‘Cure for Blindness’ Gene Therapy to be Tested on Humans Within 3 Years

Groundbreaking gene therapy that could cure blind people is to be tested on humans within three years. Scientists who are currently testing the treatment on mice have revealed they plan to be ready to test on human subjects soon. The development is to be applied to those suffering from an inherited retinal condition called Retinitis Pigmentosa (RP). Although the issue affects one in 4,000 people, there is currently no effective treatment. The research conducted by the University of Manchester, was licensed by a commercial concern, the US clinical-stage biotechnology company Acucela .
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FIT Biotech Oy: Finnish FIT Biotech and US Ichor to Collaborate on New Cost Effective Infectious Disease Treatment

FIT Biotech Oy (“FIT Biotech”) (Nasdaq: FITBIO) and Ichor Medical Systems (“Ichor”) of San Diego, California announced today that they have entered into a Research Collaboration Agreement. The purpose of the collaboration is to test FIT Biotech’s proprietary GTU® technology in combination with Ichor’s TriGrid® electroporation system in order to elicit the endogenous production of protective antibodies for passive immunoprophylaxis. By offering the prospect for rapid and sustained production of highly active antibodies from a recipient’s own cells, this approach could provide a new potential avenue to prevent the onset and progression of a wide range of infectious diseases. The ultimate goal of the research is the development of a highly adaptable and cost effective infectious disease product platform capable not only of addressing currently circulating infectious pathogens but also emerging diseases with the potential to cause worldwide pandemias.
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Inovio Pharma Announces Publication of Positive CHIKV-Targeting DNA Vaccine Data in Mice

Inovio Pharmaceuticals, Inc. (Nasdaq: INO) announced that its novel dMAb antibody and DNA vaccine targeting the chikungunya virus (CHIKV) provided 100% protection against a lethal virus challenge in mice. This breakthrough data was published in the latest issue of The Journal of Infectious Diseases in a paper, “Rapid and long-term immunity elicited by DNA encoded antibody prophylaxis and DNA vaccination against Chikungunya virus,” prepared by Inovio authors and their academic collaborators. While conventional vaccine and marketed monoclonal antibody technologies have shown limited ability to provide an effective solution to CHIKV to date, Inovio’s DNA vaccine and dMAb products show potential, separately and in combination, to offer immediate and long term protection to large populations from CHIKV infection. Over the years, CHIKV outbreaks have occurred in Africa, Asia, Europe, and throughout the Indian and Pacific Oceans, with local transmission in over 43 countries infecting millions of people. In late 2013, CHIKV was found for the first time in the Americas on islands in the Caribbean and spreading to other parts of the western hemisphere, including the United States. Along with a dramatic increase in cases and geographic spread of CHIKV infection and disease there has been a reported increase in morbidity and mortality, suggesting increased virulence. The concern for even greater potential global outbreaks underscores the need for targeted anti-viral interventions.
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Gene Therapy Approach Reported to Block Mutant Huntington’s Gene in Preclinical Studies

uniQure recently published results from preclinical studies of its gene therapy program, AMT-130, indicating that a one-time administration of AAV5-delivered therapy into the central nervous system can block the mutant HTT gene that causes Huntington’s disease (HD). These findings, in the article “Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington’s Disease,” were published in the Molecular Therapy-Nucleic Acids journal. The data also formed the basis of a presentation for the 11th Annual CHDI Huntington’s Disease Therapeutics Conference that took place on Feb. 24, 2016, in Palm Springs, California. Huntington’s disease is a neurodegenerative disorder that reduces muscle coordination and leads to behavioral and cognitive decline over time. Though the causes of HD are still not fully understood, a number of studies have shown that genetic mutations leading to the formation of an altered form of a protein called mutant Huntingtin (HTT) leads to HD development.
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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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