Gene therapy involving modified stem cells obtained from fatty tissue and bone marrow could represent a new option for the treatment of severe orthopaedic injuries to the extremities. The treatment could in future prevent threatened amputations or massive shortenings of bones.
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The first regulatory approval of a gene therapy treatment could spark broader patient access to the technology. Last month, Europe’s Committee for Medicinal Products for Human Use approved a gene therapy for a rare genetic disease, the first time a Western regulatory agency has okayed such a treatment, though gene therapies have been approved in China.
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The first regulatory approval of a gene therapy treatment could spark broader patient access to the technology. Last month, Europe’s Committee for Medicinal Products for Human Use approved a gene therapy for a rare genetic disease, the first time a Western regulatory agency has okayed such a treatment, though gene therapies have been approved in China.
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Regulatory approval of a gene therapy treatment in Europe could spark broader patient access to the technology. Last month, Europe’s Committee for Medicinal Products for Human Use approved a gene therapy for a rare genetic disease, the first time a Western regulatory agency has okayed such a treatment, though gene therapies have been approved in China.
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Gene Therapy for Eye Diseases - Dr. Donald J. D #39;Amico
Donald J. D #39;Amico, MD, the Ophthalmologist-in-Chief at New York Presbyterian/Weill Cornell Medial Center, talks about gene therapy as a potential treatment for eye diseases. You can learn more about Dr. D #39;Amico at: nyp.orgFrom:ORLivedotcomViews:1 0ratingsTime:01:55More inScience Technology
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Gene Therapy for Eye Diseases - Dr. Donald J. D'Amico - Video
SILVER SPRING, Md., Dec. 5, 2012 (GLOBE NEWSWIRE) -- Nuvilex, Inc. (NVLX), an international biotechnology provider of cell and gene therapy solutions, announced today its subsidiary Austrianova Singapore (ASPL) has published a new manuscript that describes the use of the Company's proprietary cell encapsulation technology for studies designed to create a cell based therapeutic treatment for diabetes.
The publication in the Wiener Medizinische Wochenschrift Skriptum, entitled "Cell encapsulation for the treatment of diabetes" was co-authored with Dr. Eva Brandtner, former ASPL Chief Scientific Officer who is presently at the Vorarlberg Institute for Vascular Investigation and Treatment (VIVIT) in Austria. The manuscript outlines the advantages of using cellulose sulfate for the encapsulation of cells for treatment of diabetes. The article accompanied an invited presentation on the same topic at the Diabetes Meeting in Salzburg, Austria also given by Dr. Brandtner.
ASPL and VIVIT have planned and are working closely together to advance and develop the diabetes treatment using encapsulated cells capable of expressing insulin after they detect the presence of glucose. Ultimately, the aim is to place insulin-secreting cells encapsulated in cellulose sulphate in patients with diabetes to replace the patient's own cells that no longer secrete insulin in response to elevated levels of glucose. As a result, the encapsulated cells thereby replace the normal pancreatic cells and enable the body to function normally, preventing exposure to the debilitating effects of high levels of glucose in the body.
The CEO of ASPL, Dr. Brian Salmons, said, "We are very happy to have been able to release our data through this publication from our testing of the encapsulated live cell treatment potential for diabetes. Being able to continue our work with VIVIT and Dr. Eva Brandtner, will greatly enhance our ability to develop and advance diabetes treatments."
The President and CEO of Nuvilex, Dr. Robert Ryan, stated "Our commitment to create valuable patient treatments through the proprietary cell encapsulation developed over so many years by ASPL, including treatments for diabetes, has become stronger over the past year. The important advances made have been through the funding provided by Nuvilex this past year and will prove valuable in the coming years. Clearly the ability to treat patients with this disease is a major driving force for our companies. From a market perspective, the World Health Organization has shown that the economic benefits will be substantial as the costs of diabetes are presently estimated to be over $350 Billion dollars spent annually per year worldwide."
About Nuvilex
Nuvilex, Inc. (NVLX) is an international biotechnology provider of live therapeutically valuable, encapsulated cells and services for research and medicine. Important advances are moving Nuvilex and Austrianova Singapore forward. New developments by our company and subsidiaries will be substantial as we have been working on many fronts to move us forward. We hope to bring some of these to light very soon. Our company's clinical offerings will include cancer, diabetes and other treatments using the company's cell and gene therapy expertise and live-cell encapsulation technology.
The Nuvilex, Inc. logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=13494
Safe Harbor Statement
This press release contains forward-looking statements described within the 1995 Private Securities Litigation Reform Act involving risks and uncertainties including product demand, market competition, and meeting current or future plans which may cause actual results, events, and performances, expressed or implied, to vary and/or differ from those contemplated or predicted. Investors should study and understand all risks before making an investment decision. Readers are recommended not to place undue reliance on forward-looking statements or information. Nuvilex is not obliged to publicly release revisions to any forward-looking statement, reflect events or circumstances afterward, or disclose unanticipated occurrences, except as required under applicable laws.
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AMSTERDAM, The Netherlands, December 6, 2012 /PRNewswire/ --
uniQure B.V., a leader in the field of human gene therapy, today announced the appointment of Hans Christian Rohde as Chief Commercial Officer.
Mr. Rohde joins uniQure from Basilea Pharmaceutica (SIX:BSLN) where from 2007 he was Chief Commercial Officer and member of the company's executive management committee with responsibility for global commercial operations, marketing, supply chain, medical affairs, pricing and market access.
"We are very pleased with Hans Christian's appointment," says Jrn Aldag, CEO of uniQure. "With the approval of Glybera we need to rapidly build and expand our commercial organization to enable the successful roll-out of Glybera and provide access to this crucial treatment to as many patients as possible. Hans Christian's experience and successful track-record in major biotechnology companies such as Basilea, Merck-Serono and Biogen Idec provide us with the necessary leadership qualities to successfully commercialize Glybera."
Hans Christian Rohde (M.Sc., MBA) has almost 25 years experience in commercial roles at leading biotechnology and pharmaceutical companies. Prior to Basilea Pharmaceutica, Mr. Rohde was Corporate Vice President, Head of Global Therapeutic Areas Reproductive Health and Endocrinology at Merck-Serono from 2003 until 2007. Before this he was responsible for international marketing and global market development at Biogen Idec. From 1992 until 2000, Mr Rohde held positions of increasing commercial responsibility at Novo Nordisk. Mr. Rohde started his career at Laboratoires Syntex.
About uniQure
uniQure is a world leader in the development ofhuman gene based therapies.uniQure's Glybera, a gene therapy for the treatment of lipoprotein lipase deficiency has been approved in the European Union, and is the first approved gene therapy in the Western world. uniQure's product pipeline of gene therapy products in development comprise hemophilia B, acute intermittent porphyria, Parkinson's disease and SanfilippoB. Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate probably the world's first stable and scalable AAV manufacturing platform.This proprietary platform can be applied to a large number of rare(orphan) diseases caused by one faulty gene. uniQure's largest shareholders are Forbion Capital Partners and Gilde Healthcare, two of the leading life sciences venture capital firms in the Netherlands. Further information can be found at http://www.uniqure.com.
About Glybera
uniQure has developed Glybera as a therapy for patients with the genetic disorder lipoprotein lipase deficiency, an orphan disease. The disease is caused by mutations in the LPL gene, resulting in highly decreased or absent activity of LPL enzyme in patients. This enzyme is needed in order to break down large fat-carrying particles that circulate in the blood after each meal. When such particles, called chylomicrons, accumulate in the blood, they may obstruct small blood vessels. Excess chylomicrons result in recurrent and severe acute inflammation of the pancreas, called pancreatitis, the most debilitating complication of LPLD. Glybera has orphan drug designation in the EU and US, and obtained marketing authorisation from the European Commission for commercialization in the 27 EU countries in November, 2012, making it the first gene therapy to win approval in the Western world.
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uniQure Appoints Hans Christian Rohde Chief Commercial Officer
Genes and Inheritance [12]: Gene Therapy (A Level Biology)
Thanks for watching. Please subscribe and take a look at our full list of tutorials for other revision videos: http://www.freeeschool.blogspot.co.ukFrom:Free E SchoolViews:0 0ratingsTime:07:40More inEducation
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Genes and Inheritance [12]: Gene Therapy (A Level Biology) - Video
PORT ST. LUCIE, Fla.--(BUSINESS WIRE)--
The Vaccine and Gene Therapy Institute of Florida (VGTI Florida) is pleased to announce the appointment of Catherine E. Vorwald to Director, Business Development.
We dedicate ourselves every single day to finding novel treatments for chronic diseases like cancer and HIV/AIDS, said Mel Rothberg, Chief Operating Officer, VGTI Florida. Were pleased to have Catherine onboard. By leading our business development efforts, she will facilitate the commercial translation of therapeutic discoveries so that patients around the world will ultimately be better served and suffer less, he continued.
Catherine will work closely with VGTI Florida leadership to develop and implement a comprehensive strategic approach including growing the intellectual property portfolio, commercializing therapeutic discoveries, and developing an innovation center, based on the institutes technology, for new entities and emerging companies wishing to co-locate and utilize the core facilities and expertise available at the Tradition Center for Innovation.
VGTI Florida has a wealth of expertise and therapeutic discovery potential and I am dedicated to translating these capabilities to industry so that they become a standard part of medical care, said Catherine.
As an accomplished technology commercialization executive, Catherine brings extensive domestic and international biotechnology and life science management experience to VGTI Florida. She specializes in fostering relationships between the academic and corporate communities. Catherine spent nearly six years as Director of Business Development working on marketing and leasing activities for Wexford Science + Technology, LLC, a real estate developer of university technology research parks. Her career encompasses technology commercialization from a variety of aspects, namely, marketing and business development at companies ranging from Fortune 500 to start-ups, academic research at the National Institutes of Health (NIH) and the University of California, San Francisco and technology transfer at Stanford and Johns Hopkins Universities.
Catherine has served as a board member of two notable technology incubators, the Emerging Technology Center of Baltimore and the Economic Development Corporation of South Florida. She is a former board member of The Licensing Executives Society USA-Canada, Inc., and the LES Foundation. She also founded the Licensing Executives Society Maryland Local Chapter. Catherine holds a Master of Business Administration in Finance from The Johns Hopkins University as well as a Master of Science Degree from Georgetown University and a Bachelor of Arts in Biological Sciences from the University of California.
VGTI Florida
VGTI Florida is a leading immunological research institute that is on an urgent mission to transform scientific discoveries into novel treatments and cures for devastating chronic illnesses such as cancer and HIV/AIDS. VGTI Florida is an independent non-profit 501 (c) 3 organization located in the Tradition Center for Innovation in Port St. Lucie, Florida. For more information, please visit our website at http://www.VGTIfl.org.
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AMSTERDAM, The Netherlands, December 5, 2012 /PRNewswire/ --
uniQure B.V., a leader in the field of human gene therapy, today announced a non-exclusive cross-licensing agreement with Benitec Biopharma Ltd. (BLT.AX) giving uniQure access to Benitec's proprietary DNA-directed RNA interference (ddRNAi) technology in Huntington's disease. In return, uniQure granted Benitec non-exclusive access to the Company's AAV5 delivery technology for the development of a ddRNAi therapy for Hepatitis B.
"The cross-licensing agreement with Benitec fully capitalizes on the strength of our advanced AAV platform and our proven ability to deliver therapeutic genes to target cells with high accuracy and efficacy," says Jrn Aldag, CEO of uniQure. "The agreement with Benitec opens up promising new avenues to develop therapies for high unmet medical needs such as Huntington's disease. While our current programs focus on delivering fully functioning therapeutic genes to remedy faulty or malfunctioning genes, Benitec's ddRNAi technology will allow us to do the opposite - to 'silence' the gene responsible for producing the mutant protein that lies at the basis of Huntington's disease, and to develop a therapy for this devastating disease."
Dr Peter French, CEO of Benitec Biopharma, commented, "Benitec Biopharma is very pleased to have executed this licensing agreement with uniQure, the first company to achieve market approval for a gene therapy product, Glybera, in the West. uniQure have demonstrated their unique ability to take gene therapy-based programs from pre-clinical stages to commercialization, and we are confident that they will be able to achieve a similar outcome in this program. Importantly this agreement also provides Benitec access to uniQure's AAV delivery technology enabling further development of our ddRNAi treatment for Hepatitis B."
About Huntington's disease
Huntington's disease (HD) is a rare, chronic, incurable, progressive and disabling neurological condition, which continues to challenge the medical community. About 30,000 Americans (or 1 in 10,000 people) have Huntington's disease and at least 150,000 individuals have a 50% chance of inheriting the disease. The symptoms of HD become most evident in adulthood, typically 30 to 55 years, characterized by sudden, abnormal, and uncontrolled jerky movements called chorea. Currently, therapeutics for HD are limited only to symptomatic treatments and there are no treatment options with proven safety and efficacy to slow down disease progression or enhance survival rate.
About uniQure
uniQure is a world leader in the development ofhuman gene based therapies.uniQure's Glybera, a gene therapy for the treatment of lipoprotein lipase deficiency has been approved in the European Union, and is the first approved gene therapy in the Western world. uniQure's product pipeline of gene therapy products in development comprise hemophilia B, acute intermittent porphyria, Parkinson's disease and SanfilippoB. Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate probably the world's first stable and scalable AAV manufacturing platform.This proprietary platform can be applied to a large number of rare(orphan) diseases caused by one faulty gene, and allows uniQure to pursue its strategy of focusing on this sector of the industry. uniQure's largest shareholders are Forbion Capital Partners and Gilde Healthcare, two of the leading life sciences venture capital firms in the Netherlands. Further information can be found at http://www.uniqure.com.
About Benitec Biopharma
Benitec Biopharma Ltd is developing novel treatments for chronic and life-threatening conditions based on targeted gene-silencing activity using a transformational technology: DNA-directed RNA interference (ddRNAi) - sometimes called expressed RNAi. The technology's potential to address unmet medical needs and to cure disease results from its demonstrated ability to permanently silence genes which cause the condition. Importantly, this technology's target gene and related gene pathways will rarely have presented as a therapeutic avenue for research for the traditional small molecule agents, currently accounting for the majority of today's pharmaceutical products.
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uniQure Licenses RNA Interference Technology to Advance Huntington's Disease Program
Scott C. Baraban, PhD discusses the current trend in epilepsy research
Dr. Scott Baraban, PhD, a Professor and William K. Bowes Endowed Chair in Neuroscience Research at the University of California, San Francisco, discusses the current trends of epilepsy research and the future direction of epilepsy care, particularly noting the role of gene therapy in management. This video was taken on Friday, November 30th at the American Epilepsy Society #39;s 2012 Annual Meeting in San Diego, CA.From:AJMCtvViews:5 0ratingsTime:00:59More inEducation
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Scott C. Baraban, PhD discusses the current trend in epilepsy research - Video
Paulsboro Residents Are Frustrated Over Evacuations Paulsboro Residents Are Frustrated Over Evacuations
Updated: Sunday, December 2 2012 12:59 AM EST2012-12-02 05:59:06 GMT
More than one hundred Paulsboro residents are spending the next few nights in hotels after being evacuated from their homes. It follows a Friday night spike in chemical testing levels after a Conrail
Updated: Saturday, December 1 2012 9:10 PM EST2012-12-02 02:10:01 GMT
Philadelphia Police are investigating the death of a five-year-old boy who died early Saturday morning. According to Police, the parents of the five-year-old called for help late Friday night saying their
Philadelphia Police are investigating the death of a five-year-old boy who died early Saturday morning.
Updated: Saturday, December 1 2012 8:20 PM EST2012-12-02 01:20:52 GMT
Newark Police are investigating the robbery of a 28-year-old University of Delaware student that occurred around 5:00 a.m. Saturday. The female victim was approached by two unknown males while walking
Newark Police are investigating the robbery of a 28-year-old University of Delaware student that occurred around 5:00 a.m. Saturday.
Updated: Saturday, December 1 2012 6:37 PM EST2012-12-01 23:37:42 GMT
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2 Kids Suffering From Rare Disease Admitted Into Gene Therapy Study
Episode 1. Uncovering Genetic Code
#12298;Unveiling the Secrets of Biotechnology #65306;Volume #8741; #12299; Part 1 #65306;Secrets Left by Our Ancestors Part 2 #65306;The Antidote to Rare Diseases -- Gene Therapy Part 3 #65306;Opening DNA #39;s Pandora #39;s BoxFrom:ccleestagViews:0 0ratingsTime:23:34More inScience Technology
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Metal Gear Solid: The Twin Snakes Game Movie
http://www.gamematics.net During a training mission on Shadow Moses, a nuclear weapons training facility on a remote island off the coast of Alaska, the Special Forces unit FOXHOUND rebelled against the United States government, under the command of Liquid Snake. Their target was the advanced weapon system Metal Gear REX, a gigantic robotic weapons platform able to independently launch a nuclear warhead at any target on the face of the planet. Their demand was the body of the greatest soldier who ever lived, Big Boss, which, through gene therapy, they could use to create an army of ultimate soldiers. With the safety of the entire world at stake, Colonel Roy Campbell, the former commander of FOXHOUND, summoned Solid Snake out of retirement for one last solo covert operation. Gameplay MrPlentl Developer: Silicon Knights Konami Publisher: KonamiFrom:lapman17Views:6 0ratingsTime:03:43:40More inGaming
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SCMOM 2012_Sistemic
Sistemic #39;s world leading expertise is on applying microRNA profiling in context with the biology, to areas where there is currently an unmet need for sophisticated tools delivering instructive and robust knowledge of the cell system. From their SistemQC trade; platform they have derived an extensive suite of tools for the cell therapy, gene therapy and bioprocessing community. SistemQC trade;, molecularly characterises cells including stem cells as well as aids in the optimization and monitoring (QC) of the manufacture process. More specifically the initial focus of SistemQC trade; by clients has been on generation of microRNA based ID markers, purity potency assessment and manufacture monitoring optimization. http://www.sistemic.co.uk Presenter: Jim Reid, Chairman and CEO, SistemicFrom:AllianceRegenMedViews:3 1ratingsTime:11:56More inScience Technology
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FATAL FAMILIAL INSOMNIA BIO PSA
The question for my presentation that I was looking for an answer to was "What treatments have been discovered to fight or cure Fatal Familial Insomnia?" In my research I found that there has been some treatments tested on mice models that have slowed down the deadly disease, but researchers have not found one that will completely cure or sufficiently fight Fatal Familial Insomnia. In my discussion I talk about when the disease was discovered and how rare it is. I also explain what a prion disease is and codon is being effected. I have a chart in my presentation that compares the different sleep spindles, normal sleep vs. FFI sleep, so that the viewer can understand how extremely different a person with FFI sleep stages are to some one with normal sleep functions. The majority of my presentation was focused on the treatments section in which I list the tested treatments, some examples of cases that I found, and whether they made a difference or not. I found out that sleeping medications are the least successful in helping patients with FFI and can actually worsen the condition. Gene therapy as we discussed in the course already is the replacement of a mutated gene with a repaired one. The findings from the gene therapy case and agomelatine case showed that they are a helpful treatment, but not the cure to FFI. Even though these treatments did not work sufficiently to cure FFI, I think they are leading the researchers in the right direction to actually find the cure.From:DMarie23ifyViews:0 0ratingsTime:05:23More inPeople Blogs
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Step into Authenticity with Susan Gould Fogerite
Website: shrp.umdnj.edu Susan Gould-Fogerite, PhD, is the Director of Research for ICAM. Dr. Gould-Fogerite has a long-standing interest in integrating Eastern and Western Philosophy and Medicine. She practices and teaches mind-body interventions including yoga, meditation and breathwork in the Himalayan tradition and does Reiki energy healing. Dr. Gould-Fogerite earned her BS in Medical Technology from Albany Medical College and SUNY at Albany, and her PhD in Microbiology and Immunonoloy from Albany Medical College. Her research in the fields of Vaccinology, Mucosal Immunology, Drug Delivery and Gene Therapy has resulted in numerous presentations, articles and book chapters. Dr. Gould-Fogerite is one of the Scientific Founders of BioDelivery Sciences International, Inc., the first spin-off company from UMDNJ to become publicly traded. She is looking forward to developing and supporting basic and clinical research, in addition to teaching aspects of complementary and alternative medicine. Get the whole series free at http://www.stepintoauthenticity.com Step into Authenticity without Alienating the World and Being Kicked off the Planet is an interview series by Alex Iglecia where mind body, conscious living and transformational experts and teachers give you timeless wisdom and priceless action steps for you live more true to yourself today. Get the whole series free at httpFrom:Alex IgleciaViews:3 0ratingsTime:03:08More inHowto Style
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Is Porphyria a Good Candidate for Gene Therapy?
From:Sarah CraneViews:0 0ratingsTime:04:56More inPeople Blogs
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GENETICS 3: GENE THERAPY
GENE THERAPYFrom:drjahn41Views:0 0ratingsTime:01:00More inScience Technology
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Dr. Tsilfidis on XIAP Gene Therapy
XIAP gene therapy is unique madi-in-Canada therapy for retinal disease. Developed by Dr. Catherine Tsilfidis and her colleagues at the University of Ottawa and the Ottawa Hospital Research Institute, the treatment aims to slow cell death in the retina and preserve vision. Promising findings show that the therapy can potentially halt cell death in many conditions including retinitis pigmentosa and retinal detachment, preventing further vision loss. Learn more about XIAP Gene Therapy in this short video interview with Dr. Tsilfidis (1:30 minutes). Footage provided by: Accessible Media Inc. (www.ami.ca)From:FFBCanadaViews:89 1ratingsTime:01:31More inEducation
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