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Gene Therapy 2016: The Pipeline Is Swelling

Posted on: 2 September 2016, source: GEN
Door Has Been Opened to a Range of Novel Biological Therapies for Human Diseases. Twenty-seven years after Steven A. Rosenberg, M.D., Ph.D., at the National Cancer Institute Bethesda campus performed the first human gene transfer experiment, gene therapy is poised to take its place in the armamentarium of biologic therapies available to treat human diseases.
Some examples: the EMA-approval in November 2012 of Glybera® (Uniqure), an intramuscular recombinant adeno-associated virus (rAAV) product for treatment of lipoprotein lipase deficiency; and the recently announced Phase II trial results showing efficacy of another rAAV product for a genetic cause of blindness (LCA2)—for which a Biologics Licensing Application is anticipated in the U.S. this year.
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Novel Gene Therapy Drug for Huntington's Disease

Posted on: 25 August 2016, source: PRNewswire
Data from Vybion on a novel treatment for Huntington's disease has been published in the Journal of Neurodegenerative diseases. The published study links the ability of Vybion's proprietary, novel Intrabody (INT41) blocking of cellular gene dysregulation to the delay of cognitive and motor function loss in the well-validated vR6/2 animal model. INT41 interferes with direct binding of toxic N-terminal huntingtin fragments to DNA, as well as their transport into the nucleus. The data support a direct gain of function of N-terminal huntingtin protein fragments that may lead to neuron dysfunction and brain atrophy as well as a novel therapeutic modality.
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FDA Grants Breakthrough Designation to AveXis's Gene Therapy

Posted on: 24 July 2016, source: DDD-Mag
AveXis, a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for AVXS-101, the company's lead development candidate for the treatment of spinal muscular atrophy (SMA) Type 1 in pediatric patients. The Breakthrough Therapy Designation is based on preliminary clinical results from the ongoing trial of AVXS-101, conducted in collaboration with The Research Institute at Nationwide Children's Hospital and The Ohio State University.
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Ziopharm Confirms Three Patient Deaths in Gene Therapy Trial

Posted on: 16 July 2016, source: GEN
Zopharm Oncology has disclosed the deaths of three patients in a Phase I study of its gene therapy candidate Ad-RTS-hIL-12 plus oral veledimex for a form of brain cancer. The third patient died of an intracranial hemorrhage that occurred sometime following discharge from a treating center for the Phase I study, Ziopharm said today in a statement. The study is designed to assess the combination of Ad-RTS-hIL-12 with orally administered veledimex in patients with recurrent or progressive glioblastoma (GBM) or grade III malignant glioma.
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Gene-therapy trials must proceed with caution

Posted on: 1 July 2016, source: Nature
The perils of the past must not be allowed to happen again. Jesse Gelsinger was 18 and healthy when he died in 1999 during a gene-therapy experiment. He had a condition called ornithine transcarbamylase deficiency (OTC), but it was under control through a combination of diet and medication. Like others with the disorder, Gelsinger lacked a functional enzyme involved in breaking down ammonia, a waste product of protein metabolism that becomes toxic when its levels become too high. The gene therapy that he received used a viral vector to introduce a normal gene for the enzyme. Gene therapy remains an obvious route to treat OTC. Simply adding the missing gene has been shown to repair metabolism in mice. But the memory of what happened to Gelsinger has slowed progress in gene therapy for any condition.
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Gene therapy drug approval granted to GSK

Posted on: 29 May 2016, source: BBC
Regulators have given one of the world's largest drug companies approval to sell a new gene therapy. The treatment is for an illness called ADA-SCID which prevents babies from fighting off everyday infections. This is the first approval for a genetic therapy granted to a large multinational drug company, GSK. Commentators say the development marks the beginning of many more genetic medicines from so-called "Big Pharma". The condition is extremely rare and affects around two dozen babies each year. Approval of the gene therapy paves the way for the development of treatments for more widespread illnesses such as thalassemia and sickle cell disease. Hundreds of inherited disorders such as cystic fibrosis, muscular dystrophy and many types of blindness are caused by faulty genes.
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Federal advisory committee greenlights first CRISPR clinical trial

Posted on: 22 June 2016, source: Nature
CRISPR, the genome-editing technology that has taken biomedical science by storm, is finally nearing human trials. On 21 June, an advisory committee at the US National Institutes of Health (NIH) approved a proposal to use CRISPR–Cas9 to help augment cancer therapies that rely on enlisting a patient’s T cells, a type of immune cell. “Cell therapies for cancer are so promising but the majority of people who get these therapies have a disease that relapses,” says study leader Edward Stadtmauer, a physician at the University of Pennsylvania in Philadelphia. Gene editing could improve such treatments and eliminate some of their vulnerabilities to cancer and the body’s immune system, he says.
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Abeona Therapeutics Doses First Patient in Phase 1/2 Clinical Trial for Sanfilippo Syndrome Type A

Posted on: 19 May 2016, source: Abeona Therapeutics
Cleveland-based Abeona Therapeutics has dosed its first patient in a Phase 1/ 2 Trial with ABO-102 gene therapy for patients with Sanfilippo syndrome type A (a/k/a Mucopolysaccharidosis Type IIIA or MPS IIIA). ABO-12, a single intravenous injection, is an adeno-associated viral (AAV)-based gene therapy to treat the central nervous system and the peripheral disease manifestations in Sanfilippo patients, representing a new approach to addressing the progressive disease which often prevents children from reaching adulthood. The drug is based on more than 18 years of research and development. There are currently no approved treatments available for Sanfilippo syndrome type A.
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Breakthrough Gene Therapy Reverses Vision Loss In Patients With Inherited Blindness

Posted on: 30 April 2016, source: TechTimes
A genetic therapy offers a glimmer of hope for patients with a rare form of blindness. In a clinical study conducted in Oxford University in the United Kingdom, the therapy restored vision in the patients for as long as four years, potentially benefiting not only those with the type of genetic blindness but also those afflicted by common causes of vision loss.
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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:
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:
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.

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.

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.

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.