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Category Archives: Gene Therapy

Man’s best mend Gene therapy reverses muscular dystrophy symptoms in dogs – Digital Trends

Posted: August 3, 2017 at 4:40 pm

Why it matters to you Duchenne muscular dystrophy is a horrible disease which slowly attacks every muscle in the body. Gene therapy could help treat it -- whether you're a dog or a human. As difficult as it is when our beloved pet dogs get old, its a whole lot worse if their decline is the result of a horrible disease like Duchenne muscular dystrophy, a genetic disorder characterized by progressive muscle degeneration and weakness. Fortunately, a team of gene-editing researchers from France and the U.K. have been working to develop gene therapy as an answer and its one that could help humans, too. Their solution involves using gene therapy to restore muscle strength and stabilize clinical symptoms. This is achieved by way of a shortened version of the dystrophin gene, containing just 4,000 base pairs, which is combined with a viral vector and injected into patients. Duchenne muscular dystrophy is a debilitating muscle-wasting disease affecting young boys and male animals, which is caused by inheritance of a damaged gene, George Dickson, professor of molecular cell biology at University of London, told Digital Trends. There is currently no very effective cure. Our work has involved producing a healthy functioning copy … Continue reading

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Gene Therapy Retrovirus Vectors Explained

Posted: August 1, 2017 at 11:43 pm

A retrovirus is any virus belonging to the viral family Retroviridae. All The genetic material in retroviruses is in the form of RNA molecules, while the genetic material of their hosts is in the form of DNA. When a retrovirus infects a host cell, it will introduce its RNA together with some enzymes into the cell. This RNA molecule from the retrovirus must produce a DNA copy from its RNA molecule before it can be considered part of the genetic material of the host cell. Retrovirus genomes commonly contain these three open reading frames that encode for proteins that can be found in the mature virus. Group-specific antigen (gag) codes for core and structural proteins of the virus, polymerase (pol) codes for reverse transcriptase, protease and integrase, and envelope (env) codes for the retroviral coat proteins (see figure 1).Figure 1. Genome organisation of retroviruses. The process of producing a DNA copy from an RNA molecule is termed reverse transcription. It is carried out by one of the enzymes carried in the virus, called reverse transcriptase. After this DNA copy is produced and is free in the nucleus of the host cell, it must be incorporated into the genome of the … Continue reading

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Chiesi hands back gene therapy to uniQure | BioPharma Dive – BioPharma Dive

Posted: at 11:43 pm

Dive Brief: Even as gene therapies are being touted as the next wave of innovation that could offer cures for certain genetic conditions, it remains to be seen whether these products are actually commercially viable. There has yet to be a gene therapy approved in the U.S. (although Spark Therapeutics' application is pending), but two of the transformative drugs have been on the market in Europe. Yet neither of those commercially available gene therapies have found much success. GlaxoSmithKline plc. said just last week it is looking to move away from its rare disease portfolio, including the gene therapy Strimvelis. Meanwhile, uniQureannounced back in April it would not renew the marketing authorization application in Europe for its already-approved gene therapy Glybera. This latest move by Chiesifurther exemplifies the challenges gene therapy producers face. The announcement ends a deal which has been in place since 2013. Chiesisaid in a statement that the decision was "driven by recent changes in our strategic priorities." uniQuretried to put brave face on the news, but partnership exits are rarely good news for a biotech. "By regaining unencumbered, global rights to a late-stage program that has demonstrated significant clinical benefit for patients with hemophilia B, we … Continue reading

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Vectors in gene therapy – Wikipedia

Posted: at 11:43 pm

Gene therapy utilizes the delivery of DNA into cells, which can be accomplished by several methods, summarized below. The two major classes of methods are those that use recombinant viruses (sometimes called biological nanoparticles or viral vectors) and those that use naked DNA or DNA complexes (non-viral methods). All viruses bind to their hosts and introduce their genetic material into the host cell as part of their replication cycle. This genetic material contains basic 'instructions' of how to produce more copies of these viruses, hacking the body's normal production machinery to serve the needs of the virus. The host cell will carry out these instructions and produce additional copies of the virus, leading to more and more cells becoming infected. Some types of viruses insert their genome into the host's cytoplasm, but do not actually enter the cell. Others penetrate the cell membrane disguised as protein molecules and enter the cell. There are two main types of virus infection: lytic and lysogenic. Shortly after inserting its DNA, viruses of the lytic cycle quickly produce more viruses, burst from the cell and infect more cells. Lysogenic viruses integrate their DNA into the DNA of the host cell and may live in … Continue reading

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Chiesi dumps uniQure’s hemophilia B gene therapy | FierceBiotech – FierceBiotech

Posted: at 11:43 pm

Chiesi has cut its ties to uniQures hemophilia B gene therapy. The split gives uniQure full rights to AMT-060 but leaves it without a partner to cofund R&D as it closes in on the start of a pivotal trial. Italian drugmaker Chiesi picked up the rights to commercialize AMT-060 in certain markets in 2013 as part of a deal that also gave it a piece of Glybera, the gene therapy that made history by coming to market in Europe only to flop commercially. Chiesi backed out of the Glybera agreement earlier this year and has now completed its split from uniQure by terminating the hemophilia B pact. Amsterdam, the Netherlands-based uniQure framed the termination as it reacquiring the rights to AMT-060, rather than Chiesi dumping the program. But as the deal will see money transfer from Chiesi to uniQure and the former stated a shift in priorities prompted it to sever ties to AMT-060, it seems clear the Italian drugmaker wanted to exit the agreement. That leaves uniQure facing the prospect of taking AMT-060 into a pivotal trial without the financial support of a partner. Chiesi and uniQure have evenly shared R&D costs since 2013. The loss of the support … Continue reading

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Philly drug maker seeks approval in EU for gene therapy – Philly.com

Posted: at 11:43 pm

Philadelphia gene therapy company Spark Therapeutics has applied to the European Medicines Agency for approval to sell its treatment of rare inherited blindness in the European Union. The experimental therapy, Luxturna, or voretigene neparvovec, is under priority review with the U.S. Food and Drug Administration, with a possible approval date of Jan. 12, 2018. Spark was spun out of Childrens Hospital of Philadelphia, based on research led by Katherine A. High, Sparks cofounder, president, and chief scientific officer. If approved, it would be the first gene therapy for a genetic disease in the United States. With Luxturna now in regulatory review on both sides of the Atlantic, we are building out our medical and commercial infrastructure to bring the drug to patients, said John Furey, Sparks chief operating officer. For the first time, adults and children, who otherwise would progress to complete blindness, have hope for a potential treatment option that may restore their vision, he said. About 3,500 people in the United States and Europe live with the disease. The review period will begin in Europe once the agency validates the application, Spark said. Published: August 1, 2017 3:01 AM EDT | Updated: August 1, 2017 11:40 AM EDT … Continue reading

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Transient gene therapy may help youngsters with a premature … – Cosmos

Posted: at 11:43 pm

A rose will bloom, it then will fade. Alas, not so for those afflicted with Hutchison-Gilford Progeria Syndrome (HGPS). Their lives skip the blooming stage. Within a few months of birth their growth is stunted and they begin to show the hallmarks of ageing. Their skin loses its elasticity and their hair falls out. As teenagers they resemble tiny, gnomish octogenarians, with prominent eyes, pinched noses, receding jaws and veins protruding through thin transparent skin. The average age of death is 13 usually from a heart attack or stroke. Cardiologist John Cooke is trying to help those with the disease by at least slowing the ageing and stiffening of their blood vessels. His approach involves rejuvenating this tissue by delivering transient gene therapy using messenger RNA for a gene called telomerase. Since messenger RNA does not hang around, the technique avoids the pitfalls of gene therapy, like inadvertently triggering cancer. The results of his research, published this week in the Journal of the American College of Cardiology, show the successful rejuvenation of cells in the test tube from youngsters with HGPS. It brings tears to my eyes to see these kids but despite the fact theyre trapped in the body … Continue reading

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Hemophilia B Gene Therapy AMT-060 Moves to Large-scale Production, Says UniQure – Hemophilia News Today

Posted: at 11:43 pm

Pharmaceutical firm uniQure N.V. has developed an optimized large-scale process to manufacture its lead gene, therapydate, to treat hemophilia B. This new platform will allow the Dutch company to boost production of its AMT-060 gene therapy at its Lexington, Massachusetts, facility, in accordance with Good Manufacturing Practices guidelines. This should enable uniQure to meet the requirements of both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) by early fall, the companys CEO, Matt Kapusta, said in a press release. We have made significant progress over the past several months to now be in a position where we have developed a commercial-scale process and are evaluating our completed batches to assess comparability, Kapusta said. We look forward to finalizing this work in anticipation of meetings with regulators to further discuss plans to advance our hemophilia B program into a pivotal study next year. AMT-060 gene therapy is based on a viral vector to deliver a therapeutic form of human factor IX gene, or FIX. This potential therapy is currently being evaluated in a Phase 1/2 trial (NCT02396342) in patients with severe hemophilia B and advanced joint disease. The trials most recent long-term resultsshow that AMT-060 is … Continue reading

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GSK gives up on rare diseases as gene therapy gets two customers – Reuters

Posted: at 11:43 pm

LONDON (Reuters) - GlaxoSmithKline is swimming against the tide by getting out of treatments for rare diseases at a time when rivals like Sanofi and Shire see the field as a rich seam for profits. Successful medicines for rare conditions are potentially very lucrative, since prices frequently run into hundreds of thousands of dollars, but patient numbers can be extremely low. New GSK Chief Executive Emma Walmsley announced the strategic review and potential divestment of rare diseases on Wednesday as part of a wide-ranging drive to streamline pharmaceutical operations. It follows a less than impressive experience for GSK in the field, including the fact that its pioneering gene therapy Strimvelis only secured its first commercial patient in March, 10 months after it was approved for sale in Europe in May 2016. Since then a second patient has also been treated and two more are lined up to receive the therapy commercially, a spokesman said. Strimvelis, which GSK developed with Italian scientists, is designed for a tiny number of children with ADA Severe Combined Immune Deficiency (ADA-SCID). SCID is sometimes known as "bubble baby" disease, since those born with it have immune systems so weak they must live in germ-free environments. … Continue reading

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Gene Therapy Treats Muscular Dystrophy in Dogs, Provides Hope … – Wall Street Pit

Posted: at 11:43 pm

There is new hope for human patients with Duchenne muscular dystrophy. Results released in the journal Nature Communications describe a promising gene therapy performed on dogs. Twelve Golden Labrador dogs were subjected to a breakthrough gene therapy technology and, after two years, the dogs are healthy and appears to be illness-free. Researchers are optimistic about the implication of this study on humans. Duchenne muscular dystrophy (or DMD) is a hereditary condition characterized by muscle weaknesses and muscle degeneration. Among nine types of muscular dystrophy, DMD is the most severe and life-threatening. Dystrophin protein is vital for muscles to function properly and the absence of this protein makes muscles fragile and easily damaged. At early stages, DMD will affect muscles in the shoulder, upper arms, thighs and hips that are vital to movement and balance. Patients experience muscle weaknesses by age 4 and then start losing the ability to walk by age 12. Later on, DMD will weaken the heart and respiratory muscles. For DMD cases, the average life expectancy is 26 years, with only a few patients living beyond 40. Duchenne muscular dystrophy was named after French neurologist Guillaume Benjamin Amand Duchenne who described the illness in the 1860s. It … Continue reading

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