Archive for the ‘Gene Therapy Research’ Category

AMSTERDAM, June 21, 2012 /PRNewswire/ --

uniQure, a leader in the field of human gene therapy, announced today the signing of a collaborative agreement with two leading neurology experts to develop further a gene therapy incorporating uniQure's GDNF (glial cell derived neurotrophic factor) gene for the treatment of Parkinson's disease.

Professor Krystof Bankiewicz at the University of California, San Francisco (UCSF), a world expert in GDNF gene therapy, and Professor Howard Federoff of Georgetown University, a preeminent physician-neuroscientist, have developed a product approved to start clinical trials in the U.S. using uniQure's GDNF gene incorporated into an adeno-associated virus-2 (AAV-2) delivery vector. The GDNF gene contains the information to produce a protein necessary for the development and survival of nerve cells. The positive effect of GDNF on nerve cells has already been demonstrated in early research by uniQure in collaboration with the University of Lund, Sweden.

UCSF entered into a collaboration with Dr. Russell Lonser, neurosurgeon and Chief of the Neurosurgical Branch of the NINDS, a division of the National Institutes of Health, to commence a Phase I study of the gene therapy in patients with Parkinson's disease. Patient enrollment is expected to begin mid-2012. Collaborating on the study will be Drs. Krystof Bankiewicz of UCSF, Howard Federoff of Georgetown University and NINDS co-investigator neurologists Drs. Mark Hallett and Walter Koroshetz.

"This agreement provides uniQure with access to the data from a Parkinson's disease GDNF clinical study conducted by two of the world's leading medical researchers in the field. If successful, we intend to manufacture the vector construct ourselves and with a partner progress the product into advanced clinical studies," said Jrn Aldag, CEO of uniQure. "GDNF has been shown to be involved in several other CNS disorders so if we reach the proof of concept stage in Parkinson's, we can potentially expand product development quickly and efficiently into clinical trials for other indications, such as Huntington's and Multiple System Atrophy (MSA)."

"The development of AAV2-GDNF, sponsored by both NIH and by Parkinson's foundations, has taken us 10 years to complete. We are very pleased that a path for clinical development of AAV2-GDNF as a possible treatment for PD is now in place," said Dr. Krystof Bankiewicz, UCSF Principal Investigator.

Under the terms of uniQure's agreement with UCSF, uniQure holds the exclusive commercial rights to all UCSF preclinical data and to IND enabling Phase I clinical data provided to UCSF by NINDS. In the event that the Phase 1 study shows proof of concept, uniQure will use its proprietary manufacturing system for future production of the AAV construct and take responsibility for future development of the gene therapy product. uniQure holds the exclusive license to the GDNF gene from Amgen.

About uniQure

uniQure is a world leader in the development of human gene based therapies. uniQure has a product pipeline of gene therapy products in development for 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. Further information can be found at http://www.uniqure.com.

Certain statements in this press release are "forward-looking statements" including those that refer to management's plans and expectations for future operations, prospects and financial condition. Words such as "strategy," "expects," "plans," "anticipates," "believes," "will," "continues," "estimates," "intends," "projects," "goals," "targets" and other words of similar meaning are intended to identify such forward-looking statements. Such statements are based on the current expectations of the management of uniQure only. Undue reliance should not be placed on these statements because, by their nature, they are subject to known and unknown risks and can be affected by factors that are beyond the control of uniQure. Actual results could differ materially from current expectations due to a number of factors and uncertainties affecting uniQure's business. uniQure expressly disclaims any intent or obligation to update any forward-looking statements herein except as required by law.

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uniQure Collaborates with UCSF on GDNF Gene Therapy in Parkinson's Disease

ScienceDaily (June 20, 2012) With a single drug treatment, researchers at the Ludwig Institute for Cancer Research at the University of California, San Diego School of Medicine can silence the mutated gene responsible for Huntington's disease, slowing and partially reversing progression of the fatal neurodegenerative disorder in animal models.

The findings are published in the June 21, 2012 print issue of the journal Neuron.

Researchers suggest the drug therapy, tested in mouse and non-human primate models, could produce sustained motor and neurological benefits in human adults with moderate and severe forms of the disorder. Currently, there is no effective treatment.

Huntington's disease afflicts approximately 30,000 Americans, whose symptoms include uncontrolled movements and progressive cognitive and psychiatric problems. The disease is caused by the mutation of a single gene, which results in the production and accumulation of toxic proteins throughout the brain.

Don W. Cleveland, PhD, professor and chair of the UC San Diego Department of Cellular and Molecular Medicine and head of the Laboratory of Cell Biology at the Ludwig Institute for Cancer Research, and colleagues infused mouse and primate models of Huntington's disease with one-time injections of an identified DNA drug based on antisense oligonucleotides (ASOs). These ASOs selectively bind to and destroy the mutant gene's molecular instructions for making the toxic huntingtin protein.

The singular treatment produced rapid results. Treated animals began moving better within one month and achieved normal motor function within two. More remarkably, the benefits persisted, lasting nine months, well after the drug had disappeared and production of the toxic proteins had resumed.

"For diseases like Huntington's, where a mutant protein product is tolerated for decades prior to disease onset, these findings open up the provocative possibility that transient treatment can lead to a prolonged benefit to patients," said Cleveland. "This finding raises the prospect of a 'huntingtin holiday,' which may allow for clearance of disease-causing species that might take weeks or months to re-form. If so, then a single application of a drug to reduce expression of a target gene could 'reset the disease clock,' providing a benefit long after huntingtin suppression has ended."

Beyond improving motor and cognitive function, researchers said the ASO treatment also blocked brain atrophy and increased lifespan in mouse models with a severe form of the disease. The therapy was equally effective whether one or both huntingtin genes were mutated, a positive indicator for human therapy.

Cleveland noted that the approach was particularly promising because antisense therapies have already been proven safe in clinical trials and are the focus of much drug development. Moreover, the findings may have broader implications, he said, for other "age-dependent neurodegenerative diseases that develop from exposure to a mutant protein product" and perhaps for nervous system cancers, such as glioblastomas.

Co-authors are first author Holly B. Kordasiewicz, Melissa M. McAlonis, Kimberly A. Pytel and Jonathan W. Artates, Ludwig Institute for Cancer Research and UC San Diego Department of Cellular and Molecular Medicine; Lisa M. Stanek, Seng H. Cheng and Lamya S. Shihabuddin, Genzyme Corporation; Edward V. Wancewicz, Curt Mazur, Gene Hung and C. Frank Bennett, Isis Pharmaceuticals; and Andreas Weiss, Novartis Institutes for BioMedical Research.

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Proposed drug may reverse Huntington's disease symptoms: Single treatment gives long-term improvement in animals

Single treatment produces long-term improvement in animal models

Newswise With a single drug treatment, researchers at the Ludwig Institute for Cancer Research at the University of California, San Diego School of Medicine can silence the mutated gene responsible for Huntingtons disease, slowing and partially reversing progression of the fatal neurodegenerative disorder in animal models.

The findings are published in the June 21, 2012 print issue of the journal Neuron.

Researchers suggest the drug therapy, tested in mouse and non-human primate models, could produce sustained motor and neurological benefits in human adults with moderate and severe forms of the disorder. Currently, there is no effective treatment.

Huntingtons disease afflicts approximately 30,000 Americans, whose symptoms include uncontrolled movements and progressive cognitive and psychiatric problems. The disease is caused by the mutation of a single gene, which results in the production and accumulation of toxic proteins throughout the brain.

Don W. Cleveland, PhD, professor and chair of the UC San Diego Department of Cellular and Molecular Medicine and head of the Laboratory of Cell Biology at the Ludwig Institute for Cancer Research, and colleagues infused mouse and primate models of Huntingtons disease with one-time injections of an identified DNA drug based on antisense oligonucleotides (ASOs). These ASOs selectively bind to and destroy the mutant genes molecular instructions for making the toxic huntingtin protein.

The singular treatment produced rapid results. Treated animals began moving better within one month and achieved normal motor function within two. More remarkably, the benefits persisted, lasting nine months, well after the drug had disappeared and production of the toxic proteins had resumed.

For diseases like Huntington's, where a mutant protein product is tolerated for decades prior to disease onset, these findings open up the provocative possibility that transient treatment can lead to a prolonged benefit to patients, said Cleveland. This finding raises the prospect of a huntingtin holiday, which may allow for clearance of disease-causing species that might take weeks or months to re-form. If so, then a single application of a drug to reduce expression of a target gene could reset the disease clock, providing a benefit long after huntingtin suppression has ended.

Beyond improving motor and cognitive function, researchers said the ASO treatment also blocked brain atrophy and increased lifespan in mouse models with a severe form of the disease. The therapy was equally effective whether one or both huntingtin genes were mutated, a positive indicator for human therapy.

Cleveland noted that the approach was particularly promising because antisense therapies have already been proven safe in clinical trials and are the focus of much drug development. Moreover, the findings may have broader implications, he said, for other age-dependent neurodegenerative diseases that develop from exposure to a mutant protein product and perhaps for nervous system cancers, such as glioblastomas.

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Proposed Drug May Reverse Huntington's Disease Symptoms

There's an interesting exchange on genetic engineering at the Food Politics blog, http://tinyurl.com/, featuring a review by industry critic Marion Nestle of an anti-GE paper, GMO Myths and Truths. I've skimmed the paper, which you can find at http://tinyurl.com/, and I confess to lacking the expertise to evaluate the claims. It would take more time than I have at the moment to dig into the claims, although I hope to do so in the future.

Nestle says the authors of the paper, who find nothing to like in genetic engineering, "have put a great deal of time and effort into reviewing the evidence for the claims. This is the best-researched and most comprehensive review I've seen of the criticisms of GM foods." She asks whether the pro camp can "produce something equally well researched, comprehensive, and compelling?" and concludes, "I doubt it but I'd like to see them try." She says there's enough evidence in the paper to justify labeling, at the very least.

It is, of course, the position you'd expect her to take, and several of the comments following her post challenge both her and the paper. One claims there are indeed well-researched, comprehensive and compelling pro papers. Others say the paper she cites cherry-picks evidence and relies on papers that have been debunked. An example cited in one of these critical comments asserts that it relies on a study of Bt found in human blood that used a test that couldn't detect blood at the levels the study's authors said they found.

My suspicion is most won't read these papers and will continue to think what they already think about the issue. An even worse fear is that reading the papers on both sides wouldn't convert anyone on either side. Still, I may give it a try at some point.

Follow me on Twitter: http://www.Twitter.comurbanize

Copyright 2012 DTN/The Progressive Farmer, A Telvent Brand. All rights reserved.

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Paper, Blog Heats Up GMO Discussion

18-06-2012 13:12 "Natural forces within us are the true healers of disease."-Hippocrates Research @ NOTE: (Immunotherapy Research) Dr. Cassian Yee, Drs. Zihai Li and Bei Liu,, Zheng Cui, Ph.D, William Coley, Victor Ambros, Gary Ruvkun, and David Baulcombee UConn Health Center researchers have uncovered a way to potentially use human stem cells as a kind of vaccine against colon cancer. This discovery, led by experts in immunology, Drs. Zihai Li and Bei Liu, builds upon a century old theory that immunizing with embryonic materials may generate an anti-tumor response by "fooling" the immune system into believing that cancer cells are present. The finding potentially opens up a new paradigm for cancer vaccine research. "By? using a probe to shine a light on the tumour, the antibodies at? the right place in the body can be brought to life. Any antibodies in the rest of the body will remain dormant, meaning side effects can be minimised. The activated antibodies then cause immune cells in the blood called T-cells to attack the cancer." "Just a few minutes of the light therapy directed at the? region of the tumour would activate the T-cells causing? her? body's own immune cells to attack the tumour." November 13, 2007 presentation by Edgar Engleman for the Stanford School of Medicine Medcast lecture series. Edgar Engleman, MD, medical director of the Stanford ...

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Electro-Medicine : Biological Closed Electric Circuitry - Video

19-06-2012 13:51 Aida Bytyçi is a certified genetic counselor at the McKusick-Nathans Institute of Genetic Medicine at Johns Hopkins University in the US. Her time is divided between providing genetic counseling to patients and working on several research projects, such as one attempting to sequence the whole human genome to identify genes responsible for inherited syndromes and health conditions. As a genetic counselor, Aida provides support and advice to patients and their families on the inheritance and consequences of the genetic disorder. In this ideal position between research and patient care, Aida orients herself with the polar star of creative innovation brought by science and art. She believes that these two disciplines stimulate the mind and require a developed imagination to bring change in people's lives. At TEDxPrishtina Aida tells the greatest story ever written in four letters: A, T, C, and G. These four nucleotides that make the DNA code are the core of the greatest book ever written, the Human Genome. AboutTEDx, x=independently organized event In the spirit of ideas worth spreading, TEDx is a program of local, self-organized events that bring people together to share a TED-like experience. At a TEDx event, TEDTalks video and live speakers combine to spark deep discussion and connection in a small group. These local, self-organized events are branded TEDx, where x=independently organized TED event. The TED Conference provides general guidance for the TEDx program, but ...

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TEDxPrishtina - Aida Bytyçi - Video

19-06-2012 15:05 The SickKids Centre for Genetic Medicine is bringing together the brightest minds in patient care, education, policy and research with the goal of one day making individualized treatment a standard of care for all children. The Centre for Genetic Medicine has the potential to have a significant impact on the health of children, as 90 per cent of chronic diseases have a genetic component and known genetic diseases account for over half of hospital admissions.

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The SickKids Centre for Genetic Medicine - Video

Newswise LOS ANGELES (June 19, 2012) Cedars-Sinais Regenerative Medicine Institute has pioneered research on how motor-neuron cell-death occurs in patients with spinal muscular atrophy, offering an important clue in identifying potential medicines to treat this leading genetic cause of death in infants and toddlers.

The study, published in the June 19 online issue of PLoS ONE, extends the institutes work to employ pluripotent stem cells to find a pharmaceutical treatment for spinal muscular atrophy or SMA, a genetic neuromuscular disease characterized by muscle atrophy and weakness.

With this new understanding of how motor neurons die in spinal muscular atrophy patients, we are an important step closer to identifying drugs that may reverse or prevent that process, said Clive Svendsen, PhD, director of the Cedars-Sinai Regenerative Medicine Institute.

Svendsen and his team have investigated this disease for some time now. In 2009, Nature published a study by Svendsen and his colleagues detailing how skin cells taken from a patient with the disorder were used to generate neurons of the same genetic makeup and characteristics of those affected in the disorder; this created a disease-in-a-dish that could serve as a model for discovering new drugs.

As the disease is unique to humans, previous methods to employ this approach had been unreliable in predicting how it occurs in humans. In the research published in PLoS ONE, to the team reproduced this model with skin cells from multiple patients, taking them back in time to a pluripotent stem cell state (iPS cells), and then driving them forward to study the diseased patient-specific motor neurons.

Children born with this disorder have a genetic mutation that doesnt allow their motor neurons to manufacture a critical protein necessary for them to survive. The study found these cells die through apoptosis the same form of cell death that occurs when the body eliminates old, unnecessary as well as unhealthy cells. As motor neuron cell death progresses, children with the disease experience increasing paralysis and eventually death. There is no effective treatment now for this disease. An estimated one in 35 to one in 60 people are carriers and about in 100,000 newborns have the condition.

Now we are taking these motor neurons (from multiple children with the disease and in their pluripotent state) and screening compounds that can rescue these cells and create the protein necessary for them to survive, said Dhruv Sareen, director of Cedars-Sinais Induced Pluripotent Stem Cell Core Facility and a primary author on the study. This study is an important stepping stone to guide us toward the right kinds of compounds that we hope will be effective in the model and then be reproduced in clinical trials.

The study was funded in part by a $1.9 million Tools and Technology grant from the California Institute for Regenerative Medicine aimed at developing new tools and technologies to aid pharmaceutical discoveries for this disease.

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Researchers, with Stem Cells, Advance Understanding of Spinal Muscular Atrophy

Public release date: 19-Jun-2012 [ | E-mail | Share ]

Contact: Nicole White nicole.white@cshs.org 310-423-5215 Cedars-Sinai Medical Center

LOS ANGELES (June 19, 2012) Cedars-Sinai's Regenerative Medicine Institute has pioneered research on how motor-neuron cell-death occurs in patients with spinal muscular atrophy, offering an important clue in identifying potential medicines to treat this leading genetic cause of death in infants and toddlers.

The study, published in the June 19 online issue of PLoS ONE, extends the institute's work to employ pluripotent stem cells to find a pharmaceutical treatment for spinal muscular atrophy or SMA, a genetic neuromuscular disease characterized by muscle atrophy and weakness.

"With this new understanding of how motor neurons die in spinal muscular atrophy patients, we are an important step closer to identifying drugs that may reverse or prevent that process," said Clive Svendsen, PhD, director of the Cedars-Sinai Regenerative Medicine Institute.

Svendsen and his team have investigated this disease for some time now. In 2009, Nature published a study by Svendsen and his colleagues detailing how skin cells taken from a patient with the disorder were used to generate neurons of the same genetic makeup and characteristics of those affected in the disorder; this created a "disease-in-a-dish" that could serve as a model for discovering new drugs.

As the disease is unique to humans, previous methods to employ this approach had been unreliable in predicting how it occurs in humans. In the research published in PLoS ONE, to the team reproduced this model with skin cells from multiple patients, taking them back in time to a pluripotent stem cell state (iPS cells), and then driving them forward to study the diseased patient-specific motor neurons.

Children born with this disorder have a genetic mutation that doesn't allow their motor neurons to manufacture a critical protein necessary for them to survive. The study found these cells die through apoptosis the same form of cell death that occurs when the body eliminates old, unnecessary as well as unhealthy cells. As motor neuron cell death progresses, children with the disease experience increasing paralysis and eventually death. There is no effective treatment now for this disease. An estimated one in 35 to one in 60 people are carriers and about in 100,000 newborns have the condition.

"Now we are taking these motor neurons (from multiple children with the disease and in their pluripotent state) and screening compounds that can rescue these cells and create the protein necessary for them to survive," said Dhruv Sareen, director of Cedars-Sinai's Induced Pluripotent Stem Cell Core Facility and a primary author on the study. "This study is an important stepping stone to guide us toward the right kinds of compounds that we hope will be effective in the model and then be reproduced in clinical trials."

###

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Cedars-Sinai researchers, with stem cells, advance understanding of spinal muscular atrophy

SAN MARINO, Calif.--(BUSINESS WIRE)--

Viral Genetics (VRAL), through its majority owned subsidiary VG Energy, is now verifying the chemical activity in industrial scale batches of LipidMax in response to demand for the product from research institutes and from industry. LipidMax, the first product marketed by VG Energy, is a lipid enhancement compound for use in the production of oils from algae and other plants or plant-like organisms and is based on the Companys exclusively licensed Metabolic Disruption Technology (MDT). VG Energy is launching LipidMax in response to unsolicited demand for MDT compounds from algae oil producers after it announced testing results during the preceding year.

Separately, VG Energy also reports that LipidMax has shown in an initial internal study to potentially increase the amount of extracted palm oil when applied to raw palm fruit. The enormous market for palm oil growers is heavily concentrated in Southeast Asia and this has led the Company to commence the formation of an operating subsidiary to be called VG Energy (Asia) Sdn Bhd. Its objectives are to explore the possible applications of LipidMax in the palm oil industry and act as the distribution arm for VG Energy, Inc. The global palm oil industry delivered more than 160 million metric tons of product last year, at $895/ton representing more than $140 billion annually.

The product release testing is predominantly focused on verifying that the chemical activity of industrial-scale batches of LipidMax from the manufacturers identified by VG Energy is comparable to the smaller, laboratory-sized batches used in testing. Once product release testing is complete, VG Energy will order larger quantities from its final manufacturer(s) and begin to deliver samples to prospective buyers for their own field-testing which it anticipates will lead to commercial orders thereafter.

We thought it prudent to engage a manufacturing facility to be able to secure quick delivery and access to LipidMax. We believe this marks one of the most exciting milestones in Viral Genetics and VG Energys history representing the nearest term potential to generate first time revenues, stated Haig Keledjian, President of VG Energy and Viral Genetics.

VG Energys impetus to begin contract manufacture of the LipidMax product is the direct result of demand from industry and research institutes after review of the results of both internal and external studies showing LipidMaxs ability to substantially increase the amount of lipids from algae, seeds, yeast and potential future markets in other edible oils such as palm oil.

Palm oil is an edible oil derived from the fruit of the oil palm plant and is found in foods such as ice cream, cookies, peanut butter, pies, power bars, baked goods and snacks. It is one of the leading cooking oil products in the world, especially in Southeast Asia. It can also be found in personal care products such as soaps, shampoos, cosmetics, skin care. Palm oil, like other vegetable oils, can be used to create biodiesel as either a simple, processed palm oil mixed with petroleum-based diesel, or processed through transesterification to create a palm oil methyl ester blend, which meets the international EN 14214 specification.

About VG Energy

VG Energy, Inc. is an alternative energy and agricultural biotech company that is a majority-owned subsidiary of Viral Genetics, Inc., a biotechnology company researching new treatments and methods of detection for diseases including cancer, HIV/AIDS and others. Using its Metabolic Disruption Technology (MDT), Viral Genetics cancer research led to discoveries with major consequences in a wide variety of other industries, including biofuel and vegetable oils. VG Energy holds the exclusive worldwide license to the MDT patent rights for use in the increase of production of various plant-derived oils from algae and seeds. These pivotal discoveries promise to allow the biofuel industry to overcome its major obstacle in the area of production efficiency: namely, an increase in production yields leading to feasible economic returns on investment, allowing renewable biodiesel to be competitive with fossil fuels. For more information, please visit http://www.vgenergy.net.

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VG Energy to Launch First Commercial Product, LipidMax™ for Palm and Algae Oil Production

Public release date: 20-Jun-2012 [ | E-mail | Share ]

Contact: Ruthann Richter richter1@stanford.edu 650-725-8047 Stanford University Medical Center

STANFORD, Calif. Genetics play a significant role in determining which patients will suffer the most from the disturbing side effects of opiates, commonly prescribed painkillers for severe to moderate pain, according to a new Stanford University School of Medicine study, which pinpoints nausea, slowed breathing and potential for addiction as heritable traits.

"One of the most hated side effects of these opiates, nausea, is strongly inherited," said Martin Angst, MD, professor of anesthesia and one of two principal investigators for the new study, which explores individual variations in the response to opiate use. The study will be published online June 20 in Anesthesiology. Genetics also play a likely role in determining which patients will suffer from itchiness and sedation associated with the use of these powerful medications, which include morphine, methadone and oxycodone.

"The study is a significant step forward in efforts to understand the basis of individual variability in response to opioids and to eventually personalize opioid treatment plans for patients," said Angst, director of the Stanford Human Pain Research Laboratory. "Our findings strongly encourage the use of downstream molecular genetics to identify patients who are more likely or less likely to benefit from these drugs to help make decisions on how aggressive you want to be with treatment, how carefully you monitor patients and whether certain patients are suitable candidates for prolonged treatment."

Treatment with opiates, also known as narcotics, is tricky because of this variability in drug response. Certain patients may require 10 times the amount of these painkillers to get the same level of pain relief as others. In fact, in some patients the occurrence of side effects may prevent the use of opioids for effectively alleviating pain. Side effects such as nausea or sedation can be debilitating to some, while nonexistent for others. Similarly, some patients can take medications for months with little addiction potential, while others are at risk within weeks.

Millions of U.S. patients are prescribed opiates for pain each year. A better understanding of the potential risk of side effects motivated the researchers to explore individual variation in pairs of identical and fraternal twins, Angst said. The study was prompted by past genetic studies in animals that have shown a strong genetic component in the response to opiates.

"We rely heavily on narcotics as the cornerstone medication for the relief of pain," said Angst. "Yet we don't know the answers to fundamental questions, such as why some people 'like' narcotics more than others drug liking and disliking could be key in determining addiction potential."

Researchers recruited 121 twin pairs for the randomized, double-blinded and placebo-controlled study. Pain sensitivity and analgesic response were measured by applying a heat probe and by immersing a hand in ice-cold water, both before and during an infusion of the opiate alfentanil, a short-acting painkiller prescribed by anesthesiologists. The team also compared individual variations in levels of sedation, mental acuity, respiratory depression, nausea, itch, and drug-liking/disliking a surrogate measure of addiction potential between identical twins, non-identical twins and non-related subjects. This provided an estimate of the extent to which variations in responses to opiates are inherited. For example, the finding that identical twins are more similar in their responses to opiates than non-identical twins suggested inheritance plays a significant role.

Heritability was found to account for 30 percent of the variability for respiratory depression, 59 percent of the variability for nausea and 36 percent for drug disliking. Additionally, up to 38 percent for itchiness, 32 percent for dizziness and 26 percent for drug-liking could be due to heritable factors. An earlier study published by the same researchers in the March issue of Pain reported that genetics accounted for 60 percent of the variability in the effectiveness of opiates in relieving pain.

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Stanford study shows opiates' side effects rooted in patients' genetics

Positively-charged ligands on the nanoparticles attach to the DNA, but the hydrophobic ligands of the nanoparticles became tangled with each other. As this tangling pulled the nanoparticles into clusters, the nanoparticles pulled the DNA apart. Credit: Yaroslava Yingling, North Carolina State University

New research from North Carolina State University finds that gold nanoparticles with a slight positive charge work collectively to unravel DNA's double helix. This finding has ramifications for gene therapy research and the emerging field of DNA-based electronics.

"We began this work with the goal of improving methods of packaging genetic material for use in gene therapy," says Dr. Anatoli Melechko, an associate professor of materials science and engineering at NC State and co-author of a paper describing the research. Gene therapy is an approach for addressing certain medical conditions by modifying the DNA in relevant cells.

The research team introduced gold nanoparticles, approximately 1.5 nanometers in diameter, into a solution containing double-stranded DNA. The nanoparticles were coated with organic molecules called ligands. Some of the ligands held a positive charge, while others were hydrophobic meaning they were repelled by water.

Enlarge

Because gold nanoparticles have a slight positive charge from the ligands, and DNA is always negatively charged, the DNA and nanoparticles are pulled together into complex packages. Credit: Yaroslava Yingling, North Carolina State University

"However, we found that the DNA was actually being unzipped by the gold nanoparticles," Melechko says. The positively-charged ligands on the nanoparticles attached to the DNA as predicted, but the hydrophobic ligands of the nanoparticles became tangled with each other. As this tangling pulled the nanoparticles into clusters, the nanoparticles pulled the DNA apart. Video of the process is below:

This video is not supported by your browser at this time.

The finding is also relevant to research on DNA-based electronics, which hopes to use DNA as a template for creating nanoelectronic circuits. Because some work in that field involves placing metal nanoparticles on DNA, this finding indicates that researchers will have to pay close attention to the characteristics of those nanoparticles or risk undermining the structural integrity of the DNA.

Provided by North Carolina State University

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Researchers find gold nanoparticles capable of 'unzipping' DNA

ScienceDaily (June 20, 2012) New research from North Carolina State University finds that gold nanoparticles with a slight positive charge work collectively to unravel DNA's double helix. This finding has ramifications for gene therapy research and the emerging field of DNA-based electronics.

"We began this work with the goal of improving methods of packaging genetic material for use in gene therapy," says Dr. Anatoli Melechko, an associate professor of materials science and engineering at NC State and co-author of a paper describing the research. Gene therapy is an approach for addressing certain medical conditions by modifying the DNA in relevant cells.

The research team introduced gold nanoparticles, approximately 1.5 nanometers in diameter, into a solution containing double-stranded DNA. The nanoparticles were coated with organic molecules called ligands. Some of the ligands held a positive charge, while others were hydrophobic -- meaning they were repelled by water.

Because the gold nanoparticles had a slight positive charge from the ligands, and DNA is always negatively charged, the DNA and nanoparticles were pulled together into complex packages.

"However, we found that the DNA was actually being unzipped by the gold nanoparticles," Melechko says. The positively-charged ligands on the nanoparticles attached to the DNA as predicted, but the hydrophobic ligands of the nanoparticles became tangled with each other. As this tangling pulled the nanoparticles into clusters, the nanoparticles pulled the DNA apart.

"We think gold nanoparticles still hold promise for gene therapy," says Dr. Yaroslava Yingling, an assistant professor of materials science and engineering at NC State and co-author of the paper. "But it's clear that we need to tailor the ligands, charge and chemistry of these materials to ensure the DNA's structural integrity is not compromised."

The finding is also relevant to research on DNA-based electronics, which hopes to use DNA as a template for creating nanoelectronic circuits. Because some work in that field involves placing metal nanoparticles on DNA, this finding indicates that researchers will have to pay close attention to the characteristics of those nanoparticles -- or risk undermining the structural integrity of the DNA.

The research was supported by the National Science Foundation.

Video: http://www.youtube.com/watch?v=9M-58niEOpU&feature=colike

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Gold nanoparticles capable of 'Unzipping' DNA

BYRON, MN--It's a dream for many in the medical field, to use a person's own stem cells to help them heal. And it's a reality already happening in our area.

But it's not humans who are being treated. In this case, dogs are the ones being treated.

Animal Stem Cell Regenerative Therapy has been performed a few thousand times now across the U.S. Doctors harvest stem cells and re-enter them where the animal is having problems.

Both Marley and Vinnie have bad ligaments in their legs, and like many dogs suffering from arthritis, they are subject to monthly doses of expensive drugs.

That is until today.

Dr. Garren Kelly, D.V.M. at Meadow View Veterinary Clinic just outside Rochester says, "If you'd of asked me 5 years ago if I would be doing anything like this, I would have said no. But then as soon as I saw it i'm like 'Yeah that's for me'. I kind of like staying on the cutting edge of technology and surgeries".

The two are undergoing a first of its kind surgery in minnesota, using regenerative stem cells.

Blood is taken from the dogs, as well as fat tissue.

Then stem cells are separated out from the fat, activated with an led light, and injected back into the affected area. All in the same day.

MediVet America trainer Jordan Smith says, "It's a better quality of life, we're not promising to give them 10 years or 5 years but we are promising that the years that they do have remaining are a lot more enjoyable".

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Animal Stem Cell Therapy

HAIFA, Israel, June 19, 2012 (GLOBE NEWSWIRE) -- Pluristem Therapeutics, Inc. (PSTI) (TASE:PLTR) announced today at the 2012 Bio International Convention the results of a pre clinical study it conducted measuring the effectiveness of its Placental eXpanded (PLX) cells when administered intramuscularly(IM). Cell therapies are traditionally delivered through intravenous (IV) injections for systemic effect. However, Pluristem's latest findings show that its PLX cells can be effective when injected by needle, into the muscle. Avoiding the use of an IV is simple and more cost-effective. This opens far larger markets for treatments in a wide range of potential outpatient settings and local clinics.

"The ability for IM injections of PLX cells has significant market implications that potentially broaden the indications and frequency with which our cell therapy can be used. We look forward to conducting additional testing of this very promising approach," said Zami Aberman, Chairman and CEO of Pluristem.

The study found that Intramuscularly administered PLX cells are safe, effective, easy to inject and provided systemic therapeutic benefits in a wide range of hematological disorders, as well as primary and secondary bone marrow failure, such as in radiation sickness and possibly for some complications from chemotherapy and radiotherapy.

The results of the study demonstrated a significant survival and recovery rate of bone marrow and peripheral blood counts in animals pre-irradiated by high lethal doses. These findings indicate that the IM route of administration of PLX cells stimulate the hematopoietic stem cells (HSCs) of the bone marrow to produce red and white blood cells as well as platelets crucial for the treatment of hematological disorders. The study was conducted in cooperation with the Sharett Institute of Oncology at Hadassah Hospital in Jerusalem.

"Pluristem is extremely pleased at how convincingly this study's data demonstrates that our PLX cells have the ability to stimulate the HSCs involved in rescuing bone marrow. With PLX cells, we may be able to reverse the traditional mindset that if you want to get a systemic effect, you need to inject the cells intravenously," said Liat Flaishon, MD. PhD. BD Director and the Head of the Radiation project at Pluristem.

"We had announced on May 9, 2012 the successful treatment of a pediatric patient whose bone marrow graft was rescued using our PLX cells. This data demonstrates the basis for the successful treatment. In the treatment conducted by Professor Reuven Or from the Bone Marrow Transplantation Unit at Hadassah, PLX cells were given to this patient intramuscularly as well," added Dr. Flaishon.

Prof. Raphael Gorodetsky, Head of the Laboratory of Biotechnology and Radiobiology in the Cancer Research Laboratories of Sharett Institute of Oncology at Hadassah Hospital, has been conducting the animal studies of Pluristem's PLX cells in the past several months. In these studies PLX cells and control medium were administered intramuscularly to C3H mice previously irradiated by a total body dose of 770cGy. The company previously reported initial results from these studies with respect to Acute Radiation Syndrome.

The key results of the Study include:

- After an initial sharp fall, a significant increase in the total number of bone marrow cells extracted from the major bones at 23 days was recorded: from~16million cells/mouse to ~32 million cells/mouse in the PLX treated (p<0.001). Non-irradiated animals had an average of 40 million cells.

- at 23 days a significant increase in the total number of red blood cells was recorded from 3.5 in the surviving controls to 6 million cells/microliter, in comparing the PLX (p<0.001). Non-irradiated animals had an average of 7 million cells/microliter.

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Pluristem Therapeutics' Cell Therapy Broadens Addressable Markets - Demonstrates Systemic Effectiveness of ...

Public release date: 19-Jun-2012 [ | E-mail | Share ]

Contact: Jim Sliwa jsliwa@asmusa.org 202-942-9297 American Society for Microbiology

Human Insulin Suppresses Mosquito Immune System: Increasing Cases of Type II Diabetes Could Abet Malaria's Spread

Human insulin suppresses the mosquito immune system, according to a paper in the June Infection and Immunity. And while mosquitoes and malaria might seem to go together like baseball and hotdogs, mosquitoes' immunological resistance to the malaria parasite actually slows its spread among H. sapiens.

"A fair portion actually fight off the infection," says first author Nazzy Pakpour of the University of California, Davis.

But now the rate of type 2 diabetes is climbing in Africa as in most of the rest of the world, to the point where by 2030, one in five adults there are predicted to be so-afflicted. More diabetes means more hyperinsulinemiamore human insulin to inhibit mosquitoes' immune response to Plasmodium falciparum, thus aiding and abetting transmission of this dread disease.

As horrific as the medical consequences of all this might be, the science is intriguing. "It's crazy to think something in our blood could change how mosquitoes respond to parasites," says Pakpour.

In earlier work, Pakpour and collaborators showed that ingested human insulin activates the insulin/IGF-1 signaling pathway in Anopheles stephensi mosquitoes, making them more vulnerable to invasion by P. falciparum. The new study showed that insulin signaling reduced expression of certain mosquito immunity genes that are under the same regulatory control, and that human insulin suppressed mosquito immunity by activating the so-called PI3K signaling pathway, and that artificially inhibiting that pathway could reverse the immunosuppressive effects of human insulin.

(N. Pakpour, V. Corby-Harris, G.P. Green, H.M. Smithers, K.W. Cheung, M.A.Riehle, and S. Luckhart, 2012. Ingested human insulin inhibits the mosquito NF-KAPPAB-dependent immune response to Plasmodium falciparum. Infect. Immun. 80:2141-2149.)

Download the journal article at http://bit.ly/asm061912a

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Public release date: 19-Jun-2012 [ | E-mail | Share ]

Contact: Jeremy Moore jeremy.moore@aacr.org 215-446-7109 American Association for Cancer Research

PHILADELPHIA Researchers have identified a potential new pathway in prostate cancer cells by which cancer-driving gene products can be generated, according to a study published in Cancer Discovery, a journal of the American Association for Cancer Research.

"Our work shows that cancers have many more tricks than we thought to generate potential cancer-driving genes or gene products," said Hui Li, Ph.D., assistant professor of pathology at the University of Virginia in Charlottesville, and a recipient of an Innovative Research Grant from Stand Up To Cancer (SU2C). The AACR is the scientific partner of SU2C.

Gene fusion is a common characteristic of human cancers. In many cases, the protein products of these gene fusions, which are generated via an RNA intermediate, have a key role in the genesis of the cancer. A well-characterized example of this is the protein that drives chronic myeloid leukemia, BCR-ABL, which is generated via RNA intermediates from a fusion gene formed by chromosomal translocation an event involving exchange of genomic DNA between two distinct chromosomes.

"For many years, chromosomal translocation was considered the sole way in which single RNAs consisting of copies of parts of two genes, so-called fusion RNAs, could be generated," said Li. "We have shown that fusion RNAs can be generated without changes to DNA by a new mechanism that we are calling cis-SAGe [cis-splicing of adjacent genes]." Recently, a fusion RNA formed from parts of the SLC45A3 and ELK4 genes was identified in prostate cancer cells in the absence of any DNA alterations. Li and his colleagues confirmed in two prostate cancer cells lines that the SLC45A3-ELK4 fusion RNA could be detected even though there was no evidence of genomic DNA rearrangement.

Detailed molecular analysis of the prostate cancer cell lines indicated that the SLC45A3-ELK4 fusion RNA was generated by cis-SAGe. SLC45A3 and ELK4 are neighboring genes, and cis-SAGe occurred when an RNA that crossed the boundary between the two genes was formed.

The protein CCCTC-binding factor normally acts to insulate SLC45A3 and ELK4 from each other. Li and his colleagues found that levels of this protein at the gene boundary inversely correlated with the amount of SLC45A3-ELK4 fusion RNA generated, providing molecular insight into how the quantity of this fusion RNA could be regulated.

A functional role for the SLC45A3-ELK4 fusion RNA in prostate cancer was suggested by two observations. First, it promoted the growth of the two prostate cancer cell lines in culture. Second, its levels in human prostate samples correlated with prostate cancer disease progression normal prostate tissue expressed the lowest levels and prostate cancer specimens from men with metastatic disease expressed the highest levels.

"These data are not sufficient to say that the SLC45A3-ELK4 fusion RNA has a causal role in prostate cancer," said Li. "But they are highly suggestive, and I am very excited that this high-risk project, which I would not have been able to pursue without the grant from Stand Up To Cancer, has uncovered what seems to be a new way in which cancer can be driven."

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Abnormal gene product associated with prostate cancer generated by unusual mechanism

CARLSBAD, Calif., June 19, 2012 /PRNewswire/ -- Life Technologies Corporation (LIFE) today announced it has partnered with The Hospital for Sick Children (SickKids) to advance pediatric genomic research on the Ion Proton Sequencer. Under the agreement, the semiconductor-based platform will be the primary instrument on which multiple clinical research samples will be mapped daily on four sequencers in the hospital's newly launched Centre for Genetic Medicine.

SickKids and Life Technologies will collaborate on developing sequencing workflows and protocols for the Ion Proton System that are tailored for studies of interest to researchers in the Centre. The first collaborative project will focus on sequencing clinical research samples to better understand the genetics behind autism, with a long-term goal to sequence up to 10,000 genomes per year to study various diseases in children.

"The perfect storm of unparalleled advances in genome sequencing technology and information science, and a captivated hospital striving for new ways to move forward in medical treatment, bring us to this important day," says the new Centre's Co-Director, Dr. Stephen Scherer, who also leads The Centre for Applied Genomics at SickKids and the University of Toronto's McLaughlin Centre. "We are very excited to work with Life Technologies to enhance our sequencing capabilities, such that 'genomic surveillance' may soon become the first line of investigation in all clinical research studies ongoing at our institution."

"Since the first published draft sequence of the human genome, our knowledge in genetics has exponentially increased," says Dr. Ronald Cohn, Co-Director of the SickKids Centre for Genetic Medicine. "With the help of this new technology, we will be able to further deepen our understanding of the genetic basis of human disease and translate this directly into daily clinical practice. We have finally reached a point, where individualized medicine is not just a theoretical concept, but will become an integral part of clinical care and management."

The Ion Proton Sequencer is designed to sequence an entire human genome in a day for $1,000. Unlike traditional next generation systems, it relies on semiconductor chips to map human exomes and genomes, making it much faster and less expensive to analyze DNA at unprecedented throughput levels and generate accurate sequencing data.

The Ion Proton Systemis based on the same proven technology as its predecessor, the Ion Personal Genome Machine (PGM), which is designed for sequencing small genomes or sets of genes. Combined with Life Technologies' AmpliSeq targeted sequencing technology, researchers can sequence panels of genes associated with disease on the PGM or exomes and genomes on the Ion Proton Sequencer in just a few hours.

"SickKids has a rich history of being at the forefront of pediatric medicine and we are pleased that its leaders have chosen the Ion Proton Sequencer as the Centre's primary technology to push the boundaries of genomics," said Mark Stevenson, President and Chief Operating Officer of Life Technologies. "Ion semiconductor technology's speed, simplicity and scalability are democratizing sequencing, and it will now be applied in disease research to benefit children."

The above mentioned technology is for research use only and not intended for human diagnostic or therapeutic use.

About Life Technologies Life Technologies Corporation (LIFE) is a global biotechnology company with customers in more than 160 countries using its innovative solutions to solve some of today's most difficult scientific challenges. Quality and innovation are accessible to every lab with its reliable and easy-to-use solutions spanning the biological spectrum with more than 50,000 products for translational research, molecular medicine and diagnostics, stem cell-based therapies, forensics, food safety and animal health. Its systems, reagents and consumables represent some of the most cited brands in scientific research including: Ion Torrent, Applied Biosystems, Invitrogen, GIBCO, Ambion, Molecular Probes, Novex, and TaqMan. Life Technologies employs approximately 10,400 people and upholds its ongoing commitment to innovation with more than 4,000 patents and exclusive licenses. LIFE had sales of $3.7 billion in 2011. Visit us at our website: http://www.lifetechnologies.com.

Life Technologies' Safe Harbor StatementThis press release includes forward-looking statements about our anticipated results that involve risks and uncertainties. Some of the information contained in this press release, including, but not limited to, statements as to industry trends and Life Technologies' plans, objectives, expectations and strategy for its business, contains forward-looking statements that are subject to risks and uncertainties that could cause actual results or events to differ materially from those expressed or implied by such forward-looking statements. Any statements that are not statements of historical fact are forward-looking statements. When used, the words "believe," "plan," "intend," "anticipate," "target," "estimate," "expect" and the like, and/or future tense or conditional constructions ("will," "may," "could," "should," etc.), or similar expressions, identify certain of these forward-looking statements. Important factors which could cause actual results to differ materially from those in the forward-looking statements are detailed in filings made byLife Technologies with the Securities and Exchange Commission.Life Technologies undertakes no obligation to update or revise any such forward-looking statements to reflect subsequent events or circumstances.

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The Hospital for Sick Children in Toronto Adopts Life Technologies' Ion Proton™ Sequencer to Launch New Centre for ...

Psychiatrists often try two or more medications in a patient suffering with major depression before settling on the one that seems to work best for that individual. Sometimes, after several are tried and abandoned, two (or even three) are used in combination.

Medication selection is part of the art of psychiatry, but, now, testing is available that promises to make it more of a science. A company called AssureRx Health now offers what it calls GeneSightRxpharmacogenomic laboratory testing that helps identify which antidepressants are a good match for a persons genetic makeup, and which are not so good a match.

Sometimes, the testing reveals why three or four antidepressants havent worked for a patient, while pointing in the direction of one that might.

This is extremely good news, because psychiatrists have several different kinds of antidepressants to choose fromsome which increase the activity of the brain chemical messenger serotonin, some which increase the activity of the brain chemical messenger norepinephrine and some which increase both. And they do so by varying mechanisms, requiring the activity of different enzymes.

The technology behind GeneSightRx actually determines which genetic variantsin terms of the enzymes that are activated by antidepressantsa person possesses.

Different antidepressants affect the enzymes very differently. Hence, the testing can literally predict with some accuracy which antidepressants are likely to work in a particular person, and which are likely to cause the fewest side effects.

Recent studies have revealed that antidepressants dont work much better than placebo medications (sugar pills) for many patients. But those studies werent conducted by first selecting patients who are more likely (as determined by GeneSightRx) to respond to the particular medicine being studied.

Its very possible that patients given medications suggested by such testing would do far better than those given placebosbecause they arent being lumped together and given one medicine, regardless of their individual genetic makeup.

Moreover, since many patients discontinue their antidepressants due to side effects like sexual dysfunction and sleeplessness, choosing a medication that is metabolically and genetically less likely to cause these and other side effects makes good sense.

GeneSightRx also predicts which ADHD medications, antipsychotics and pain medications patients are likely to respond to and from which they are likely to experience fewer side effects.

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Genetic testing to choose the right antidepressant

AMES, Iowa, June 19, 2012 (GLOBE NEWSWIRE) -- NewLink Genetics Corporation (NLNK) today announced that in the second quarter of 2012 it passed the midpoint in enrollment in its Phase 3 HyperAcute Pancreas immunotherapy product candidate (algenpantucel-L) trial as number of participating institutions reached to 74 sites around the US.

"Now that we have passed the midway point of trial enrollment, we are confident that the trial will be fully enrolled before the end of 2013," said Dr. Nick Vahanian, President and Chief Medical Officer of NewLink Genetics. He added, "We are very thankful for the support we have received from our patients and investigators and based on currently available data we anticipate reaching the trigger event for the first interim analysis in early 2013."

The current Phase 3 study is designed to evaluate the benefit of HyperAcute Pancreas in up to 722 patients who have undergone resection of pancreatic cancer. The clinical trial design was approved by the FDA under Special Protocol Assessment prior to opening in May 2010 and the HyperAcute Pancreas was granted Fast Track and Orphan Drug designation in the fourth quarter of 2010.

This Phase 3 trial was initiated based on encouraging results from the Company's Phase 2 study of HyperAcute Pancreas in patients who had undergone resection of pancreatic cancer. The Phase 2 results were recently updated and showed one, two and three year survival of 86% , 51% and 42% respectively, based on Kaplan-Meier analysis with a 33 months of median follow up. Demonstrating improvements of 37%, 59% and 121% over predicted one, two and three outcomes respectively based upon nomogram analysis of these same patients.

About algenpantucel-L

NewLink's algenpantucel-L immunotherapy product candidate consists of a group of two allogeneic pancreatic cancer tumor cell lines that were modified to express Alpha-Gal. These cell lines were chosen to provide a broad coverage of pancreatic cancer antigens. Each of the modified cell lines is grown in large cultures, harvested, irradiated and packaged. Approximately 150 million cells of each HyperAcute Pancreas cell line are given by intradermal injection with each treatment. A series of up to 14 treatments using both cell lines over a period of six months was used in NewLink's Phase 2 clinical trial. In NewLink's Phase 3 trial protocol, NewLink is adding an additional series of monthly, maintenance treatments, to be given during the next six months.

About algenpantucel-L clinical trials

About the Phase 2 Study

The multi-institutional, open-label, dose-finding, Phase 2 trial evaluated the use of algenpantucel-L in addition to chemotherapy with chemoradiotherapy in the adjuvant setting for resected pancreatic cancer. Adjuvant therapy was to start within seven weeks after surgery. The first cycle of treatment consisted of vaccination with either 100 million or 300 million cells per dose given intradermally on days 1 and 8. One week after the second vaccination, gemcitabine was administered at 1000mg/m2/week for three weeks, on days one, eight, and 15, in conjunction with HyperAcute Pancreas immunotherapy dosed on days 1 and 15 of cycle two. Chemoradiotherapy was initiated one to two weeks after the completion of cycle two. Continuous infusion 5-FU was administered at 250 mg/m2/day for the entire duration of radiation therapy. HyperAcute Pancreas immunotherapy was administered on days 1, 15, 29, and 43 of the chemoradiotherapy stage. A total of up to 14 vaccinations were dosed for patients who completed the entire study treatment.

About the Phase 3 Study

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NewLink Updates Enrollment Information for Its Pivotal Phase 3 HyperAcute(R) Pancreas Immunotherapy (algenpantucel-L ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--

bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, announced today that both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted an orphan drug designation to its investigational gene therapy product for the treatment of adrenoleukodystrophy (ALD). The product consists of the patients own CD34+ hematopoietic stem cells transduced with bluebird bios lentiviral vector, Lenti-D, encoding the human ABCD1 cDNA. Based on promising early clinical proof of concept results, bluebird bio plans to initiate a Phase 2/3 clinical study in childhood cerebral ALD in both the United States and Europe in 2013.

Receiving orphan drug designation is a positive step forward in our efforts to bring hope to ALD patients and their families, said David Davidson, M.D., chief medical officer of bluebird bio. We believe our lentiviral technology has the potential to be a one-time transformative therapy for patients suffering from rare genetic disorders like ALD for whom there are limited treatment options. bluebird is committed to advancing the clinical and commercial development of our gene therapy platform because of the dramatic benefit it may have on the lives of patients.

Orphan drug designation, which is intended to facilitate drug development for rare diseases, provides substantial benefits to the sponsor, including the potential for funding for certain clinical studies, study-design assistance, and several years of market exclusivity for the product upon regulatory approval.

About ALD

Adrenoleukodystrophy (ALD) is a rare X-linked, inherited neurological disorder that, in its most severe form, causes damage to the myelin sheath (an insulating layer of membranes that surrounds nerve cells in the brain) and progressive dysfunction of the adrenal glands. Also known as Lorenzo's Oil disease, ALD is estimated to affect one in every 21,000 boys worldwide. In the childhood cerebral form (CCALD), symptoms usually occur between the ages of 4 and 10. Boys afflicted with this form of ALD develop normally until the onset of symptoms. The symptoms of this disorder often progress rapidly and, in a matter of years, can lead to a vegetative state and, ultimately, death. Current treatment options are limited to allogeneic stem cell transplantation when there is an appropriate donor. Allogeneic transplants carry a significant risk of serious morbidity and death.

About bluebird bio's CCALD Product Development

bluebird bios CCALD product program has the potential to halt the progression of CCALD by providing a functional ABCD1 gene to the patients own stem cells. These stem cells proliferate, and some of the progeny cells travel to the brain where they become microglial cells incorporating the corrective gene. Data from the first clinical study treating X-linked CCALD patients with the companys lentiviral gene therapy product demonstrated continued stable expression of the transgene and the corresponding ABCD-1 protein for over four years in two CCALD patients, resulting in prolonged disease stabilization. bluebird bio plans to initiate a Phase 2/3 clinical study in CCALD in both the United States and Europe in 2013.

About bluebird bio

bluebird bio is developing innovative gene therapies for severe genetic disorders. At the heart of bluebird bios product creation efforts is its broadly applicable gene therapy platform for the development of novel treatments for diseases with few or no clinical options. The companys novel approach uses stem cells harvested from the patients own bone marrow into which a healthy version of the disease causing gene is inserted. bluebird bios approach represents a true paradigm shift in the treatment of severe genetic diseases by eliminating the potential complications associated with donor cell transplantation and presenting a one-time potentially transformative therapy using a patients own stem cells. bluebird bio has two later stage clinical products in development for childhood cerebral adrenoleukodystrophy (CCALD) and beta-thalassemia/sickle cell anemia. Led by a world-class team, bluebird bio is privately held and backed by top-tier life sciences investors, including Third Rock Ventures, TVM Capital, ARCH Venture Partners, Forbion Capital Partners, Easton Capital and Genzyme Ventures. Its operations are located in Cambridge, Mass. and Paris, France. For more information, please visit http://www.bluebirdbio.com.

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bluebird bio Receives U.S. and European Orphan Drug Designation for Novel Gene Therapy to Treat Adrenoleukodystrophy

17-06-2012 23:59 Gary B Stem Cell Therapy for CMT Part 2 - For more info. visit

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Stem Cell Therapy for CMT-Gary B-part 2.mp4 - Video

SOUTH SAN FRANCISCO, CA--(Marketwire -06/19/12)- Full Spectrum Genetics, Inc., a privately-held protein analysis and engineering platform and product company, today announced a strategic collaboration with an undisclosed leading global pharmaceutical company for the purpose of generating multiple novel therapeutic protein and antibody product candidates.

Under the terms of the agreement, Full Spectrum Genetics will be responsible for engineering and analyzing variants of specified molecules using its MapEng platform. The pharmaceutical company will receive worldwide rights to develop and commercialize product candidates arising from the collaboration. Full Spectrum Genetics will receive an upfront payment to initiate the collaboration and is eligible for additional financial consideration. Additional terms were not disclosed.

About Full Spectrum Genetics

Founded in 2010, Full Spectrum Genetics, Inc. is a privately-held protein analysis and engineering platform and product company. The Company's MapEng platform enables the ultra-high throughput quantification of the effect on binding of every possible single amino acid substitution within a protein binding site. The MapEng platform provides a comprehensive analysis of protein structure-function relationships, with multiple applications for generating better biotherapeutics and diagnostics. For more information on Full Spectrum Genetics and its MapEng platform, visit http://www.fsgene.com.

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Full Spectrum Genetics Enters Strategic Collaboration With a Leading Global Pharmaceutical Company

17-06-2012 02:12 Gary B. Stem Cell Therapy for CMT - For more info. visit

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Stem Cell Therapy for CMT - Gary B-part 1 - Video

BOSTON--(BUSINESS WIRE)--

Sistemic Ltd., a leading provider of microRNA-based problem-solving services and kit-based products to the Cell Therapy community, announced today that chairman and CEO Jim Reid is moderating a panel discussion at the 2012 Bio International Convention on Wednesday, June 20, in Boston. Featuring leaders from the regenerative medicine space, the panel is titled Stem Cell Therapies Fact or Fiction, and will share the lessons learned to-date from Scottish, European and American perspectives on the path to successfulcommercialization of stem cell therapies.

Jim Reid, Sistemic CEO, commented, "Sistemic is very active in the CellTherapyarena and aremembersof Alliance for Advanced Therapies (AAT) and the Alliance for Regenerative Medicine (ARM). We see the ability to raise this topic at the leading world event, BIO 2012, as animportantstep on the path to commercialization of these products which will be transformational in healthcare, and bring hope and cures to many people around the globe."

More information on the panel at BIO 2012:

What: Panel Discussion Featuring Leaders in the Regenerative Medicine Space

When: Wednesday, June 20, 3:00PM EDT

Where: Boston Convention Center, Room 254A

Who: Leaders of the Regenerative Medicine space:

Panel objectives include evaluating lessons learned and best practice including from the Scotland Roadmap for the commercialization of stem cell therapies; identifying global (US and EU) examples of progress in stem cell therapy commercialization; and facilitating a debate on the need for a global, multi-disciplinary approach to successful commercialization of stem cell therapies.

About Sistemic Ltd

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Sistemic to Moderate Regenerative Medicine Panel at 2012 Bio International Convention