University of Florida to treat a rare, incurable form of blindness have regained some of their vision, according to a paper published online today in Human Gene Therapy.
The patients with a type of hereditary blindness called Leber congenital amaurosis type 2, volunteered to test the safety of an experimental gene-transfer technique in a phase 1 clinical research study. In this form of LCA disease, photoreceptor cells cannot respond to light because a gene called RPE65 does not properly produce a protein necessary for healthy vision. In the study, researchers used an adeno-associated virus to deliver RPE65 to a small area of the retina. Not only were there no ill effects other than routine postsurgical soreness, the subjects said the vision in their treated eyes was slightly improved in dim lighting conditions.

Introgen Submits ADVEXIN® Regulatory Applications in the U.S. and Europe

Posted on: 1 July 2008, source: FierceBiotech
Introgen Therapeutics, Inc. submitted a Biologics License Application (BLA) to the FDA requesting marketing approval for ADVEXIN p53 therapy to treat recurrent, refractory head and neck cancer. Simultaneously, Gendux Molecular Limited, an Introgen subsidiary, submitted a Marketing Authorization Application to the EMEA for the same indication. ADVEXIN represents the first of a new class of tumor suppressor cancer therapy and is the first of its kind to be submitted for regulatory approval in the United States and Europe.

Gene Therapy Improves Vision In Nearly Blind Patients

Posted on: 28 April 2008, source: ScienceDaily
In a clinical trial at The Children's Hospital of Philadelphia, researchers from The University of Pennsylvania have used gene therapy to safely restore vision in three young adults with a rare form of congenital blindness. Although the patients have not achieved normal eyesight, the preliminary results set the stage for further studies of an innovative treatment for this and possibly other retinal diseases.
An international team led by The University of Pennsylvania, The Children's Hospital of Philadelphia, the Second University of Naples and the Telethon Institute of Genetics and Medicine (both in Italy), and several other American institutions reported their findings in an online article in the New England Journal of Medicine.

Boy develops leukemia after gene therapy in UK

Posted on: 18 December 2007, source: Reuters
A three-year-old "bubble boy" undergoing pioneering gene therapy in London has developed leukemia, marking another setback for the experimental treatment. Doctors at Great Ormond Street Hospital said on Tuesday the boy had been successfully treated for SCID-X1, or x-linked severe combined immunodeficiency, often known as "baby in the bubble syndrome", but had developed leukemia two years later. The news is a blow to the treatment program at the London hospital, which has a worldwide reputation from treating sick children.