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Could gene therapy help you live forever? CEO of controversial firm claims she has successfully carried out first anti-ageing treatment - on herself

Posted on: 27 April 2016, source: DailyMail
Eternal youth could be one step closer. A woman from Seattle claims to have become the first human to have become younger using gene therapy. Elizabeth Parrish, CEO of controversial firm Bioviva, says she has been using her own company's experimental gene therapies to battle ageing - but not everyone is convinced of the results. The self-treatment, which has been ongoing for the past six months, has been met with a mixture of praise for innovation and criticism for a lack of scientific rigour. But now, Parrish claims to have become the first human being to be successfully rejuvenated by reversing 20 years of normal telomere shortening.

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U.S. FDA reports rise in gene and cell therapy applications

Posted on: 19 April 2016, source: RAPS
The US Food and Drug Administration’s Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) met in February to discuss updates of research programs in the Office of Cellular, Tissue and Gene Therapy (CTGT), which is part of FDA’s Center for Biologics Evaluation and Research (CBER). The research updates came as Raj Puri, MD, PhD, Director, Division of Cellular, and Gene Therapies (DCGT) at CBER told the committee that although his division has only licensed 12 products in its entire history, more than 1,530 active investigational new drugs (INDs), investigational device exemptions (IDEs) and master files are currently under review, and 106 of those INDs deal specifically with natural killer cells, which play a role in the rejection of tumors and virally-infected cells.

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Chinese Scientists Defy Ethics, Double Down on Editing Human Embryos

Posted on: 14 April 2016, source: GEN
In April of 2015, Chinese researchers announced to the world that they had used the new genome-editing technology CRISPR/Cas9 on human preimplantation embryos to modify the HBB gene, mutations of which have been implicated in the disease β-thalassemia. The news had sparked a media firestorm and eventually led scientists from across the globe to discuss intently the ethics of such studies, ultimately leading to the decision for an international moratorium on the use of CRISPR with human embryos.

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EU Regulatory Panel Recommends Approval Of Glaxo's Gene Therapy For Bubble Boy Disease

Posted on: 3 April 2016, source: TechTimes
An EU regulatory panel has recommended the approval of a gene therapy from GlaxoSmithKline (GSK) for the treatment of Bubble Boy Disease. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), together with the Committee for Advanced Therapies (CAT) issued a positive opinion last April 1 with regards to marketing the drug called Strimvelis for the treatment of ADA-SCID (Adenosine Deaminase Deficiency-Severe Combined Immunodeficiency).

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Dal researcher develops technique for enhancing gene therapy

Posted on: 5 March 2016, source: Dalhousie University
Using his knowledge of how genes are organized and repaired in human cells, Dr. Graham Dellaire, Dalhousie Medical School’s Cameron Research Scientist in Cancer Biology, has developed a technique that could make gene therapy more effective and safer to use. His work was recently published in Nucleic Acids Research and Nature.

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Gene therapy in the womb for ill babies: Plan to give injections to mothers whose unborn children have stopped growing due to failure of the placenta

Posted on: 11 March 2016, source: Daily Mail
Desperately ill babies could have their lives saved by a pioneering treatment given while they are still in the womb. In a world first, doctors at a British hospital plan to give injections of genes to mothers-to-be whose unborn children have stopped growing. It is hoped that the genes will boost blood flow to the placenta, the lifeline between mother and baby, providing the child with the oxygen and nutrients needed to thrive and survive.

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Researchers to use nano-tech in gene therapy of cancer cells

Posted on: 5 March 2016, source: Mehr News
Iranian researchers of Tabriz University of Medical Sciences managed to apply laboratory synthesis of nano-polymeric carriers which can be used in gene therapy of cancer cells. Gene therapy is an experimental technique through which defective genes are modified and sent into the cell by a carrier. Vectors used to transfer genes are divided into two Viral and Non-viral categories.

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U.S. Intelligence Official Calls Gene Editing a Threat

Posted on: 14 February 2016, source: Technology Review
That’s according to James Clapper, U.S. director of national intelligence, who on Tuesday, in the annual worldwide threat assessment report of the U.S. intelligence community, added gene editing to a list of threats posed by “weapons of mass destruction and proliferation.” Gene editing refers to several novel ways to alter the DNA inside living cells. The most popular method, CRISPR, has been revolutionizing scientific research, leading to novel animals and crops, and is likely to power a new generation of gene treatments for serious diseases. It is gene editing’s relative ease of use that worries the U.S. intelligence community, according to the assessment. “Given the broad distribution, low cost, and accelerated pace of development of this dual-use technology, its deliberate or unintentional misuse might lead to far-reaching economic and national security implications,” the report said.

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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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