‘Major Advance’: Leukemia Treatment Could be First U.S. Gene Therapy

A treatment for a common childhood blood cancer could become the first gene therapy available in the U.S. A Food and Drug Administration advisory panel voted 10-0 on Wednesday in favor of the leukemia treatment developed by the University of Pennsylvania and Novartis Corp. The FDA usually follows recommendations from its expert panels but isn’t obligated to do so. The therapy could be the first of a wave of treatments custom-made to target a patient’s cancer. Called CAR-T, this type of therapy involves removing immune cells from a patients’ blood, reprogramming them to create an army of cells that can zero in on and destroy cancer cells and injecting them back into the patient. “This is a major advance,” said panel member Dr. Malcolm A. Smith of the National Cancer Institute. He said the treatment is “ushering in a new era.”
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New Partnership to Advance Novel Airway-Delivered Gene Therapy for Treating Pulmonary Hypertension

Mount Sinai has partnered with Theragene Pharmaceuticals, Inc. to advance a novel airway-delivered gene therapy for treating pulmonary hypertension (PH), a form of high blood pressure in blood vessels in the lungs that is linked to heart failure. If the therapy succeeds in human clinical trials, it will provide patients for the first time with a way to reverse the damage caused by PH. This gene therapy technique comes from the research of Roger J. Hajjar, MD, Professor of Medicine and Director of the Cardiovascular Research Center at the Icahn School of Medicine at Mount Sinai, and has been proven effective in rodent and pig animal models. PH is a deadly disease that disproportionately affects young adults and women; 58 percent of cases are found in young adults and 72 percent are women. There is currently no effective cure for PH, and about 50 percent of people who are diagnosed will die from the disease within five years.
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First Gene Therapy — ‘a True Living Drug’ — on the Cusp of FDA Approval

PHILADELPHIA — When doctors saw the report on Bill Ludwig’s bone-marrow biopsy, they thought it was a mistake and ordered the test repeated. But the results came back the same: His lethal leukemia had been wiped out by an experimental treatment never used in humans. “We were hoping for a little improvement,” remembers the 72-year-old retired New Jersey corrections officer, who had battled the disease for a decade. He and his oncologist both broke down when she delivered the good news in 2010. “Nobody was hoping for zero cancer.” The pioneering therapy with Ludwig and a few other adults at the University of Pennsylvania hospital paved the way for clinical trials with children. Six-year-old Emily Whitehead, who was near death, became the first pediatric recipient in 2012. Like Ludwig, she remains cancer-free. Such results are why the treatment is on track to become the first gene therapy approved by the Food and Drug Administration. An FDA advisory committee will decide Wednesday whether to recommend approval of the approach, which uses patients’ own genetically altered immune cells to fight blood cancers.
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Gene Therapy for Juvenile Batten Disease Gets Orphan Drug Designation

The FDA has granted Abeona Therapeutics Inc. Orphan Drug Designation (ODD) for ABO-201 (AAV-CLN3), its gene therapy program for juvenile Batten disease, (juvenile neuronal ceroid lipofuscinosis, JNCL). The AAV-based gene therapy is being designed as a single intravenous infusion to treat children with the currently fatal rare disease. Simply put, the gene therapy is being developed not to treat these children, but to cure them. This is the 4th gene therapy the company has in development that has obtained an Orphan Drug Designation. The other 3 are being developed to treat recessive dystrophic epidermolysis bullosa and both sanfilippo syndrome type A and type B. The company also has a variety of other gene therapies in development for other rare diseases.
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One Step Closer to a DNA Vaccine Against Dengue Virus

In a new study, researchers inoculated mice with a new DNA vaccine candidate (pVAX1-D1ME) in order to evaluate its efficiency. They found that the vaccine candidate was able to induce persistent humoral and cellular immune responses and provided efficient protection against lethal challenge from one of the four serotypes of dengue virus (DV1). They also evaluated the immunoprotective potential of a combined (bivalent) DNA vaccine, which was found to generate a balanced immunogenic response to two serotypes of dengue virus (DV1 & DV2). These results are encouraging for the future development of a tetravalent vaccine that could provide efficient protection against all four serotypes of the virus. "Our DNA vaccine candidate induced effective immune responses and protection in mice. Importantly, the bivalent vaccine generated a balanced immunity against DV1 and DV2 infection, which emits light for development of new type of tetravalent vaccine against dengue viruses," says corresponding author Dr. Jing An (Capital Medical University, China). "However, it was noted that the end-point titers of anti-DV1 and anti-DV2 in the bivalent vaccine-immunized mice were lower than those in the monovalent vaccine-immunized mice, indicating interference between the DV1 and DV2 vaccine candidates. This evidence should be considered in further research on dengue virus tetravalent vaccine."
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Immunotherapy with DNA Vaccine Shows Promise for HPV-Related Head and Neck Cancer

A novel vaccine therapy can generate immune responses in patients with head and neck squamous cell carcinoma (HNSCCa), according to researchers at the Abramson Cancer Center of the University of Pennsylvania. The treatment specifically targets human papillomavirus (HPV), which is frequently associated with HNSCCa, to trigger the immune response. Researchers will present the results of their pilot study during the 2017 American Society of Clinical Oncology Annual Meeting in Chicago. HNSCCa is a cancer that develops in the mucous membranes of the mouth, and throat. While smoking and tobacco use are known causes, the number of cases related to HPV infection -- a sexually transmitted infection that is so common, the Centers for Disease Control says almost all sexually active adults will contract it at some point in their lifetimes -- is on the rise. The CDC now estimates 70 percent of all throat cancers in the United States are HPV-related. Sixty percent are caused by the subtype known as HPV 16/18.
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Bluebird Reports Early Results From Upgraded Gene Therapy

One of the year’s most closely watched clinical studies could lead to a landmark approval of a gene therapy and throw wide open the debate over how to pay for expensive drugs. The first drips of data have emerged. Bluebird Bio (NASDAQ: BLUE) says the first three patients—of 15 total expected—have had good results from a revised version of its LentiGlobin gene therapy to treat certain genetic variants of the rare blood disease beta-thalassemia, which causes severe anemia and requires frequent transfusions. Bluebird has changed the way it manufactures the product, which requires extracting a patient’s bone marrow cells, altering their DNA outside the body, then reintroducing the cells to the patient. This study, called NORTHSTAR-2, is the first test of the improved process, which regulators said last year would not require rewinding its clinical program back to the beginning—a sigh of relief at the time for the company and its shareholders.
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Gene Therapy Biotech AveXis Targets SOD1 ALS

AveXis is one step closer to developing a potential gene therapy for SOD1 ALS. The gene therapy company, based in Cleveland, Ohio, announced this month it has obtained the rights to develop treatments for ALS using REGENXBIO’s gene therapy delivery vehicle. The emerging vector, derived from adeno-associated virus 9 (AAV9), is being increasingly utilized to deliver potential therapies into the CNS for neurological diseases. The strategy is one of a growing number of potential gene therapies for SOD1 ALS that aims to reduce levels of misfolded SOD1 in the CNS and in the muscles by silencing the expression of the SOD1 gene (see May 2017 conference news). The approach is being developed by a research team led by Nationwide Children’s Hospital’s Brian Kaspar in Ohio, who is also AveXis’ chief scientific officer and scientific founder (see December 2015 conference news; Thomsen et al., 2014; Foust et al., 2013).
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DNA Vaccine Protects Against Toxic Proteins Linked to Alzheimer’s

A new DNA vaccine when delivered to the skin prompts an immune response that produces antibodies to protect against toxic proteins associated with Alzheimer's disease -- without triggering severe brain swelling that earlier antibody treatments caused in some patients. Two studies from the Peter O'Donnell Jr. Brain Institute demonstrate in animals how a vaccine containing DNA of the toxic beta-amyloid protein elicits a different immune response that may be safe for humans. The vaccine, which will likely be tested further by the U.S. Food and Drug Administration, is on a shortlist of promising antibody treatments that may eventually help settle a high-stakes debate of whether amyloid is a vital target for preventing or curing Alzheimer's.
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Abeona Therapeutics Receives FDA Orphan Designation for Gene Therapy

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for Abeona Therapeutics’s EB-101 gene therapy program for patients with dystrophic epidermolysis bullosa (DEB), including recessive dystrophic epidermolysis bullosa (RDEB), which are life-threatening genetic skin disorders characterised by skin blisters and erosions that cover the body. “Abeona is committed to advancing innovative gene therapies that address the unmet needs of patients suffering with dystrophic epidermolysis bullosa, a devastating rare skin disease. We are grateful that the FDA has recognised EB-101 as a rare disease product that may offer a significant therapeutic benefit for patients with dystrophic forms of epidermolysis bullosa, including RDEB.
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