eXTReMe Tracker
Gene Therapy Net RSS feed Follow Gene Therapy Net on Twitter LinkedIn - Gene Therapy Net discussion group Facebook - Gene Therapy Net

International Consortium

Related Links

FDA warns against ‘do it yourself’ gene therapy kits

Posted on: 28 November 2017, source: Bizjournals
The U.S. Food and Drug Administration has issued a stern warning against the use of “do it yourself” gene therapy kits, pushing back against a nascent “biohacker” movement that seeks to make experimental medicines and technologies available to the masses, often by circumventing regulators. A statement posted on the FDA’s website on Tuesday cautions that it is illegal to sell gene therapy products and kits intended for self-administration. Gene therapy involves inserting new, healthy genes into cells to cure diseases that are caused by faulty genes. The FDA has classified gene editing — cutting out and replacing parts of faulty genes — as a form of gene therapy. A host of local biotechs, including Cambridge-based Editas Medicine (Nasdaq: EDIT), are seeking to develop such treatments.

Register to read more ...

Alarmed by Recent Events, Scientists Speak Out Against DIY Gene Therapy

Posted on: 13 December 2017, source: Gizmodo
It seems hard to fathom that scientific progress has advanced to a point where it’s suddenly possible for people to undertake something as complicated as gene therapy outside the safety and confines of a well-funded lab, but it has. In just the past few months we’ve seen not one, but two examples of people pursuing unregulated gene therapy. That’s right, people are genetically modifying their bodies in DIY labs and friend’s apartments. Now, the largest organization of scientists who work in gene and cell therapy have come out against DIY gene therapies. A statement from the American Society of Gene and Cell Therapy comes on the heels of a stern warning against DIY gene therapy from the US Food and Drug Administration. For years, when it came to biohacking, regulators and establishment science have mainly looked the other way. That seems to be changing.

Register to read more ...

First gene-editing attempted in human body to cure disease

Posted on: 21 November 2017, source: The Telegraph
Doctors have edited a patient's genes in a world-first attempt to alter DNA from within the human body. Medics in California administered the experimental intravenous treatment, which provides the body with instructions to change DNA, on Brian Madeux, who suffers from Hunter's syndrome. People with Hunter's syndrome are unable to break down long sugary molecules called mucopolysaccharides. This leads to the molecules building up and causing brain and organ damage, and in severe cases it can be fatal. Sufferers require regular enzyme replacement therapy to break down the molecules, but Mr Madeux said he opted to take part in a trial as he is "in pain every second of the day". The 44-year-old from Arizona underwent the treatment at UCSF Benioff Children's Hospital in Oakland, California on Monday.

Register to read more ...

Gene therapy helps a boy get new skin

Posted on: 21 November 2017, source: Biothin.Asia
A medical team at the Ruhr-Universität Bochum’s burn unit and the Center for Regenerative Medicine at the University of Modena (Italy) were the first ever to successfully treat a child suffering from extensive skin damage using transplants derived from genetically modified stem cells. The boy is a so-called butterfly child: he suffers from epidermolysis bullosa, a genetic skin disease that had destroyed approximately 80 percent of his epidermis. After all established therapies had failed, the medical team from Bochum decided to try an experimental approach: they transplanted skin derived from genetically modified stem cells onto the wound surfaces. Thanks to the successful therapy, the boy is now – two years after the treatment – able to participate in his family’s life and social life. The scientists published their report in “Nature”.

Register to read more ...

Gene Therapy in Recurrent Glioma Shows Promise in Early Trial

Posted on: 7 November 2017, source: CancerNetwork
More than 25% of patients with recurrent, high-grade glioma treated with a gene therapy combination were alive more than 3 years after treatment, according to data from a subset of patients in a phase I clinical trial presented at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics, held October 26–30 in Philadelphia. The combination treatment involved the retroviral vector Toca 511 and the prodrug of the chemotherapy 5-fluorouracil (Toca FC). Toca 511 is a replicating virus that only infects actively dividing tumor cells. Once inside the cancer cell, the virus delivers a gene for an enzyme, cytosine deaminase (CD). As the virus replicates and spreads to other cancer cells, it programs them to make CD. Next, patients receive a pill, Toca FC, which is an inert compound. Once inside the cancer cell, CD converts Toca FC into the anticancer drug 5-fluorouracil, which kills the cancer cell. 5-fluorouracil also kills certain immune suppressive myeloid cells, boosting the patient’s immune system to recognize and attack the cancer cells.

Register to read more ...

FDA approves a game-changing treatment for blood cancer

Posted on: 20 October 2017, source: StatNews
The Food and Drug Administration on Wednesday approved a promising new treatment for a particularly deadly form of cancer, bringing hope to desperate patients while rekindling a global conversation about the escalating cost of new therapies. The treatment, made by Gilead Sciences, is made by extracting patients’ white blood cells and re-engineering them to home in on tumors. Called a CAR-T, the one-time treatment has shown unprecedented results for patients with dire diagnoses. Gilead’s treatment, to be sold as Yescarta, is the second CAR-T to win FDA approval but the first cleared for use in adults. A similar therapy from Novartis, approved in August, is used for children with an aggressive form of blood cancer.

Register to read more ...

FDA experts offer a unanimous endorsement for pioneering gene therapy for blindness

Posted on: 13 October 2017, source: ScienceMag
A pioneering AAV gene therapy from Spark Therapeutics took a giant stride toward an FDA approval yesterday as an outside panel of experts offered their support for getting this game-changing treatment into the market after looking over the data and hearing from some of the severely sight-impaired patients whose lives had been transformed by this therapy. The vote was 16 to 0 favoring the benefit-risk profile of the drug, backing an OK for voretigene neparvovec by the agency’s Cellular, Tissue and Gene Therapies Advisory Committee and providing a compelling reason for the U.S. Food and Drug Administration to follow through with an historic first U.S. approval of a vector-delivered gene therapy.

Register to read more ...

DNA surgery on embryos removes disease

Posted on: 5 October 2017, source: BBC News
Precise "chemical surgery" has been performed on human embryos to remove disease in a world first, Chinese researchers have told the BBC. The team at Sun Yat-sen University used a technique called base editing to correct a single error out of the three billion "letters" of our genetic code. They altered lab-made embryos to remove the disease beta-thalassemia. The embryos were not implanted. The team says the approach may one day treat a range of inherited diseases.

Register to read more ...

Gene therapy reverses multiple sclerosis in mice

Posted on: 25 September 2017, source: BioNews
A novel gene therapy can reverse the symptoms and progression of disease in a mouse model of multiple sclerosis (MS). The neurodegenerative illness is caused by the body's own immune system degrading and destroying nerve cells. US researchers have used a gene therapy approach in the livers of affected mice to produce more regulatory T-cells and reduce this autoimmune response. 'Using a clinically tested gene therapy platform, we are able to induce very specific regulatory cells that target the self-reactive cells that are responsible for causing multiple sclerosis,' said Dr Brad Hoffman at the University of Florida, College of Medicine in Gainesville, who led the study. 'Most current therapies for autoimmune diseases such as multiple sclerosis are based on general immune suppression, which has various side effects or complications.'

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...

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."

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...

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.

Register to read more ...