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 →

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 →

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 →

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 →

Why it matters to you Duchenne muscular dystrophy is a horrible disease which slowly attacks every muscle in the body. Gene therapy could help treat it -- whether you're a dog or a human. As difficult as it is when our beloved pet dogs get old, its a whole lot worse if their decline is the result of a horrible disease like Duchenne muscular dystrophy, a genetic disorder characterized by progressive muscle degeneration and weakness. Fortunately, a team of gene-editing researchers from France and the U.K. have been working to develop gene therapy as an answer and its one that could help humans, too. Their solution involves using gene therapy to restore muscle strength and stabilize clinical symptoms. This is achieved by way of a shortened version of the dystrophin gene, containing just 4,000 base pairs, which is combined with a viral vector and injected into patients. Duchenne muscular dystrophy is a debilitating muscle-wasting disease affecting young boys and male animals, which is caused by inheritance of a damaged gene, George Dickson, professor of molecular cell biology at University of London, told Digital Trends. There is currently no very effective cure. Our work has involved producing a healthy functioning copy … 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 →