Category Archives: Gene Therapy

By Ben Hirschler LONDON (Reuters) - The science of gene therapy is finally delivering on its potential, and drugmakers are now hoping to produce commercially viable medicines after tiny sales for the first two such treatments in Europe. Thanks to advances in delivering genes to targeted cells, more treatments based on fixing faulty DNA in patients are coming soon, including the first ones in the United States. Yet the lack of sales for the two drugs already launched to treat ultra-rare diseases in Europe highlights the hurdles ahead for drugmakers in marketing new, extremely expensive products for genetic diseases. After decades of frustrations, firms believe there are now major opportunities for gene therapy in treating inherited conditions such as haemophilia. They argue that therapies offering one-off cures for intractable diseases will save health providers large sums in the long term over conventional treatments which each patient may need for years. In the past five years, European regulators have approved two gene therapies - the first of their kind in the world, outside China - but only three patients have so far been treated commercially. UniQure's Glybera, for a very rare blood disorder, is now being taken off the market given … Continue reading →

LONDON (Reuters) - Gene therapy, which aims to patch faulty genes with working DNA, has been a long time in development. The following are major milestones: 1972 - Researchers first suggest gene therapy as a treatment for genetic diseases but oppose its use in humans "for the foreseeable future", pending greater understanding of the technology. 1990 - A four-year-old girl with severe immunodeficiency became the first patient to undergo gene therapy in the United States. 1999 - American patient Jesse Gelsinger dies following a gene therapy experiment, setting the field back several years as U.S. regulators put some experiments on hold. 2002-03 - Cases of leukaemia are diagnosed in French children undergoing gene therapy in a further blow to the field. 2003 - The world's first gene therapy is approved in China for the treatment of head and neck cancer. 2007 - Doctors carry out the world's first operation using gene therapy to treat a serious sight disorder caused by a genetic defect. 2012 - Europe approves Glybera, the first gene therapy in a Western market, for an ultra-rare blood disorder. 2016 - Europe approves Strimvelis for a very rare type of immunodeficiency. 2017 or 2018 - The first gene … Continue reading →

Regenxbio has joined forces with investment firm OrbiMed and a new nonprofit foundation to create Prevail Therapeutics, a startup focused on new biologics and gene therapiesfor Parkinson's disease (PD). Prevail will draw on the expertise of the Silverstein Foundation for Parkinson's with GBA, which concentrates on a particular form of the disease caused by mutations in the glucocerebrosidase gene. The foundation was set up this year by OrbiMed's co-head of private equity Jonathan Silverstein, who was diagnosed with GBA-linked PD in February and is mobilizing efforts to discover a cure for the disease. Silverstein backed the foundation with $10 million of his own money, and is intent on accelerating research into PD with GBA as well as other forms of the disease. Prevail says it will focus initially on research coming out of the lab of its co-founder and CEO Asa Abeliovich, M.D., Ph.D., who is on the faculty of Columbia University as well as being a scientific adviser to the Silverstein Foundation and co-founder of neurodegenerative disease biotech Alector. By joining forces with Regenxbio, Prevail launches with an exclusive license to the gene therapy specialist's adeno-associated virus (AAV) based vector technology NAV AAV9 for PD and other neurodegenerative disorders. … Continue reading →

Pfizer has chosen a site in Sanford, North Carolina for a gene therapy production plant, just 40 miles from its recent acquisition Bamboo Therapeutics Inc. The US drug firm had been search for a site since March. According to North Carolina Governor Roy Cooper, Pfizer will spend $100m (85m) on the new facility and has also committed $4m to support postdoctoral fellowships in North Carolina universities for training in gene therapy research. The project will create jobs that deliver a total payroll impact of more than $3.9m each year to the community according to the North Carolina Department of Commerce and the Economic Development Partnership. The project will be part funded by a $250,000 grant previously awarded to Wyeth which was acquired by Pfizer in 2009 - by the One North Carolina Fund, which helps local Governments attract economic investment. Bamboo buy The decision follows a little over a year after the US drug manufacturer acquired Bamboo Therapeutics, a North Carolina-based gene therapy developer. The deal included a recombinant Adeno-Associated Virus (rAAV) vector design and production technology, a Phase I candidate for Giant Axonal Neuropathy and a preclinical programme targeting Duchenne Muscular Dystrophy (DMD). Pfizer also gained a 11,000sq ft … Continue reading →

A University of Chicago-based research team has overcome challenges that have limited gene therapy and demonstrated how their novel approach with skin transplantation could enable a wide range of gene-based therapies to treat many human diseases. In a study inthe journal Cell Stem Cell, the researchers provide proof-of-concept. They describe gene-therapy administered through skin transplants to treat two related and extremely common human ailments: Type 2 diabetes and obesity. We resolved some technical hurdles and designed a mouse-to-mouse skin transplantation model in animals with intact immune systems, said study author Xiaoyang Wu, assistant professor in the Ben May Department for Cancer Research at the University of Chicago. We think this platform has the potential to lead to safe and durable gene therapy in mice and, we hope, in humans, using selected and modified cells from skin. Beginning in the 1970s, physicians learned how to harvest skin stem cells from a patient with extensive burn wounds, grow them in the laboratory, then apply the lab-grown tissue to close and protect a patients wounds. This approach is now standard. However, the application of skin transplants is better developed in humans than in mice. The mouse system is less mature, Wu said. It … Continue reading →

Alliance for Gene Cancer Therapy Executive Director Margaret C. Cianci and President and CEO John E. Walter outside the nonprofits headquarters in Stamford. Photo by Phil Hall. The development of an experimental gene-targeting therapy in cancer treatment that could be approved for the U.S. market this year was sparked in large part by the research funding support of a Stamford nonprofit. The chimeric antigen receptor T-cell (CAR-T) drug, labeled tisagenlecleucel by its manufacturer, Novartis, in July was unanimously recommended for approval by the oncologic drugs advisory committee of the U.S. Food and Drug Administration. If the FDA grants final approval as expected this fall, it will be the first drug treatment targeting human genes approved for the U.S. market. In Stamford, the Alliance for Cancer Gene Therapy since 2004 has provided a total of $1.8 million to Dr. Carl June at the University of Pennsylvania, the lead researcher in developing the CAR-T therapy. John E. Walter, president and CEO of the Stamford organization, said Junes work has helped to redefine perceptions of what gene therapy can accomplish. Oftentimes, gene therapy is perceived as taking the bad genes out and putting some good genes in, Walter said. In this case, a … Continue reading →

Agilis Biotherapeutics has formed a joint venture with Japans Gene Therapy Research Institution (GTRI). The alliance gives Agilis a base in Japan and a partnership with a fellow CNS specialist to support its development of adeno-associated virus (AAV) vectors and gene therapies. Cambridge, Massachusetts-based Agilis set up the joint venture using a grant from the Japanese government. The agreement will establish an AAV manufacturing facility in Japan, from where Agilis and GTRI will work on vectors using Sf9 baculovirus and HEK293 mammalian cell systems. Agilis and GTRI plan to develop and manufacture AAV gene therapy vectors through the joint venture. Agilis and GTRI also plan is to collaborate on the development and commercialization of certain CNS gene therapies. GTRIs background suggests it is well-equipped to contribute to the project. The Japanese company grew out of the work of Shin-ichi Muramatsu, M.D., a scientist who sequenced AAV3 in the 1990s before going on to create AAVs designed to cross the blood-brain barrier. GTRI is working on gene therapies against diseases including Alzheimers, amyotrophic lateral sclerosis and Parkinsons that build on this research into AAVs. Both biotechs are developing gene therapies to treat aromatic l-amino acid decarboxylase (AADC) deficiency. GTRI aims to … Continue reading →

Weve always said in the past gene editing shouldnt be done, mostly because it couldnt be done safely, said Richard Hynes, a cancer researcher at the Massachusetts Institute of Technology who co-led the committee. Thats still true, but now it looks like its going to be done safely soon, he said, adding that the research is a big breakthrough. What our report said was, once the technical hurdles are cleared, then there will be societal issues that have to be considered and discussions that are going to have to happen. Nows the time. Scientists at Oregon Health and Science University, with colleagues in California, China and South Korea, reported that they repaired dozens of embryos, fixing a mutation that causes a common heart condition that can lead to sudden death later in life. If embryos with the repaired mutation were allowed to develop into babies, they would not only be disease-free but also would not transmit the disease to descendants. The researchers averted two important safety problems: They produced embryos in which all cells not just some were mutation-free, and they avoided creating unwanted extra mutations. It feels a bit like a one small step for (hu)mans, one giant leap … Continue reading →

Genetically engineered skin cells grafted onto mice can treat the animals diabetes and obesity, according to new research published August 2, 2017 in Cell Stem Cell. Researchers edited skin stem cells from newborn mice using CRISPR-based technology so that the cells secreted a peptide that regulates blood sugar. Transplanting the cells onto mice showed the grafts increased insulin secretion and reversed weight gain from a high-fat diet, as well as overturned insulin resistance. The result is a small step toward developing a safe and durable gene therapy to treat diabetes in humans. Weve had this idea for a long time, so its exciting to see that, indeed, it can work to deliver therapeutics, coauthor Xiaoyang Wu, a stem cell biologist at the University of Chicago, tells GEN. In the study, Wu and colleagues worked with skin because it is a large organ and easily accessible. The cells multiply quickly and are easily transplanted. And, transplanted cells can be removed, if needed. Skin is such a beautiful system, Wu says, noting that its features make it a perfect medium for testing gene therapies. The team worked with the gene that produces glucagon-like peptide 1 (GLP-1), a hormone that stimulates the pancreas … Continue reading →

Shares of Spark Therapeuticssurged nearly 20 percentWednesday after the Philadelphia gene therapy company revealed promisingresults from a study of its potential one-time therapy for hemophilia A. Preliminary data from a Phase 1/2 dose-escalation clinical trial of SPK-8011showed human proof-of-concept in three participants, the drug maker said. The encouraging start for hemophilia A reinforces the strength of our gene-therapy platform and positions us well to potentially transform the current treatment approach for this life-altering disease with a onetime intervention, said Katherine A. High, Sparks president and chief scientific officer. Hemophilia is a genetic disorder caused by missing or defective factor VIII, a clotting protein. About 20,000 Americans live with hemophilia. The way the medical community has addressed the disorder is to ensure that patients have continuous injections of blood-clotting factors. Patients infuse themselves two to three times a week for the rest of their lives. In the study, three patientsreceived infusions of vector genomes and no serious adverse events were reported, Spark said. One person has been followed for 23 weeks and another for 12 weeks. The initial dose created stable factor VIII levels with no spontaneous bleeds, the company said. For a third patient, the genome dose was doubled and … Continue reading →