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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Wet AMD: Small Trial Shows New Gene Therapy is Safe in Humans

One treatment for neovascular or wet age-related macular degeneration neutralizes the protein linked to the disease using repeated eye injections, an uncomfortable, onerous regimen that causes much anxiety for patients. Gene therapy - if proven to be safe and effective - offers an attractive alternative because one injection could provide lasting effects. Now, a small clinical trial shows that one experimental gene therapy for this significant cause of vision loss in older people appears to be safe and well tolerated. A paper on the trial, which was led by Johns Hopkins University School of Medicine in Baltimore, MD, and funded by Sanofi Genzyme of Framingham, MA, is published in The Lancet. Senior author Peter Campochiaro, a professor of ophthalmology at Johns Hopkins, says: "Even at the highest dose, the treatment was quite safe. We found there were almost no adverse reactions in our patients."
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Tiny Bubbles and a Bit of Gene Therapy Heal Major Bone Fractures in Pigs

It takes more than a cast and a little time to heal many broken bones. Whether it’s a soldier wounded in battle, a car accident victim, or an elderly person who has fallen, bone damage can be so extensive that the bones never heal properly, leaving people crippled or with other severe problems. Now, researchers have combined ultrasound, stem cells, and gene therapy to stimulate robust bone repair. So far the work has only been performed in animals. But it has already been so successful that it’s expected to move quickly toward human clinical trials. The new research “has huge clinical significance,” says David Kulber, who directs the Center for Plastic and Reconstructive Surgery at Cedars-Sinai Medical Center in Los Angeles, California, and who was not part of the study. “The technology of being able to stimulate bone growth is really remarkable.” It’s also one for which there is a glaring need. In the United States alone, some 100,000 people a year suffer from what is known as a nonunion fracture. In these cases, parts of a bone may be missing altogether or so badly splintered that the bone can’t be reassembled. In such cases, doctors typically graft other bone into the site. Ideally this bone comes from the same person—often taken from the pelvis, a painful procedure that compounds a person’s injuries and recovery time. When this isn’t possible, physicians will turn to cadavers for the extra bone. But this bone must be sterilized before it’s implanted, robbing it of proteins and other signaling molecules that encourage its regrowth once transplanted, and lessening the chances of a full recovery.
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Gene Therapy to Strengthen Nerve-Muscle Connection Prevents ALS Progression in Mice

A gene therapy aimed at strengthening the connection between motor neurons and muscle prevented nerve degeneration and increased the life span of mice with amyotrophic lateral sclerosis (ALS), a study showed. Since researchers believe that neurodegeneration in ALS starts at the junction between nerves and muscle, the findings suggest that preventing the connection from breaking down could be a good way to stop the disease from progressing. The study, “DOK7 gene therapy enhances motor activity and life span in ALS model mice,”was published in the journal EMBO Molecular Medicine. Interestingly, the University of Tokyo researchers did not develop the gene therapy with ALS in mind. They were trying to prevent a disease called DOK7 myasthenia that is caused by mutations of the DOK7 gene.
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Type 1 Diabetes Cured in Mice Using Gene Therapy

Researchers from the University of Texas Health Science Center in San Antonio have found a way to cure type 1 diabetes in mice. It is hoped that the novel technique - which boosts insulin secretion in the pancreas - will reach human clinical trials in the next 3 years. Study co-author Dr. Bruno Doiron, Ph.D., of the Division of Diabetes, and colleagues recently reported their findings in the journal Current Pharmaceutical Biotechnology. Type 1 diabetes is estimated to affect around 1.25 million children and adults in the United States. Onset of the condition is most common in childhood, but it can arise at any age.
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Expensive Gene Therapy Receives its First Patient in a Commercial Treatment

A patient with an extremely rare immune disease has been treated with commercial gene therapy for the first time, GlaxoSmithKline, the company behind the therapy, told MIT Technology Review on Tuesday. The treatment come almost a year after the therapy was approved for sale in Europe. Known as Strimvelis, the therapy treats a rare inherited immune deficiency by fixing a problem within the patient’s DNA. Gene therapy has been used extensively in clinical trials but has had a slow start commercially. This is only the second commercial use of gene therapy, the first of which was with a drug called Glybera in 2015.
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First Clear-cut Risk Genes for Tourette Disorder Revealed

Tourette disorder (also known as Tourette syndrome) afflicts as many as one person in a hundred worldwide with potentially disabling symptoms including involuntary motor and vocal tics. However, researchers have so far failed to determine the cause of the disorder, and treatments have only limited effectiveness, in part because the genetics underlying the disorder have remained largely a mystery. Now, as reported online May 3, 2017 in Neuron, a consortium of top researchers -- led by scientists at UC San Francisco, Rutgers University, Massachusetts General Hospital, the University of Florida, and Yale School of Medicine -- has made a significant advance, identifying the first "high-confidence" risk gene for Tourette disorder as well as three other probable risk genes. These findings are a step forward in understanding the biology of the disorder, the authors said, which will aid in the search for better treatments.
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GenSight Biologics’ Gene Therapy Candidate May Help Vision Loss in LHON Patients

An investigational gene therapy, GS010, is showing some promise in helping people who recently began to lose their vision due to Leber’s hereditary optic neuropathy (LHON), according to data from an ongoing clinical trial. A presentation of the data, “Intravitreal rAAV2/2-ND4 (GS010): A gene therapy for vision loss in Leber’s Hereditary Optic Neuropathy (LHON) caused by the G11778A ND4 mitochondrial mutation,” is set for April 25 at the 2017 annual meeting of the American Association of Neurology (AAN), taking place through April 28 in Boston. It will be given by José-Alain Sahel, MD, co-founder of GenSight Biologics, the developer of GS010. The Paris-based company’s lead product candidate, GS010, was designed to be the first gene replacement therapy targeting LHON, a rare inherited mitochondrial disease caused by ND4 mitochondrial gene mutation. Mainly affecting adolescents and young adults, LHON is characterized by irreversible vision loss due to a degeneration of retinal ganglion cells. In more severe cases, this disease can lead to blindness.
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