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Gene therapy for cancer halted after patient death

Posted on: 13 September 2017, source: BioNews
Two trials assessing gene therapy for blood cancer have been put on hold by the US Food and Drug Administration (FDA), following a patient fatality. The therapy, known as 'off-the-shelf' CAR-T immunotherapy, used genetically modified immune system T cells to target cancer, yet unlike other trials the cells were taken from a healthy donor instead of the patient. It was hoped this therapy would prove easier and less expensive to produce.

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FDA makes the first gene therapy Kymriah available for treatment of pediatric ALL

Posted on: 31 August 2017, source: News Medical Life Sciences
The U.S. Food and Drug Administration (FDA) has issued a press release making the first even gene therapy available for treatment of children with a form of acute lymphoblastic leukemia (ALL). The drug Kymriah (tisagenlecleucel) is the first of its kind that uses gene therapy to treat patients with this condition. The release was updated yesterday identifying the designantions that were granted to Kymriah.

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Gene Therapy Corrects Factor VIII Levels in Hemophilia A

Posted on: 7 August 2017, source: Medscape
Patients with hemophilia A who received a gene for coagulation factor VIII (FVIII) packaged in a viral vector had sustained levels of FVIII during 1 year of observation, eliminating spontaneous bleeds[2]. Most of them did not need FVIII infusions even in the case of major trauma or surgery. In a late-breaking abstract session here at the International Society on Thrombosis and Hemostasis 2017 Congress, Dr John Pasi (Barts and the London School of Medicine and Dentistry, Queen Mary University of London, UK) said six out of the seven patients in one dose cohort have achieved normal factor VIII levels.

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First gene therapy — 'a true living drug' — on the cusp of FDA approval

Posted on: 14 July 2017, source: Chicago Tribune
Food and Drug Administration advisers on Wednesday enthusiastically endorsed a first-of-its-kind cancer treatment that uses patients' revved-up immune cells to fight the disease, concluding that the therapy's benefits for desperately ill children far outweigh its potentially dangerous side effects. The unanimous recommendation from the Oncologic Drugs Advisory Committee means the treatment could be approved by the FDA by the end of September, forging a new path in the immunotherapy frontier.

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South Korea approves first gene therapy drug

Posted on: 12 July 2017, source: Yonhap News
South Korea's health authorities said Wednesday that they have approved the use of the country's first gene therapy drug to help patients with degenerative joint disease. The drug, Invossa, manufactured by local drug firm Kolon Life Science Inc. has obtained the green light from the Ministry of Food and Drug Safety for sales in the domestic market. Invossa is the world's first cell-medicated gene therapy for osteoarthritis, a disorder involving movable joints. The company said the drug can be injected into the joint cavity of a patient. The drug has proven to reduce osteoarthritis pain and improve cartilage through clinical trials, said Kolon Life Science, expecting to help an estimated five million patients in the country.


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Gene Therapy & Mesothelioma

Posted on: 18 June 2017, source: mesothelioma.com
Technological innovations are changing the world we live in every day, including the way society views diseases that have been around for hundreds of years. Cancer continues to be a leading cause of death around the world, but day by day we, as a society, are getting closer to finding a cure. In 2016, former President Barack Obama asked former Vice President Joe Biden to head the Cancer Moonshot Initiative (CMI), whose overarching goal would be to find a cure for cancer by the year 2020. In the time that’s passed, Biden has formed a committee of governmental organizations and a Blue Ribbon Panel that provided recommendations of how to achieve the lofty goals, and set to bringing this moonshot into reality.

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Altered virus may expand patient recruitment in human gene therapy trials

Posted on: 14 June 2017, source: Science Daily
For many patients, participating in gene therapy clinical trials isn't an option because their immune system recognizes and fights the helpful virus used for treatment. Now, University of Florida Health and University of North Carolina researchers have found a solution that may allow it to evade the body's normal immune response.

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Allergy Breakthrough with Gene Therapy

Posted on: 8 June 2017, source: World Health
It might soon be possible for a single treatment to provide life-long protection against harsh allergies including asthma. An immunology research team at The University of Queensland led by Professor Ray Steptoe has figured out how to disable the immune response that triggers allergic reactions. The research team operates out of the university's Diamantina Institute. Professor Steptoe's lab is situated at the Translational Research Institute. The research was funded by the National Health and Medical Research Council and the Asthma Foundation. The research team's findings are published in JCI Insight.

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European Child Receives Strimvelis Gene-Therapy Treatment

Posted on: 3 May 2017, source: Rare Disease Report
Before March, only 1 patient had ever been treated via commercial gene therapy. GlaxoSmithKline (GSK) doubled that total with their announcement this week. In 2015, a European patient with familial chylomicromenia received Glybera to restore their LPL enzyme levels. On Tuesday, GSK announced that it treated its first patient in March, almost a full calendar year after their drug, Strimvelis, was approved for sale. Strimvelis is assumed to provide an outright cure for Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency (ADA-SCID), a rare disorder caused by the absence of the essential protein adenosine deaminase (ADA). ADA is required to produce lymphocytes. It leaves babies without a fully-functioning immune system and extremely vulnerable to infections.

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uniQure Announces It Will Not Seek Marketing Authorization Renewal for Glybera in Europe

Posted on: 20 April 2017, source: UniQure
uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will not pursue the renewal of the Glybera® (alipogene tiparvovec) marketing authorization in Europe when it is scheduled to expire on October 25, 2017. "The decision to not pursue marketing authorization renewal of Glybera in Europe involved a thoughtful and careful evaluation of patient needs and the clinical use of the therapy, and is not related to any risk-benefit concern," stated Matthew Kapusta, chief executive officer of uniQure. "Glybera's usage has been extremely limited and we do not envision patient demand increasing materially in the years ahead."

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Interview with Daniel Leonard, Director, Global Patient Advocacy, UniQure

Posted on: 11 April 2017, source: Gene Therapy for Rare Disorders 2017
Daniel Leonard is the Director of Global Patient Advocacy at uniQure, a pioneering company in gene therapy. Prior to joining uniQure, Dan spent 16 years at Genzyme, a company that was known as a leader in patient centricity. In his current role Dan works primarily with hemophilia organizations in the US and abroad. In this role Dan serves as an ambassador to the patient community, but also as an internal advocate for patients’ interests. Daniel recently sat down with David Snowdon, Program Director of Gene Therapy for Rare Disorders to discuss his work in this space and how he sees the field evolving. Full interview.

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AVROBIO, Inc. Expands Rare Disease Pipeline with Gene Therapy to Treat Gaucher Disease

Posted on: 23 March 2017, source: press release
AVROBIO, Inc., a clinical-stage biotechnology company developing transformative, life-changing gene therapies for rare diseases, today announced the expansion of its pipeline to Gaucher disease. This late-stage preclinical program becomes AVROBIO’s second therapy for lysosomal storage disorders (LSDs), following on the heels of the Company’s Phase 1 Fabry program. The program for Gaucher disease was licensed from Dr. Stefan Karlsson at Lund University in Sweden, where preclinical proof-of-concept has been demonstrated. Terms of the license agreement were not disclosed.

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Doctors reverse teen's sickle cell disease with innovative gene therapy

Posted on: 5 March 2017, source: normangeestar.net
A 16-year-old boy in France has become the first person ever to be successfully treated with a new first-of-its-kind gene therapy for sickle cell disease. Doctors removed the teen's bone marrow and genetically altered it in the laboratory to correct the defect in his DNA that causes sickle cell disease. The study stated 15 months after the very first treatment that the boy who once had significant internal damage - so much so that it led to a removed spleen and a hip replacement - now has no sign of the disease.

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Researchers Use Gene Therapy To Restore Hearing In Deaf Mice

Posted on: 13 February 2017, source: Medical News
A team of researchers at Harvard Medical School and Boston Children's Hospital reported back in 2015 that they were able to restore rudimentary hearing in genetically deaf mice using gene therapy. The Boston Children's hospital research team recently reports that they have restored a much higher level of hearing - down to 25 decibels, the equivalent of a whisper. They used an improved gene therapy vector developed at Massachusetts Eye and Ear. Although previous vectors have only been able to penetrate the cochlea's inner hair cells, the first Nature Biotechnology study reveals that a new synthetic vector, "Anc80," is capable of transferring genes to the inaccessible outer hair cells when introduced into the cochlea.

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Sarepta eyes a different approach to Duchenne: Gene Therapy

Posted on: 10 January 2017, source: Business Journal
After winning approval in September for the first-ever Duchenne muscular dystrophy drug, Cambridge-based Sarepta Therapeutics is broadening its approach to treating the disease through a pair of gene therapy programs. Sarepta (Nasdaq: SRPT) announced Tuesday that it had reached an agreement with Ohio-based Nationwide Children’s Hospital to exclusively license a pre-clinical program that seeks to treat DMD through gene therapy. The company also said it had entered into a research agreement with the hospital to develop a second pre-clinical gene therapy program, with an exclusive option to license it later.

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New Gene Therapy Can Reverse Aging Process

Posted on: 18 December 2016, source: University Herald
The results of the latest research on gene therapy done by a team of scientists at the Salk Institute in La Jolla, California looked like it's part of a science fiction movie. Better yet it's reminiscent of a Mel Gibson movie, Forever Young, where the main character has remained young over the decades. What the team has discovered was a new form of gene therapy which showed signs that it can reverse the aging process. They conducted the experiment on aging lab rats which showed signs of rejuvenation after undergoing the said treatment for six weeks. The most notable effects they noticed were improved cardiovascular health, straighter spines, and fast regeneration when hurt.

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After one dose of gene therapy, hemophilia B patients maintain near-normal levels of clotting factor

Posted on: 6 December 2016, source: Science Daily
Researchers are reporting the highest and most sustained levels to date of an essential blood-clotting factor IX in patients with the inherited bleeding disorder hemophilia B. After receiving a single dose of an experimental gene therapy in a clinical trial, patients with hemophilia produced near-normal levels of clotting factor IX, allowing them to stop clotting factor infusions and to pursue normal activities of daily life without disabling bleeding episodes.

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American Society of Gene & Cell Therapy Issues ‘White Paper’ Explaining, Supporting Treatment

Posted on: 1 December 2016, source: ASGCT
People interested in knowing more about gene editing and its therapeutic applications can now download a document prepared by the American Society of Gene & Cell Therapy (ASGCT). The document, titled 'Therapeutic Gene Editing: An ASGCT White Paper.' is intended as background information for policymakers, patients, and the general public to help them better understand the concept and its related therapies.
Its release is intended to assist people in understanding a report on human gene editing by the National Academy of Sciences and National Academy of Medicine. That report, expected in early 2017, will likely cover the ethical, legal, and social implications of gene editing processes and, possibly, to guide policy.

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Researchers take step toward gene therapy for sickle cell disease

Posted on: 8 November 2016, source: MedicalXpress
A team of researchers at the Stanford University School of Medicine has used a gene-editing tool known as CRISPR to repair the gene that causes sickle cell disease in human stem cells, which they say is a key step toward developing a gene therapy for the disorder. The team went on to demonstrate that the mended cells could make a functioning hemoglobin molecule, which carries oxygen in normal red blood cells, and then successfully transplanted the stem cells into mice. The researchers say the study represents a proof of concept for the repair of blood-borne genetic diseases, such as sickle cell disease and thalassemia.

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Gene therapy for blistering skin disease appears to enhance healing in clinical trial

Posted on: 3 November 2016, source: ScienceBlog
Grafting sheets of a patient’s genetically corrected skin to open wounds caused by the blistering skin disease epidermolysis bullosa appears to be well-tolerated and improves wound healing, according to a phase-1 clinical trial conducted by researchers at the Stanford University School of Medicine. The results mark the first time that skin-based gene therapy has been demonstrated to be safe and effective in patients. The findings will be published Nov. 1 in JAMA. Associate professors of dermatology Peter Marinkovich, MD, and Jean Tang, MD, PhD, share senior authorship of the study. Senior scientist Zurab Siprashvili, PhD, is the lead author.

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HIV Cure: Portable 'Gene Therapy In A Box' A Breakthrough In Fight Against AIDS, Cancer?

Posted on: 24 October 2016, source: iTechPost
Human Immunodeficiency Syndrome, better known as the HIV virus is one of the world's most notorious sexually-transmitted diseases. If left untreated, it can lead to AIDS or Acquired Immunodeficiency Syndrome. Going back to its history, HIV infection has existed in the US at least since the mid-to-late 1970s. Recently, it has been found that gene therapy can now be made possible even for the poorest of countries. Through the help of a handheld portable device that has been discovered, medical personnel can manipulate a patient's blood to carry out potential new medications for cancer, HIV and other types of diseases.

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Smallest-reported artificial virus could help advance gene therapy

Posted on: 10 October 2016, source: EurekAlert
Gene therapy is a kind of experimental treatment that is designed to fix faulty genetic material and help a patient fight off or recover from a disease. Now scientists have engineered the smallest-reported virus-like shell that can self-assemble. It could someday carry potentially therapeutic DNA or RNA and transfer it to human cells. The report appears in the Journal of the American Chemical Society.

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New gene therapy technique can stop the spread of malignant cancer cells

Posted on: 20 September 2016, source: India Times
MIT scientists have developed a new gene therapy technique that can stop the spread of malignant cells around the body - the leading cause of mortality in women with breast cancer. The treatment uses microRNAs - small noncoding RNA molecules that regulate gene expression - to control the spread of cancer cells around the body, known as metastasis. The therapy could be used alongside chemotherapy to treat early-stage breast cancer tumours before they spread, according to Natalie Artzi, a principal research scientist at Massachusetts Institute of Technology (MIT) in the US.

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Novartis Drops Gene Therapy Research

Posted on: 8 September 2016, source: Reuters
Healthcare company Novartis has either fired or reassigned roughly 400 researchers previously tasked with developing cell and gene therapies. While most of those scientists will be employed by the company elsewhere, 120 positions will be eliminated, according to Reuters. The Cell and Gene Therapies Unit, led by Usman Azam, attracted attention for a promising experimental therapy based on chimeric antigen receptor T-cells (CAR-T), called CTL109, designed to treat lymphomas in children, reports BusinessInsider.

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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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