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Cell and gene therapy in Cambridge, London and Oxford: an invitation to collaborate

Posted on: 10 February 2016, source: londonandpartners.com
The golden triangle is the world-leading life sciences cluster of Cambridge, London, Oxford and the greater south east region of England. It comprises multiple award-winning research institutions, thousands of talented scientists, deep experience in clinical trials, and a thriving global business and life sciences community. Key to the region’s success has been its collaborative mindset – a drive to embrace and exchange ideas with scientists and professionals from around the world. This report features Life Sciences Minister George Freeman, the world’s first dedicated life sciences minister. Along with case studies and interviews with high profile academics and decision-makers, this report will help to understand the current cell and gene research climate in London.

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British researchers get green light to genetically modify human embryos

Posted on: 2 February 2016, source: The Guardian
Britain’s first genetically modified human embryos could be created within months, after scientists were granted permission by the fertility regulator to carry out the procedure. The Human Fertilisation and Embryology Authority (HFEA) regulator approved a licence application by Kathy Niakan, a stem cell scientist at the Francis Crick Institute in London, to perform so-called genome editing on human embryos. The decision permits Niakan to study the embryos for 14 days for research purposes only. It does not permit them to be implanted into women. Niakan’s research is aimed at finding the genes at play in the early days of human fertilisation.

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AGTC and BCM Families Foundation Announce Collaboration to Develop AAV-Based Gene Therapy for Blue Cone Monochromacy

Posted on: 22 January 2016, source: BCM Families Foundation
Applied Genetic Technologies Corporation (Nasdaq:AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare eye diseases, and the BCM Families Foundation, a non-profit organization focused on eradicating Blue Cone Monochromacy (BCM), today announced a collaboration to develop an AAV-based gene therapy for the disease. Blue Cone Monochromacy, also known as X-linked achromatopsia, is a rare genetic disease of the retina that almost exclusively affects males. It is a hereditary condition linked to the X chromosome that manifests with a partial dysfunction of the cones of the retina. BCM can result in reduced visual acuity, impaired color vision, photosensitivity, myopia and infantile-onset nystagmus. These manifestations are similar to those in achromatopsia, caused by mutations in the CNGB3 or CNGA3 gene, for each of which AGTC has ongoing clinical development activities.

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Stanford bringing gene editing to patients with deadly diseases

Posted on: 8 January 2016, source: Mercury News
Tiny vials of recently repaired blood cells are thriving in a Stanford incubator, proof that a powerful new gene-editing technique is fixing errant genes that cause so much human suffering. Until recently, gene therapy was laborious, crude and unsafe for human testing. But the new technology, called CRISPR-Cas9, acts as a microscopic scalpel, performing genomic surgery with a precision, efficiency and affordability once thought unimaginable. The research being done at the Stanford School of Medicine, led by Dr. Matthew Porteus, is part of an accelerating research movement made possible using the new technique to try to cure genetic diseases such as sickle cell anemia and muscular dystrophy. These labs are steadily advancing through cell-based and animal trials, as fledgling biotech companies raise large sums of money needed to bring the therapies to market.

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UniQure Announces Preliminary Topline Results from Low-Dose Cohort in Hemophilia B Phase I/II Gene Therapy Clinical Trial

Posted on: 7 January 2016, source: UniQure
uniQure N.V. (Nasdaq: QURE), a leader in human gene therapy, today announced preliminary topline results from the low-dose cohort of an ongoing Phase I/II clinical trial being conducted in adult hemophilia B patients treated with uniQure’s novel AAV5/FIX gene therapy, AMT-060. All five patients in the low-dose cohort had Factor IX (FIX) phenotypic features of severe or moderately-severe hemophilia including documented Factor IX (FIX) levels less than 1-2% and required chronic treatment with prophylactic recombinant FIX (rFIX) therapy at the time of enrollment.

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CRISPR Gene Therapy Has Cured a Mouse With Muscular Dystrophy

Posted on: 1 January 2016, source: Inverse.com
For the first time, a living mammal was cured of a genetic disease with a treatment we could use on humans.Continuing mankind’s formidable strut towards an age of gods and monsters, for the first time ever researchers have successfully treated a genetic disease in a living mammal with a method that could be used on humans. Thanks to CRISPR — the incredibly powerful genetic engineering process — and some bioengineers at Duke University, an adult mouse with muscular dystrophy will enter 2016 much healthier. The findings are out today in a paper at Science.

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Cancer Gene Therapies: FDA Officials Highlight Regulatory Approaches

Posted on: 27 November 2015, source: FDA
As gene therapies begin to get a foothold among other cancer treatments, the US Food and Drug Administration (FDA) is taking a more flexible, data-driven approach for the preclinical testing programs of these biologically complex products, according to a new review from five FDA officials in Cancer Gene Therapy.
Although FDA has yet to approve a gene therapy to treat cancer, the authors note that about two-thirds of gene therapy clinical trials are for cancer treatments. In order to help industry and academia with these trials, the agency has released a number of guidance documents, including recent recommendations for microbial vectors used for gene therapy and how shedding studies for virus and bacteria-based gene therapies and oncolytic products should be designed.

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Symposium and educational session of the Netherlands Society of Gene and Cell Therapy (NVGCT)

Posted on: 19 November 2015, source: NVGCT
The 2016 NVGCT Spring Symposium will be held on March 10 and 11, 2016 in Lunteren, The Netherlands. The theme is gene delivery. Several stablished keynote speakers have already confirmed their presence. In addition several educational lectures will be presented. Register now before 31 January 2016.
Recent clinical successes have boosted the interest for Gene Therapy. The number of trials both for acquired and inherited diseases will increase significantly in the near future. Because of this and the changes in legislation the NVGCT has decided to also organize an educational session in collaboration with UniQure, the COGEM, Bureau GGO, ARM and the CCMO, on how to fill in the applications to help your with the obtaining the permits in an efficient way and to start performing your gene therapy trial in the Netherlands without delays.

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Gene-Edited Cells Reverse Cancer in Baby Girl

Posted on: 8 November 2015, source: Oracle Herald
Layla was born in the United Kingdom, and was diagnosed with what's known as acute lymphoblastic leukaemia - an aggressive cancer of the bone marrow - at just three months old. Following the diagnosis, the child was admitted to London's Great Ormond Street Hospital intensive care unit. Two months later, once the doctors confirmed that the leukaemia cells had all been removed, she was given another bone marrow transplant. The disease returned seven weeks later. The improvement on Layla using the treatment according to the doctors was "staggering" and "miraculous".

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FDA Grants Gene Therapy Orphan Drug Status for Rare Genetic Disorder

Posted on: 5 November 2015, source: FDA
Agilis Biotherapeutics announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to the investigational gene therapy AGIL-AS for the treatment of Angelman syndrome (AS), a rare neuro-genetic disorder characterized by severe intellectual and developmental disability.

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Where in the world could the first CRISPR baby be born?

Posted on: 15 October 2015, source: Nature
They are meeting in China; they are meeting in the United Kingdom; and they met in the United States last week. Around the world, scientists are gathering to discuss the promise and perils of editing the genome of a human embryo. Should it be allowed — and if so, under what circumstances? A look at the legal landscape suggests where human genome editing might be used in research or reproduction.

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A Tale of Do-It-Yourself Gene Therapy

Posted on: 14 October 2015, source: Technology Review
An American woman claims she is the first to undergo gene therapy to reverse aging. Judge for yourself. Can aging be slowed by using gene therapy to make permanent changes to a person’s DNA? One Seattle-area woman says she has tried exactly that. Her claim has entangled some high-profile American academics in a strange tale of do-it-yourself medicine that involves plane flights to Latin America, an L.A. film crew, and what’s purported to be the first attempt to use gene therapy to forestall normal aging.

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Experimental gene therapy doubles glioblastoma survival rate

Posted on: 3 October 2015, source: UpdatedNews
An experimental gene therapy drug doubled the survival rate for glioblastoma, an aggressive brain cancer that kills two-thirds of patients within five years, researchers reported at a cancer conference. Typically, glioblastoma patients whose cancer comes back are expected to survive for weeks or months. Researchers reported the results of a Phase 2 clinical trial at the European Cancer Congress 2015 as Phase 3 of the trial begins.

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Promising Results on Gene Therapy for Congestive Heart Failure

Posted on: 10 September 2015, source: MD Magazine
San Diego-based Renova Therapeutics reported that its RT-100 gene therapy treatment for patients with reduced left-ventricular ejection fraction congestive heart failure (CHF) is proving safe and effective. The treatment involves delivering a therapeutic gene encoding adenylyl clyclase type 6 (AC6) directly into the heart via a modified adenovirus. It is a single-dose procedure done in an outpatient setting. Reporting on the results of a multi-center, placebo-controlled Phase 2 trial, company co-founder Kirk Hammond, MD, said the therapy showed a strong safety profile and clear dose response. The study assessed the safety of various doses of RT-100 in people with CHF.

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How CRISPR Could Change the World

Posted on: 15 August 2015, source: Tech Insider
Before 2012, researchers interested in studying how bacteria worked knew that microbes defended themselves against certain viruses using something they called a CRISPR/Cas system. There are a number of studies published in the early 2000s — starting in 2002 — discussing the presence of these systems and investigating how they work. There weren't many people studying that system then, especially compared to now, and those working in that area were doing what's called basic, or fundamental science, investigating something for the sake of curiosity or interest — not because they knew that research would have a practical or profitable result. They didn't know they were studying something that could be used to change the world, something that would give scientists the ability to rewrite DNA — the building blocks of life.

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Resurrection Of Extinct Virus Could Open The Doors To Gene Therapy Innovations

Posted on: 2 August 2015, source: Medical Daily
Gene therapy involves using viruses to replace missing or malfunctioning genes in order to treat some diseases, such as cancer. Experts believe this technique is at the forefront of medical treatment, but unfortunately it has one massive flaw: the immune system often destroys the viruses before they have the chance to deliver the therapy. According to a recent study, the way to get around this flaw could be by resurrecting an ancient virus.

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Positive Data Announced in Phase IIb Clinical Trial of Cystic Fibrosis Gene Therapy Manufactured by VGXI, Inc

Posted on: 11 July 2015, source: VGXI
Recently, the United Kingdom Cystic Fibrosis Gene Therapy Consortium (UKCFGTC) published the first clinical data from a Phase IIb, multi-dose clinical trial for cystic fibrosis. In this trial, patients received aerosolized DNA plasmid expressing Cystic Fibrosis Transmembrane Conductance Receptor (CFTR), manufactured at the VGXI cGMP production facility. This trial is the first to show that gene therapy can have a meaningful effect on the disease and benefit the lung function of patients with cystic fibrosis.

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Disruptive CRISPR gene therapy is 150 times cheaper than zinc fingers and CRISPR is faster and more precise

Posted on: 13 June 2015, source: Nextbigfuture
Biologists have long been able to edit genomes with molecular tools. About ten years ago, they became excited by enzymes called zinc finger nucleases that promised to do this accurately and efficiently. But zinc fingers, which cost US$5,000 or more to order, were not widely adopted because they are difficult to engineer and expensive, says James Haber, a molecular biologist at Brandeis University in Waltham, Massachusetts. CRISPR works differently: it relies on an enzyme called Cas9 that uses a guide RNA molecule to home in on its target DNA, then edits the DNA to disrupt genes or insert desired sequences. Researchers often need to order only the RNA fragment; the other components can be bought off the shelf. Total cost: as little as $30. “That effectively democratized the technology so that everyone is using it,” says Haber. “It's a huge revolution.”

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Chinese Gene-Editing Experiment Creeps Out Scientists

Posted on: 24 April 2015, source: Nature
Germ-line gene therapy: A group of scientists in China has just crossed one of biotechnology's red lines.
Chinese scientists have caused an uproar by trying to permanently edit the DNA of human embryos — created genetic changes that could be passed along from generation to generation. Their attempt didn't work very well, but the report, published in a small, online journal called Protein & Cell, has worried experts who have been watching out for such experiments. The motivation is to cure disease. In this experiment, the researchers were trying to correct defects in a gene called HBB that can cause a deadly blood disorder called beta-thalassemia. Gene therapy in adults and children is still experimental; the idea is to fix faulty disease-causing genes. But done in a very early embryo, the repair, called germline editing, would be permanent. It could also be passed along to future generations.

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Man-made virus helps blind mice see the light

Posted on: 21 April 2015, source: horizon-magazine.eu
European scientists have reproduced the sensation of sight in blind mice by inserting light-reactive molecules into their optical nerve cells, and are now developing this treatment for use in humans. EU-funded researchers Dr Deniz Dalkara and Dr Jens Duebel at the Vision Institute in Paris, France, have designed a way of adding genes into a mouse's eye so that it responds to light independently of the natural mechinisms of the retina, the part of the eye concerned with light response. They have done this by using a light-sensitive molecule found in single-celled algae, which normally helps the algae swim towards light. The potential of this molecule to activate neurons in other species has been under investigation for years. The Vision Institute researchers have now found a way of capitalising on this, by using a virus to transport the algae-based molecule into the mouse's optical nerve cells.

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Researchers develop a harmless artificial virus for gene therapy

Posted on: 9 April 2015, source: Nanowerk.come
Researchers of the Nanobiology Unit from the UAB Institute of Biotechnology and Biomedicine, led by Antonio Villaverde, managed to create artificial viruses, protein complexes with the ability of self-assembling and forming nanoparticles which are capable of surrounding DNA fragments, penetrating the cells and reaching the nucleus in a very efficient manner, where they then release the therapeutic DNA fragments. The achievement represents an alternative with no biological risk to the use of viruses in gene therapy.

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FDA Approves Further Study Of Promising Gene Therapy HIV Treatment

Posted on: 19 March 2015, source: Towlerode
Experimental stem cell gene therapy that could act as functional cure for HIV infection has been approved by the Food and Drug Administration to move into early human test trials. Unlike other treatments that use healthy stem cells from uninfected donors, this form of therapy uses cells harvested from a positive person’s own body. The stem cells are genetically manipulated to develop into white blood cells that are missing the key cellular receptors that the HIV virus uses to insert its genetic code into healthy cells. The modification effectively models a HIV-positive person’s white blood cells after the cells of people who have a natural resistance to HIV.

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Will gene therapy make humans masters of own evolution?

Posted on: 12 March 2015, source: Discover Maganzine
Human genetic engineering is not new; it has been going on for a long, long time — naturally. Ancient viruses are really good at inserting themselves and modifying human gene code. Over millennia, constant infections would come to mean that 8 percent of the entire human genome is made up of inserted virus code. All this gene recoding of our bodies occurred under Darwin’s rules, natural selection and random mutation. But nonrandom, deliberate human genetic engineering is new, and it is a big deal.

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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.

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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.

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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.

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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.

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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.

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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.

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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

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