Clinical Trial Uses Gene Therapy to Target Mutations in Mitochondrial Genes

A multidisciplinary research team of scientists, clinicians and biostatisticians led by John Guy, M.D., professor of ophthalmology and director of the ocular gene therapy laboratory at the Bascom Palmer Eye Institute of the University of Miami Miller School of Medicine, has pioneered a gene therapy approach for Leber Hereditary Optic Neuropathy (LHON), an inherited genetic disorder that causes rapid, permanent, and bilateral loss of vision in people of all ages, but primarily men ages 20-40. Guy’s preclinical research has been funded since 2007 by National Institutes of Health and National Eye Institute (NEI) grants totaling $6.1 million. A similar amount will fund a new five-year clinical trial that started April 1.
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Erasing a Genetic Mutation: Researchers Reverse a Liver Disorder in Mice by Correcting a Mutated Gene

Using a new gene-editing system based on bacterial proteins, MIT researchers have cured mice of a rare liver disorder caused by a single genetic mutation. The findings, described in the March 30 issue of Nature Biotechnology, offer the first evidence that this gene-editing technique, known as CRISPR, can reverse disease symptoms in living animals. CRISPR, which offers an easy way to snip out mutated DNA and replace it with the correct sequence, holds potential for treating many genetic disorders, according to the research team.
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FDA Grants Orphan Designation to Kite Cancer Immunotherapy

Kite Pharma Inc. (Kite), a clinical-stage biotechnology company focused on developing engineered autologous T cell therapy (eACT) products for cancer, announced that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development granted orphan drug designation for the company's lead investigational therapy, an autologous engineered T cell product that targets CD19 expression on B cell malignancies, for the treatment of diffuse large B cell lymphoma (DLBCL).
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Novel Cancer Vaccine Promising Against Ovarian Cancer and Mesothelioma

A protein engineered to combine a molecule targeting a tumor-cell-surface antigen with another protein that stimulates several immune functions was found to prolong survival in animal models of both ovarian cancer and mesothelioma. This novel approach to cancer immunotherapy may provide a new and cost-effective weapon against some of the most deadly tumors. Cancer immunotherapy uses strategies designed to induce the immune system to attack cancer cells.
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Scientists Build First Synthetic Yeast Chromosome

An international team of scientists has built a modified yeast chromosome from scratch, the latest step in the quest to make the world's first synthetic yeast genome, an advance that would lead to new strains of the organism to help produce industrial chemicals, medicines and biofuels. Instead of just copying nature, the team did extensive tinkering with their chromosome, deleting unwanted genes here and there. It then successfully incorporated the designer chromosome into living yeast cells, endowing them with new capabilities not found in naturally occurring yeast. "It is the most extensively altered chromosome ever built," said Jef Boeke of New York University's Langone Medical Center, who led the effort. The findings were published on Thursday in an online edition of the journal Science.
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Researchers Testing Gene Therapy to Thwart Effects of Multiple Sclerosis

In patients with multiple sclerosis, the body turns on itself, launching an immune system attack that destroys the coating around nerve fibers in the central nervous system, leaving them exposed like bare wires. Similar to exposed electrical lines, the unprotected fibers touch and short out, leading to the neurodegenerative effects that are a hallmark of multiple sclerosis. But what if doctors could stop the immune response that destroys the protective coating before the disease becomes debilitating? University of Florida researchers have received a $40,000 grant from the National Multiple Sclerosis Society to test a gene therapy technique in mice that aims to help the body not treat itself like a foreign invader — a process referred to as immune tolerance — in the earliest stages of multiple sclerosis. If the researchers can re-establish this tolerance, they could thwart the immune system attack, all with a technique that could be used on a wide number of patients.
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Adelaide Researchers Claim Breakthrough in Tackling HIV and Hepatitis C with DNA Vaccine

South Australian researchers claim they have made a significant breakthrough in tackling HIV and hepatitis C, using a new type of DNA vaccine which protects against the viruses and could possibly provide a cure in five years. Adelaide University's Professor Eric Gowans says the vaccine has already had positive results in animals, with human trials to start next year. "DNA vaccines in general have enormous potential, but haven't worked very well in large animals and in patients," he said.
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Spark Therapeutics and Genable Technologies Announce Collaboration to Advance a Gene Therapy Treatment for a Rare Form of Retinitis Pigmentosa

Spark Therapeutics and Genable Technologies announced today that they have entered into a collaboration agreement for Genable's lead therapeutic to treat rhodopsin-linked autosomal dominant retinitis pigmentosa (RHO adRP), GT038. Under the terms of the collaboration, Genable will license certain adeno-associated virus (AAV) vector manufacturing patents from Spark. The parties have entered into a broad agreement in which Spark will be the exclusive manufacturer of the product and provide development advice and expertise to Genable to help in the ongoing development of GT038. Spark will receive milestone payments and royalties on future sales of GT038, as well as near-term revenue from the manufacture and supply of product.
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New DNA-editing Technology Spawns Novel Strategies for Gene Therapy

The University of California, Berkeley, and UC San Francisco are launching the Innovative Genomics Initiative (IGI) to lead a revolution in genetic engineering based on a new technology already generating novel strategies for gene therapy and the genetic study of disease. The Li Ka Shing Foundation has provided a $10 million gift to support the initiative, establishing the Li Ka Shing Center for Genomic Engineering and an affiliated faculty chair at UC Berkeley. The two universities also will provide $2 million in start-up funds.
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XstalBio Launches New Analytics Vaccine Service

Scotland-based privately held formulation and drug delivery firm XstalBio has launched a new vaccine analysis service to benefit biopharmaceutical and vaccine sectors. Basis of the new vaccine formulation and analysis service are advanced, precise and highly informative analytical techniques. The new service will be centred on XstalBio's proprietary solid state Circular Dichroism (ssCD) capability alongside highly specialised orthogonal analytical techniques and an increased range of vaccine analysis, formulation and stability testing services. This increased range is comprised of physico-chemical methodologies for vaccine characterization and release, formulation development and stability testing.
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