Gene Therapy Startup Immusoft Grows, Gets Access to Critical Technology with Acquisition of Discovery Genomics

Immusoft Corporation, a Seattle, Wash.-based gene therapy company, announced today it has purchased Minneapolis, Minn.-based Discovery Genomics, Inc. The acquisition brings to Immusoft renowned scientific expertise and key technology, the Sleeping Beauty Transposon System, which Discovery Genomics uses to deliver genes into cells without using a virus. “We welcome the Discovery Genomics team,” says Matthew Scholz, CEO and founder of Immusoft. “Our two companies share a culture of innovation and a desire to change the way many diseases can be treated. It is my hope and expectation that combining our expertise and technology will help us in this pursuit.” R. Scott McIvor, Ph.D., Discovery Genomics’ CEO, says he and his company are pleased with the acquisition.
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New Technique Enhances Gene Therapy

Using his knowledge of how genes are organized and repaired in human cells, Dr. Graham Dellaire, Dalhousie Medical School’s Cameron research scientist in cancer biology, has developed a technique that could make gene therapy more effective and safer to use. His work was recently published in Nucleic Acids Research and Nature. The technique uses CRISPR, a genome-editing platform that was named Science’s breakthrough discovery of the year for 2015. Short for ‘clustered regularly interspaced short palindromic repeats’, CRISPR can accurately target and edit DNA, offering the potential to cure genetic diseases and find new treatments for cancer.
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Spark Therapeutics Buys Dublin Gene-Therapy Company

Philadelphia drugmaker Spark Therapeutics Inc. has acquired for $15.1 million a private, Ireland-based gene-therapy company. Spark, spun out of Children's Hospital of Philadelphia, said Monday that it bought Genable Technologies Ltd. for $6 million in cash and 265,000 shares of Spark stock, valued at $9.1 million based on Friday's closing price. Spark said Genable's potential treatment, RhoNova, will target a common form of a rare inherited retinal disease, which impacts about 30,000 people worldwide. There currently is no approved pharmacologic treatment for the condition. The experimental therapy has shown promising data in animals, and received orphan-drug, or rare-disease, designation in the United States and Europe. Spark has collaborated with Genable on developing the therapy since 2014, the company said.
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BioMarin Receives Orphan Drug Designation From FDA for First AAV-Factor VIII Gene Therapy, BMN 270, for Patients With Hemophilia A

BioMarin Pharmaceutical Inc. announced today that BMN 270, an investigational gene therapy for the treatment of patients with hemophilia A, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). BioMarin is currently conducting a Phase 1/2 study to evaluate the safety and efficacy of BMN 270 gene therapy in up to 12 patients with severe hemophilia A and will provide a program update in April. BMN 270 is an AAV 5 factor VIII vector, designed to restore factor VIII plasma concentrations, essential for blood clotting in patients with hemophilia A. The FDA Orphan Drug program provides orphan designation to drugs and biologics that are intended for the treatment of rare diseases (those affecting fewer than 200,000 people in the United States).
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Stem Cell Gene Therapy Could be Key to Treating Duchenne Muscular Dystrophy

Scientists at the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research and Center for Duchenne Muscular Dystrophy at UCLA have developed a new approach that could eventually be used to treat Duchenne muscular dystrophy. The stem cell gene therapy could be applicable for 60 percent of people with Duchenne, which affects approximately 1 in 5,000 boys in the U.S. and is the most common fatal childhood genetic disease. The approach uses a technology called CRISPR/Cas9 to correct genetic mutations that cause the disease. The study, which was led by co-senior authors April Pyle and Melissa Spencer and first author Courtney Young, was published in the journal Cell Stem Cell. The researchers designed the approach to be useful in a clinical setting in the future.
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Pitt Experts Lead Gene Therapy Clinical Trial in Parkinson’s Disease Patients

Experts at the University of Pittsburgh School of Medicine are leading the second arm of a clinical trial using gene therapy to relieve the symptoms of tremor and mobility impairment in patients with Parkinson's disease. The technique shows promise in prolonging the effectiveness of levo-dopa, the mainstay treatment for the progressive neurodegenerative condition, by increasing production of a key enzyme essential to convert the drug into the neurotransmitter dopamine. An estimated 7 to 10 million people worldwide have Parkinson's disease, which is caused by degeneration of dopamine-producing neurons, said Mark Richardson, M.D., Ph.D., assistant professor of neurological surgery, Pitt School of Medicine, and director of Epilepsy and Movement Disorders Surgery at UPMC. Levo-dopa can replace the deficient dopamine for a while, but eventually the drug loses effectiveness, and subsequent increases in dosage may cause disabling side effects.
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Inovio Pharmaceutical’s DNA Vaccine for Zika Virus Induces Robust Immune Responses in Preclinical Study

Inovio Pharmaceuticals, Inc. announced today that preclinical testing of its synthetic vaccine for the Zika virus induced robust and durable immune responses, demonstrating the potential for a SynCon® vaccine to prevent and treat infections from this harmful pathogen. Health authorities have observed neurological and autoimmune complications potentially associated with Zika virus, including microcephaly in newborns and Guillain-Barre syndrome. Inovio is developing its Zika vaccine with GeneOne Life Sciences and academic collaborators.
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UNC Gene Therapy Spinout Bamboo Therapeutics Raises $49.5M Series A

North Carolina gene therapy startup Bamboo Therapeutics has raised a stunning $49.5 million, according to an SEC filing. The startup is focused on advancing the work of Dr. Richard Jude Samulski, director of the gene therapy center at the University of North Carolina, into the clinic to treat rare neurologic diseases like Duchenne’s muscular dystrophy. Bamboo says that Samulski was the first person to use adeno-associated viruses to replace defective genes with healthy ones; as a result, the company holds more than 20 patents in using AAV for therapeutic applications.
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Researchers Identify Way Radiation May Fight Cancer Cells Escaping Immune System

A team of Georgia State University researchers is fighting cancers using a combination of therapies and recently found ways that radiation could maximize responses to novel immune-based therapeutic approaches to fight cancer. "Radiation can increase the expression of genes, which allows the immune system to attack tumor cells that may have previously escaped elimination," said Charlie Garnett Benson, assistant professor in the Department of Biology at Georgia State and lead author on the study, whose findings have been published in the International Journal of Oncology.
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Gene Therapy for Rare Bleeding Disorder Achieves Proof-of-Concept

Hematology researchers have used a single injection of gene therapy to correct a rare bleeding disorder, factor VII deficiency, in dogs. This success in large animals holds considerable potential for a safe, effective and long-lasting new treatment in humans with the same bleeding disorder. "Our finding has great clinical relevance for patients with factor VII deficiency," said study leader Paris Margaritis, D. Phil., a hematology researcher at the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (CCMT) at The Children's Hospital of Philadelphia (CHOP). "These dogs have the type of mutation found in the majority of patients with this disorder, so this approach could lead to a sustained gene therapy in people." The study appeared online Dec. 23 in Blood.
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