AMSTERDAM, The Netherlands, March 28, 2012 /PRNewswire/ --

Amsterdam Molecular Therapeutics (Euronext: AMT - News), a leader in the field of human gene therapy, announced today data demonstrating that one-time administration of the gene therapy Glybera (alipogene tiparvovec) is able to markedly improve chylomicron (fat particles in the blood) metabolism following consumption of a low fat meal. This results in a much reduced level of newly-formed chylomicrons in the bloodstream, which are considered to be the cause of the acute and recurring bouts of pancreatitis seen in lipoprotein lipase deficiency (LPLD) subjects. LPLD is a very rare inherited condition that is associated with increased levels of chylomicrons. These particles carry certain types of fat in the blood, which because they are not removed from the body can cause recurrent pancreatitis. Data were published online in the Journal of Clinical Endocrinology & Metabolism (JCEM, Mar 2012).

"These data show that Glybera has a profound impact on chylomicron metabolism 14 weeks after a single administration. Although the patient cohort is small, due to the rare nature of LPLD, these results are very encouraging," explained Dr. Andr Carpentier, Division of Endocrinology at the Universit de Sherbrooke, Quebec, Candada. "LPLD patients often suffer from extremely painful bouts of pancreatitis, which is believed to be caused by the accumulation of chylomicron particles in the blood."

"This publication provides additional, independent support on the ability of Glybera to restore chylomicron metabolism in LPLD patients. We believe by restoring the body's ability to metabolize these particles in LPLD patients, Glybera treatment results in fewer pancreatitis attacks," stated Carlos Camozzi, Chief Medical Officer at AMT. "LPLD patients are under constant risk of these attacks and the associated excruciating pain."

Study Details

In an open label clinical trial (CT-AMT-011-02), 5 LPLD subjects in Quebec, Canada, were administered alipogene tiparvovec at a dose of 1 x 1012 genome copies per kg. Two weeks before and 14 weeks after administration, chylomicron metabolism, and plasma palmitate (fatty acid) and glycerol appearance rates were determined following ingestion of a low fat meal. Following administration of alipogene tiparvovec, the triglyceride (TG) content of the chylomicron fraction and the chylomicron-triglyceride (TG)/total plasma TG ratio were reduced throughout the postprandial period. The postprandial peak chylomicron level and chylomicron AUC were greatly reduced (by 79% and 93%, 6- and 24 hours after the test meal, respectively). There were no significant changes in plasma fatty acid and glycerol appearance rates. Plasma glucose, insulin and C-peptide also did not change. The data was obtained from AMT's study in patients treated with Glybera in 2009.

About Glybera

AMT has developed Glybera as a treatment for patients with the genetic disorder lipoprotein lipase deficiency.

LPLD is an orphan disease for which no treatment exists today. The disease is caused by mutations in the LPL gene, resulting in highly decreased or absent activity of LPL protein in patients. This protein 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 and life threatening clinical complication of LPLD. Glybera has orphan drug status in the EU and US.

About Amsterdam Molecular Therapeutics

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ScienceDaily (Mar. 27, 2012) A team of researchers led by scientists at Weill Cornell Medical College has designed what appears to be a powerful gene therapy strategy that can treat both beta-thalassemia disease and sickle cell anemia. They have also developed a test to predict patient response before treatment.

This study's findings, published in PLoS ONE, represents a new approach to treating these related, and serious, red blood cells disorders, say the investigators.

"This gene therapy technique has the potential to cure many patients, especially if we prescreen them to predict their response using just a few of their cells in a test tube," says the study's lead investigator, Dr. Stefano Rivella, Ph.D., an associate professor of genetic medicine at Weill Cornell Medical College. He led a team of 17 researchers in three countries.

Dr. Rivella says this is the first time investigators have been able to correlate the outcome of transferring a healthy beta-globin gene into diseased cells with increased production of normal hemoglobin -- which has long been a barrier to effective treatment of these disease.

So far, only one patient in France has been treated with gene therapy for beta thalassemia, and Dr. Rivella and his colleagues believe the new treatment they developed will be a significant improvement. No known patient has received gene therapy yet to treat sickle cell anemia.

A Fresh Approach to Gene Therapy

Beta-thalassemia is an inherited disease caused by defects in the beta-globin gene. This gene produces an essential part of the hemoglobin protein, which, in the form of red blood cells, carries life-sustaining oxygen throughout the body.

The new gene transfer technique developed by Dr. Rivella and his colleagues ensures that the beta-globin gene that is delivered will be active, and that it will also provide more curative beta-globin protein. "Since the defect in thalassemia is lack of production of beta-globin protein in red blood cells, this is very important," Dr. Rivella says.

The researchers achieved this advance by hooking an "ankyrin insulator" to the beta-globin gene that is carried by a lentivirus vector. During the gene transfer, this vector would be inserted into bone marrow stem cells taken from patients, and then delivered back via a bone marrow transplant. The stem cells would then produce healthy beta-globin protein and hemoglobin.

This ankyrin insulator achieves two goals. First, it protects delivery of the normal beta-globin gene. "In many gene therapy applications, a curative gene is introduced into the cells of patients in an indiscriminate fashion," Dr. Rivella explains. "The gene lands randomly in the genome of the patient, but where it lands is very important because not all regions of the genome are the same." For example, some therapeutic genes may land in an area of the genome that is normally silenced -- meaning the genes in this area are not expressed. "The role of ankyrin insulator is to create an active area in the genome where the new gene can work efficiently no matter where it lands," Dr. Rivella says. He adds that the small insulator used in his vector should eliminate the kind of side effects seen in the French patient treated with beta-thalassemia gene therapy.

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Volunteers will stake out locations all over Athens today from the downtown Waffle House at 2 a.m., to Athens City Hall at 4 p.m. to encourage people across the city to register for a bone marrow donor list in the hopes of finding a match for two sick locals.

The need is even more urgent because former Clarke County school nurse Thomasene Smith and Athens Academy sixth-grader Kajal Patel are minorities, said Caitlin Martin, a representative of Be The Match, the national bone marrow donor registry program. Be The Match, the University of Georgia, the Omni Club and Athens Academy have joined together to host a marathon, continuing today at locations across Athens, to help find donors for Smith and Patel by signing more people to the donor registry list.

Minorities have such a poor chance of finding a match because more than 90 percent of the people signed up for the registry are white, Martin said.

Race matters when trying to find a match for a bone marrow donation, and often, family members arent the best fit, Martin said.

Only 30 percent of our patrons have matches within their family, she said.

Holding the marathon for Smith and Patel will help people of minority groups learn that sick people need them to register for the bone marrow donor list, said Kelin Johnson, Omni Ambassador and former Georgia defense back.

Once people know that race matters when finding a bone marrow donor, more donors likely will come forward, Johnson said.

I think it just comes from a lack of education or awareness, he said.

Potential donors might also shy away from registering because they think the process will hurt too much, Martin said.

One of the biggest myths is that its painful, and thats not true, she said.

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Displaying rare unanimity on an issue, the full U.S. 9th Circuit Court of Appeals on Tuesday rejected a request by the federal government thatit reconsidera rulingthat most bone marrow donors can be compensated for providing life-saving marrow stem cells from their blood.

A three-judge panel of the appeals court ruled on Dec. 1 that the process of harvesting marrow cells by filtering a donor's blood wasn't covered by the 1984 National Organ Transplant Act's prohibitionof payment for organs or organ parts.The statute was enacted by Congress before the blood-filtering process was developed and donors were subjected to painful and medically risky surgical extraction of marrow by insertion of a siphoning needle into the hip bone. Compensation for that form of donation remains illegal.

Atty. Gen. Eric H. Holder Jr., on behalf of the federal government, petitioned the court in Januaryfor a new hearing by an 11-judge panel. Department of Justice lawyers argued that the December ruling ignored the clear intent of Congress to prevent money from influencing donation decisions.

The 9th Circuit panel said in its latest ruling thatall 25 active judges on the court were informed of the government's request and none called for a vote on it, signaling their agreement with the December decision. That unusualaccord among the judges who span a broad ideological spectrum might also indicate that the U.S. Supreme Court will be unlikely to take the case for review.

The lawsuit challenging the ban on bone marrow compensation was brought by a group of cancer patients and their families, as well as a marrow transplant specialist and a California nonprofit organization, MoreMarrowDonors.org, aiming to expand the registry of available donors by offering up to $3,000 in housing assistance or scholarships for promising genetic matches.

Violation of the organ transplant act's prohibition on sales of organs or parts thereofcarries heavy fines and up to five years in prison.The 1984 act defined bone marrow as an organ part, while the 9th Circuit's ruling said it was a blood part and not subject to theban on compensation.

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Appeals court stands united on compensation for bone marrow donors

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ScienceDaily (Mar. 26, 2012) A breakthrough using cutting-edge stem cell research could speed up the discovery of new treatments for motor neuron disease (MND).

The international research team has created motor neurons using skin cells from a patient with an inherited form of MND.

Role of protein

Using patient stem cells to model MND in a dish offers untold possibilities for how we study the cause of this terrible disease as well as accelerating drug discovery by providing a cost-effective way to test many thousands of potential treatments said Professor Siddharthan Chandran, Director of the University's Euan MacDonald Centre for MND Research.

The study discovered that abnormalities of a protein called TDP-43, implicated in more than 90 per cent of cases of MND, resulted in the death of motor neuron cells.

This is the first time that scientists have been able to see the direct effect of abnormal TDP-43 on human motor neurons.

The study, led by the University of Edinburgh's Euan MacDonald Centre for Motor Neuron Disease Research, was carried out in partnership with King's College London, Columbia University, New York and the University of San Francisco.

Motor neuron disease

MND is a devastating, untreatable and ultimately fatal condition that results from progressive loss of the motor nerves -- motor neurons -- that control movement, speech and breathing.

The study, funded by the MND Association, is published in the journal Proceedings of the National Academy of Sciences.

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MADISON, Wis., March 28, 2012 /PRNewswire/ --Cellular Dynamics International, Inc. (CDI) today announced an expansion of its existing distribution agreement with iPS Academia Japan, Inc. to include iCell Neurons and iCell Endothelial Cells. The original distribution agreement, announced on June 8, 2011, covered the distribution of CDI's iCell Cardiomyocytes, the first commercially available product based on induced pluripotent stem cells (iPSCs), in Japan.

CDI is the world's largest manufacturer of human cellular tools for drug discovery and safety derived from iPSCs. The company currently manufactures iCell Cardiomyocytes, iCell Neurons and iCell Endothelial Cells with several other cell types, including liver cells, in development.

iPS Academia Japan was originally established to manage the patents and technology arising from the work of Shinya Yamanaka, MD, PhD of Kyoto University. CDI was the first foreign company granted a license to Yamanaka's iPSC patent portfolio by iPS Academia Japan, announced in May 2010.

"The reliability and consistent quality of CDI's cardiomyocytes have proven to be a valuable product offering to our academic and pharmaceutical customers," said Shosaku Murayama, President and CEO of iPS Academia Japan. "We're already seeing demand for additional human cell types manufactured by CDI by our Japanese customers."

Robert Palay, CEO and chairman of the board of CDI, noted, "We view the expansion of our distribution agreement with iPS Academia Japan as a vote of confidence in our ability to provide human iPSC-derived cells in the quantity, quality and purity required for scientists to realize the full potential of their experiments. We look forward to future growth of our relationship with iPS Academia Japan as we launch new human cell types and in vitro human disease models."

About Cellular Dynamics International Cellular Dynamics International, Inc. (CDI) is a leading developer of next-generation stem cell technologies for drug development, cell therapy, tissue engineering and organ regeneration. CDI harnesses its unique manufacturing technology to produce differentiated tissue cells from any individual's stem cell line in industrial quality, quantity and purity. CDI is accelerating the adoption of pluripotent stem cell technology, adapting its methods to fit into standard clinical practice by the creation of individual stem cell lines from a standard blood draw. CDI was founded in 2004 by Dr. James Thomson, a pioneer in human pluripotent stem cell research at the University of Wisconsin-Madison. CDI's facilities are located in Madison, Wisconsin. See http://www.cellulardynamics.com.

About iPS Academia Japan, Inc. iPS Academia Japan, Inc. (AJ) is an affiliate of Kyoto University, and its main role is, among other activities, to manage and utilize the patents and other intellectual properties held/controlled by Kyoto University and other universities in the field of iPSC technologies so that the research results contribute to health and welfare worldwide.

AJ was established in Kyoto in June 2008. AJ's patent portfolio consists of about 60 patent families (the total number of patent applications is about 220 cases) in the iPSC technology as of March 2012, and about 50 license arrangements have been executed with domestic or international enterprises. See http://ips-cell.net.

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27-03-2012 13:19 G. Steven Burrill of Burrill & Company, reflects on current trends, challenges and the future of personalized medicine.

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G. Steven Burrill on Personalized Medicine - Video

Recommendation and review posted by sam

March 27, 2012

According to a new study, using a patients own bone marrow may help repair damaged areas of the heart caused by heart failure.

Researchers found that left ventricular ejection fraction increased by 2.7 percent in patients who received stem cell therapy.

The study, which was presented at the American College of Cardiologys 61st Annual Scientific Session, revealed that the improvement in ejection fraction correlated with the number of CD34+ and CD133+ cells in the bone marrow.

This is the kind of information we need in order to move forward with the clinical use of stem cell therapy, Emerson Perin, MD, PhD, director of clinical research for cardiovascular medicine at the Texas Heart Institute and the studys lead investigator, said at the event.

The study included 92 patients who were randomly selected to receive stem cell treatment or placebo. The patients all had chronic ischemic heart disease and an ejection fraction of less than 45 percent along with heart failure.

Doctors placed a catheter in the hearts left ventricle to inject 3 ccs, or 100 million stem cells, into an average of 15 sites of the stem cell patients hearts.

The doctors used electromechanical mapping of the heart to measure the voltage in areas of the heart muscle and create a real-time image of the heart.

With this mapping procedure, we have a roadmap to the heart muscle, said Dr. Perin. Were very careful about where we inject the cells; electromechanical mapping allows us to target the cell injections to viable areas of the heart.

The trial was designed to determine whether left ventricular end systolic volume and myocardial oxygen consumption improved in patients who received stem cell treatment.

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Cell Therapy Improves Damaged Heart In Study

Recommendation and review posted by simmons

The Worldwide Innovative Networking (WIN) Consortium in personalized cancer medicine proudly announces today that Foundation Medicine, Inc. (FMI), Cambridge, MA, joins WIN Consortium and becomes a member of the Consortium. The organizations' collaborative research strategy will be presented at the WIN2012 Symposium in Paris, France, on 28-29 June.

WIN Consortium, WIN2012, WIN Symposium, Worldwide Innovative Networking in cancer personalized medicine (Photo: Business Wire)

WIN Consortium, is a network of 22 academic institutions and industries across the globe, initiated by Cancer Institute Gustave Roussy (IGR), France and University of Texas MD Anderson Cancer Center, USA (MDACC), focusing on biomarker-driven therapeutic clinical trials conducted worldwide.

"The membership of Foundation Medicine, a CLIA certified laboratory using a next generation sequencing platform (NGS), is an opportunity for WIN to access their extensive screening test for aberrations in genes known to be associated with human cancer. WIN Consortium is sponsoring a unique clinical trial matching the genetics of patients' tumors and their responses to targeted therapies, to enable treatment with the therapies most likely to be effective against each individual patient's cancer. Foundation Medicine's participation will be a key strategic feature of WIN Consortium's innovative trial, conducted by Drs. Jean Charles Soria (IGR, France), Razelle Kurzrock (MDACC, USA), Josep Tabernero (VHIO, Spain) and Raanan Berger, (CSMC, Israel)," said Dr. John Mendelsohn, chairman of WIN Consortium.

"Foundation Medicine's industry and academic partnership through this international network complements the company's core cancer diagnostics capability, a fully informative genomic profile that provides physicians with clinically relevant molecular information designed to assist Oncologists in the selection of therapies for their patients and that to match patients with clinical trials specific for their tumor," said Michael J. Pellini, M.D., president and chief executive officer of Foundation Medicine. "The clinical trial to be announced during WIN2012, represents an important opportunity to use cutting edge NGS technology in an international setting for treatment decisions and we are proud to be supporting WIN Consortium with our platform."

"To hear more about the collaborative efforts of Foundation Medicine and WIN Consortium, join the WIN2012 Symposium in Paris on 28-29 June, a unique scientific event dedicated to the efficacy of personalized cancer therapeutics. Participants include outstanding investigators from academic institutions and industry - in an open exchange format. The WIN2012 scientific program received the endorsements of ASCO, ESMO, UICC and INCa (*). The WIN Symposium is a unique forum and networking opportunity. Plan your travel and register now as June is a busy period in Paris," said Vladimir Lazar, Chief operating officer of WIN Consortium.

Online abstract submission deadline is May 1st. Abstract will be published in a prestigious international journal, referenced on Pub Med. Early registration closed on April 1st.

About Foundation Medicine

Foundation Medicine is dedicated to improving cancer care through the development of comprehensive NGS cancer diagnostics that identify and interpret an ever-growing set of actionable genomic alterations. Founding advisors are world leaders in genome technology, cancer biology and medical oncology. Please visit the company's website at http://www.foundationmedicine.com.

Disclaimers:

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Foundation Medicine, Cambridge, MA, Joins WIN Consortium (Paris) and Becomes Member of the Worldwide Innovative ...

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Posted: Tuesday, March 27, 2012 4:00 am | Updated: 7:09 am, Tue Mar 27, 2012.

The former Pine Tree ISD administration building is filled with vendors selling their wares lots of bling, cosmetics, sunglasses, watches, pashmina ponchos, scarves, purses, cookware, kitchen items, T-shirts and more. The vendors, all women, giggle as they talk to each other and customers alike.

A stranger wouldnt know its a fundraiser for Pine Tree High School sophomore Christopher Sartor, who suffered a spinal cord injury in September while lifting weights at school under the supervision of the high school coaching staff.

No, I dont know him, Sassy Lady Designs vendor Evelyn Hughes said. But Chris is one of ours. And in Pine Tree, we take care of our own.

We still consider ourselves a small community.

Terry Barrett, teacher and sponsor of the Pine Tree Leadership Class, said the students are always involved in community activities, so doing a fundraiser for the student wasnt ever a question.

At Pine Tree, we take care of our own, she said, echoing the phrase that came up repeatedly. This has been a tremendous change of lifestyle for Chris ... and for his family. With this change has come many out of pocket expenses for his family. We are trying to help alleviate part of the burden.

Barrett said the class brainstormed about possible fundraising activities.

There are so many car washes and dinners, she said. The class settled on holding a bazaar and charging per booth. The exception is Masquerade, which is donating a percentage of its proceeds.

During the event Monday, a teenage girl picked up earrings, exclaiming, Oh, mom, can I have these?

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Pine Tree comes to the aid of student with spinal cord injury

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CANTON, Mass., March 27, 2012 /PRNewswire/ --Today MassBio members elected Geoff MacKay, president & CEO of regenerative medicine leader Organogenesis Inc., as chairman of the board of directors at the 2012 MassBio Annual Meeting.

(Photo: http://photos.prnewswire.com/prnh/20120327/NE76866)

During this year's meeting, MassBio members also elected three new board members: Caren Arnstein, senior vice president and Head of Communications for Genzyme, James Hoyes, President of EMD Serono, Inc., and David Lucchino, CEO of Semprus BioSciences. MacKay will serve a two-year term as board chair.

"Geoff has long been a champion and an advocate for the biotechnology industry in Massachusetts and we look forward to his vision for the future of the industry association," Robert K. Coughlin, President & CEO of MassBio.

Mr. MacKay has served as president and CEO of Organogenesis since 2003 and provides significant global, commercial experience spanning the pharmaceutical and biotechnology sectors. He has held numerous leadership positions throughout his career, including in the areas of transplantation, immunology and tissue engineering, and specifically in the regenerative medicine field for the last decade.

"It is an honor to be elected to serve the country's oldest biotech trade association, especially during a time of tremendous growth and change for the industry," said MacKay. "Organogenesis, which was founded as an MIT spin-off, is a Massachusetts success story. Our company has benefitted throughout its development from the state's biotech super-cluster, including the robust confluence of academia, skilled workforce, innovation and investment. During my term, I hope to bring the experiences of a fast-growing life sciences company, as well as the examples it can provide for the industry, to MassBio as it works to strengthen and build the biotech industry of the future."

About MassBio

MassBio, a not-for-profit organization that represents and provides services and support for the Massachusetts biotechnology industry, is the nation's oldest biotechnology trade association. Founded in 1985, MassBio is committed to advancing the development of critical new science, technology and medicines that benefit people worldwide. Representing over 600 biotechnology companies, academic institutions, research hospitals, and service organizations involved in life sciences and health care, MassBio works to advance policy and promote education, while providing member programs and events, industry information, and services.

For more information, visit the Massachusetts Pavilion at the 2012 BIO International Convention (# 137 on the Exhibit Floor) or the organization's website at http://www.massbio.org.

About Organogenesis Inc.

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Organogenesis CEO Geoff MacKay Elected Chairman of MassBio Board of Directors

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25-03-2012 17:39 Dr Timothy Henry is Director of research at the Minneapolis Heart Institute Foundation and an Interventional cardiologist at the Minneapolis Heart Institute/ Abbott Northwestern Hospital. The interview was conducted on 24 March 2012 at the American College of Cardiology's (ACC's) 61st Annual Scientific Session & Expo in Chicago. See more ACC.12 Coverage: http://www.getinsidehealth.com

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Cell therapy to improve quality of life - Video

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March 27, 2012

According to a new study, using a patients own bone marrow may help repair damaged areas of the heart caused by heart failure.

Researchers found that left ventricular ejection fraction increased by 2.7 percent in patients who received stem cell therapy.

The study, which was presented at the American College of Cardiologys 61st Annual Scientific Session, revealed that the improvement in ejection fraction correlated with the number of CD34+ and CD133+ cells in the bone marrow.

This is the kind of information we need in order to move forward with the clinical use of stem cell therapy, Emerson Perin, MD, PhD, director of clinical research for cardiovascular medicine at the Texas Heart Institute and the studys lead investigator, said at the event.

The study included 92 patients who were randomly selected to receive stem cell treatment or placebo. The patients all had chronic ischemic heart disease and an ejection fraction of less than 45 percent along with heart failure.

Doctors placed a catheter in the hearts left ventricle to inject 3 ccs, or 100 million stem cells, into an average of 15 sites of the stem cell patients hearts.

The doctors used electromechanical mapping of the heart to measure the voltage in areas of the heart muscle and create a real-time image of the heart.

With this mapping procedure, we have a roadmap to the heart muscle, said Dr. Perin. Were very careful about where we inject the cells; electromechanical mapping allows us to target the cell injections to viable areas of the heart.

The trial was designed to determine whether left ventricular end systolic volume and myocardial oxygen consumption improved in patients who received stem cell treatment.

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Cell Therapy Improves Damaged Heart In Study

Recommendation and review posted by Bethany Smith

Dublin - Research and Markets (http://www.researchandmarkets.com/research/01d4e5c9/from_genes_to_geno) has announced the addition of John Wiley and Sons Ltd's new book "From Genes to Genomes: Concepts and Applications of DNA Technology, 3rd Edition" to their offering.

Rapid advances in a collection of techniques referred to as gene technology, genetic engineering, recombinant DNA technology and gene cloning have pushed molecular biology to the forefront of the biological sciences. From Genes to Genomes: Concepts and Applications of DNA Technology explains key ideas underlying the most central techniques in the context of the ways in which they are used. The book opens with a brief review of the basic concepts of molecular biology, before moving on to describe the key molecular methods and how they fit together. This ranges from the cloning and study of individual genes to the sequencing of whole genomes, and the analysis of genome-wide information. Finally, the book moves on to consider some of the applications of these techniques, in biotechnology, medicine and agriculture, as well as in research that is causing the current explosion of knowledge across the biological sciences.

Features:

- Major revision of a concise, well-written introduction to genome sequencing technologies.

- Excellent balance between clarity of coverage and level of detail.

- Includes clear, two-colour diagrams throughout.

- Dedicated website will include all figures.

The latest edition of this highly successful textbook introduces the key techniques and concepts involved in cloning genes and in studying their expression and variation.

Key Topics Covered:

1 From Genes to Genomes

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Newswise MAYWOOD, Ill. -- Researchers have identified genetic causes in nearly 1 in 5 patients who suffer a type of heart failure called dilated cardiomyopathy. Carolyn Jones, MD, PhD, of Loyola University Medical Center, is co-author of the study, published in the Journal of Cardiac Failure. First author is Neal Lakdawala, MD, of Brigham and Women's Hospital.

Researchers did genetic testing on 264 patients with dilated cardiomyopathy and found that 17.4 percent had gene mutations associated with the disease. Pediatric patients were more likely to have the mutations than older patients.

The findings will help in the development of new treatments, Jones said. "By understanding the genes involved in dilated cardiomyopathy, we possibly will be able to circumvent the defect."

Also, if a genetic test shows a patient has an inherited form of the disease, it would indicate that other family members also should be tested, Jones said.

Dilated cardiomyopathy is a condition in which the heart becomes weakened, enlarged and unable to pump efficiently. It is the leading reason for heart transplants. In addition to genetic causes, there are environmental causes, including alcohol abuse, atrial fibrillation (irregular heartbeat) and autoimmune diseases such as lupus.

Earlier studies involved genetic testing on carefully selected research subjects. The new study, by contrast, involved genetic testing in real-life clinical practices. Jones was among the physicians in the study who saw patients, obtained their family histories and arranged for their genetic testing, which was done at the Laboratory for Molecular Medicine at the Partners HealthCare Center for Personalized Genetic Medicine in Cambridge, Mass.

The study included an ethnically diverse sample of patients ranging in age from newborn to 71 years. The average age was 26. Children with dilated cardiomyopathy frequently tested positive for mutations, even if they did not have a family history. Conversely, no patient over age 40 had mutations, unless they also had a family history.

Jones is director of Clinical and Cytogenetics and an associate professor in the departments of Pediatrics and Pathology of Loyola University Chicago Stritch School of Medicine.

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Genetic Causes Found in 17 Percent of Patients with Dilated Cardiomyopathy Heart Failure

Recommendation and review posted by Bethany Smith

Public release date: 27-Mar-2012 [ | E-mail | Share ]

Contact: Jim Ritter jritter@lumc.edu 708-216-2445 Loyola University Health System

MAYWOOD, Ill. -- Researchers have identified genetic causes in nearly 1 in 5 patients who suffer a type of heart failure called dilated cardiomyopathy.

Carolyn Jones, MD, PhD, of Loyola University Medical Center, is co-author of the study, published in the Journal of Cardiac Failure. First author is Neal Lakdawala, MD, of Brigham and Women's Hospital.

Researchers did genetic testing on 264 patients with dilated cardiomyopathy and found that 17.4 percent had gene mutations associated with the disease. Pediatric patients were more likely to have the mutations than older patients.

The findings will help in the development of new treatments, Jones said. "By understanding the genes involved in dilated cardiomyopathy, we possibly will be able to circumvent the defect."

Also, if a genetic test shows a patient has an inherited form of the disease, it would indicate that other family members also should be tested, Jones said.

Dilated cardiomyopathy is a condition in which the heart becomes weakened, enlarged and unable to pump efficiently. It is the leading reason for heart transplants. In addition to genetic causes, there are environmental causes, including alcohol abuse, atrial fibrillation (irregular heartbeat) and autoimmune diseases such as lupus.

Earlier studies involved genetic testing on carefully selected research subjects. The new study, by contrast, involved genetic testing in real-life clinical practices. Jones was among the physicians in the study who saw patients, obtained their family histories and arranged for their genetic testing, which was done at the Laboratory for Molecular Medicine at the Partners HealthCare Center for Personalized Genetic Medicine in Cambridge, Mass.

The study included an ethnically diverse sample of patients ranging in age from newborn to 71 years. The average age was 26. Children with dilated cardiomyopathy frequently tested positive for mutations, even if they did not have a family history. Conversely, no patient over age 40 had mutations, unless they also had a family history.

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NEW YORK (AP) -- Analysts said that this week's Supreme Court decision to order a new review of patents held by Myriad Genetics Inc. is not bad news for Myriad, and its shares should trade higher.

THE OPINION: On Monday the Supreme Court ordered a federal appeals court to conduct a new review of a case that challenges two patents held by Myriad. The company has patents on genetic mutations that are linked to increased risk of breast cancer, and its BRACAnalysis test looks for those mutations. The Court of Appeals for the Federal Circuit was ordered look at the case again in light of a ruling the Supreme Court made on Wednesday.

In that case, the court threw out patent claims that were used in a blood test. It said the patents were not valid because they were based on the laws of nature, and those laws cannot be patented.

However analysts say that the two cases are different and the appeals court will probably not change its decision in the Myriad case.

"The Supreme Court's rationale in the Mayo v Prometheus decision does not seem to suggest a conflicting precedent to the relevance of Myriad Genetics' composition claims," said Jefferies & Co. analyst Jon Wood. Wood said the appeals court could make a new decision late in 2012 or early in 2013. After that, Myriad or its opponents can then appeal to the Supreme Court again. If the court hears the case, a final ruling may come in spring 2014.

Myriad, based in Salt Lake City, has many other patents supporting BRACAnalysis, and Wood said those patents should keep similar products off the market until at least 2018. He said the shares should trade higher and maintained a price target of $28 per share.

William Blair & Co. analyst Amanda Murphy said the new review reduces the risk that patents will be ruled invalid.

"We see no reason to believe the Court of Appeals for the Federal Circuit would reach a different decision on the product claims than it did in 2011," she said.

Both analysts rate the stock at the equivalent of "Buy."

THE STOCK: Myriad shares dipped 10 cents to $23.24 in afternoon trading. The shares rose 2.5 percent on Monday, but before that, they had fallen 11.2 percent since March 20.

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Analysts see court positives for Myriad Genetics

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Ambry Genetics, a global leader in genetic services with a focus on clinical diagnostics and genomics, announces the First-Step Exome test. The First-Step Exome reports on all Human Gene Mutation Database (HGMD)-defined genes.

The role of genes in human disease has only been defined in approximately 20% (~4,400 of ~20,000 genes) of the human genome. The First-Step Exome analyzes the DNA sequence of the exons (coding regions) and flanking intronic regions of these ~4,400 genes. Ambry Genetics believes that the targeted exome sequencing provided by the First-Step Exome is appropriate for a variety of whole exome sequencing indications and will yield the answers clinicians are seeking in many clinical scenarios without the added expense and complexity of whole exome analysis.

With the launch of the First-Step Exome, Ambry Genetics now offers unparalleled flexibility in exome testing, said Charles Dunlop, Chief Executive Officer of Ambry Genetics. The First-Step Exome reports on all HGMD-defined genes at a lower price point than other similar tests offered by competitors. Moreover, after clinicians receive results, we offer them the flexibility to easily reflex to our whole-exome Clinical Diagnostic Exome test.

Four individuals with rare genetic conditions for which the cause could not previously be identified were recently successfully diagnosed using Ambry Genetics proprietary new Clinical Diagnostic Exome, three at Kennedy Krieger Institute in Baltimore and one at a large, Ivy League-affiliated university hospital in New York City.

Building on our successful launch of the Clinical Diagnostic Exome, the First-Step Exome provides a realistic option for clinicians who want to utilize whole exome sequencing in the diagnosis of their patients, but are not prepared to explore the uncharted territory of novel genes or incidental findings, said Elizabeth Chao, M.D., Director of Translational Medicine at Ambry Genetics. We believe that the First-Step Exome will become a popular option for clinicians considering exome testing for a variety of indications. For example, this test may be the prudent exome sequencing option for disease phenotypes that have many previously defined genes in the diagnostic differential, but for which traditional genetic testing options for clinical testing are unavailable or cost-prohibitive.

About Ambry Genetics

Ambry Genetics is a College of American Pathologists (CAP)-accredited and Clinical Laboratory Improvement Amendments (CLIA)-certified commercial clinical laboratory with headquarters in Aliso Viejo, Orange County, Calif. Since its founding in 1999, it has become a leader in providing genetic services focused on clinical diagnostics and genomic services, particularly in sequencing and array services. Ambry has established a reputation for unparalleled service and has been at the forefront of applying new technologies to the clinical molecular diagnostics market and to the advancement of disease research. To learn more about testing and services available through Ambry Genetics, visit http://www.ambrygen.com.

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Ambry Genetics Introduces "First-Step Exome™" Sequencing Test

Recommendation and review posted by Bethany Smith

Blocking "don't destroy me" signals that normally sit on the surface of tumor cells and render them resistant to immune-cell attack slows the growth of a broad range of human cancers when they're implanted in mice, researchers have found.

The approach, reported by immunologists at the Stanford University School of Medicine, was effective against ovarian, breast, colon, bladder, liver, prostate and brain cancer cells. If the work can be repeated in people, the approach may someday help doctors marshal defender cells in patients' own bodies to fight cancers, the researchers said.

Key to the work is a cell protein called CD47, which is already being investigated in the treatment of leukemia.

CD47 sits on cell membranes and communicates with various immune cells, including macrophages, which gobble up foreign invaders in the body. It plays an important role in the normal life cycle of healthy red blood cells, telling macrophages to leave the cells alone.

In the study, the scientists injected the animals with antibodies that bind to CD47 and block out its protective signal.

"If we can block this signal, we can get the immune system to eat [the cancer cells] up," said Stephen Willingham, a postdoctoral researcher in the laboratory of immunologist Dr. Irving Weissman at Stanford and first author of a paper about the work.

The Stanford team examined cancer cells removed from patients with a variety of types of solid tumors. They found that CD47 studded the membranes of almost all of the cancer cells in their sample, suggesting that it is a molecule common to all cancers.

Placing the cells in lab dishes, the team administered an antibody: a protein that binds to CD47 and blocks it from warding off immune system cells. Macrophages ate the cells.

The researchers then implanted human tumor cells in mice for further study. They allowed the cancers to grow, and administered the antibody against CD47.

Antibody treatment inhibited the growth of almost all of the solid tumors and was able to wipe out some smaller cancers altogether, according to the report, which was published Monday in the journal Proceedings of the National Academy of Sciences.

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Cancer research targets a key cell protein

Recommendation and review posted by simmons

Nature | Health

A Monsignor and Officer for Studies at the Pontifical Academy for Life called the cancellation a "sad event." Attendees are set to receive an official explanation

March 26, 2012|

By Ewen Callaway of Nature magazine

The Vatican has abruptly cancelled a controversial stem-cell conference that was set to be attended by the Pope next month.

The Third International Congress on Responsible Stem Cell Research, scheduled for 25-28 April, was to focus on clinical applications of adult and reprogrammed stem cells. But a number of the invited speakers, including Alan Trounson, president of the California Institute for Regenerative Medicine in San Francisco, and keynote speaker George Daley, a stem-cell scientist at Children's Hospital Boston in Massachusetts, are involved in research using human embryonic stem cells, which the Catholic Church considers unethical. The previous two congresses had also included scientists who worked on such cells, without generating much controversy.

Father Scott Borgman, secretary of the Church's Pontifical Academy for Life, one of the conference organizers, says that logistical, organizational and financial factors forced the cancellation, which was announced on 23 March. The academy weighs in on bioethical and theological issues that are relevant to Church teachings.

The Catholic News Agency, an independent news service based in Englewood, Colorado, quoted an unnamed academy member who called the cancellation an "enormous relief to many members of the Pontifical Academy for Life, who felt that the presence on its program of so many speakers, including the keynote speaker, committed to embryonic stem cell research, was a betrayal of the mission of the Academy and a public scandal".

"I think the only interpretation is that we are being censored. It is very disappointing that they are unwilling to hear the truth," says Trounson. He had hoped to provide a "balanced perspective" on the potential clinical applications of stem cells, both adult and embryonic.

Meanwhile, some European scientists, who had called for a boycott because they believed the conference unfairly maligned embryonic stem cell research, cheered its cancellation.

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Vatican Calls Off Stem-Cell Conference

Recommendation and review posted by simmons

SALT LAKE CITY, March 26, 2012 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (Nasdaq:MYGN - News) reported today that the Supreme Court of the United States remanded the case of The Association for Molecular Pathology, et al., v. Myriad Genetics, Inc., et al (Docket No. 11-725) to the Federal Circuit Court of Appeals. As a result of this decision by the Supreme Court, the United States Court of Appeals for the Federal Circuit will reconsider their decision dated July 29, 2011, which upheld Myriad's gene patents. In that decision, the Federal Circuit declared that the composition of matter claims covering isolated DNA of the BRCA 1 and BRCA 2 genes are patent-eligible under Section 101 of the United States Patent Act.

"While, this case should not have any direct impact to Myriad and its operations because of our extensive patent estate, it has great importance to the medical, pharmaceutical, biotechnology and other commercial industries, as well as the hundreds of millions of people whose lives are bettered by the products these industries develop based on the promise of strong patent protection," said Peter Meldrum, President and CEO of Myriad Genetics. "Thus, we are prepared to vigorously defend the patent claims granted to Myriad by the U.S. Patent and Trademark Office and believe that we will be successful."

Importantly, Myriad's intellectual property for the BRACAnalysis(R) test is strong with 23 issued patents and approximately 500 claims, including approximately 245 composition of matter claims and 240 method claims. Only 15 claims are at issue in this case; the rest of the claims remain in full force and effect providing Myriad with extensive patent protection.

Myriad is committed to researching and commercializing innovative molecular diagnostics tests, such as the BRACAnalysis test, to assess a person's risk of developing disease, guide treatment decisions and help improve patients' quality of life. As such, the Company plans to continue its strong commitment to promoting women's health in the areas of hereditary breast and ovarian cancer, advancing and fostering research on the BRCA genes, and providing excellent patient access to its test, including offering financial assistance programs to qualifying individuals.

It is important to correct some common misconceptions on the societal impact of "gene" patents; namely that such patents impede research, result in high-cost testing and takeaway a patient's option for confirmatory testing. Myriad believes that statements made to these points in the public press are incorrect. To set the record straight,

Brian M. Poissant, Gregory A. Castanias, Laura A. Coruzzi, Eileen Falvey and Sasha Mayergoyz and other members of the law firm of Jones Day represented Myriad in this matter.

About Myriad Genetics

Myriad Genetics, Inc. (Nasdaq:MYGN - News) is a leading molecular diagnostic company dedicated to developing and marketing transformative tests to assess a person's risk of developing disease, guide treatment decisions and assess a patient's risk of disease progression and disease recurrence. Myriad's portfolio of nine molecular diagnostic tests are based on an understanding of the role genes play in human disease and were developed with a focus on improving an individual's decision making process for monitoring and treating disease. With fiscal year 2011 annual revenue of over $400 million and more than 1,100 employees, Myriad is working on strategic directives, including new product introductions, companion diagnostics, and international expansion, to take advantage of significant growth opportunities. For more information on how Myriad is making a difference, please visit the Company's website: http://www.myriad.com.

Myriad, the Myriad logo, BRACAnalysis, Colaris, Colaris AP, Melaris, TheraGuide, Prezeon, OnDose, Panexia and Prolaris are trademarks or registered trademarks of Myriad Genetics, Inc. in the United States and foreign countries. MYGN-G

Safe Harbor Statement

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Supreme Court of the United States Remands Gene Patenting Case

Recommendation and review posted by Bethany Smith

A benefit fundraiser for two young girls seriously injured in a car accident two weeks ago will be held at Say Cheese Pizza in Mount Shasta Monday, April 2.

A portion of all in-house sales between 5 and 9 p.m. will be donated to the benefit for Ashlynn and Alexis Myrtle account at PremierWest Bank.

Alexis, age 4, suffered a severe head injury in the March 16 accident, and underwent surgery to repair her right eye. She is recovering well but is having some problems coming to terms with the traumatic incident, said Becky Gordon, a family friend who set up the PremierWest community fund.

Ashlynn, 3, suffered a spinal cord injury and is paralyzed from the waist down. Shes now getting used to her wheelchair, said Gordon.

She loves being in it. Shes learning how to use it... its a little hard, because her arms are so short, but shes working on her upper body strength, Gordon said, adding Ashlynn is resilient and "always has a smile on her face."

Ashlynn and Alexis recently moved from Mount Shasta to Redding with their mother, Amanda Pevehouse. They previously attended Chestnut Preschool in Mount Shasta.

The girls were improperly restrained in the back seat of their fathers car when it drifted off the road and hit a concrete bridge abutment in Cottonwood on the evening of March 16, according to the California Highway Patrol. Their father, Nicolaas Myrtle, pleaded not guilty last week to felony DUI and two counts of child endangerment.

While Alexis is recovering at Mercy Medical Center Redding, Ashlynn was transferred to Santa Clara Valley Medical Center in San Jose, where shes getting specialized care for pediatric spinal injuries.

Pevehouse, who has full custody of the girls, has been traveling back and forth between Redding and San Jose to be with both her daughters.

Donations for the family of Ashlynn and Alexis can be made at any PremierWest Bank branch. The account number is 68016754. Donations can also be made the night of the benefit at Say Cheese in a donation jar at the front counter.

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Benefit for injured girls planned for April 2

Recommendation and review posted by sam

From being unable to move the majority of his body in 2008 to taking steps with leg braces today, Mount Shastas Corben Brooks is proving that a spinal cord injury isnt the end of the world.

Three and a half years after a high school football injury left him a quadriplegic, Mount Shastas Corben Brooks is focused on recovery while in New Delhi, India, where hes receiving a third round of stem cell treatments not yet available in the United States.

The ever-optimistic 20 year old can now stand with minimal assistance, take steps with leg braces, wiggle his toes, partially close his hands and feel the majority of his legs.

Corben said hes looking forward to Labor Day Weekend, when his family will host Thunder in the Park in Mount Shasta, an event which will include the raffle drawing for a custom built motorcycle dubbed Corbens Ride, as well as live music, a chili cookoff, pancake breakfast and a poker run. Thunder in the Park will coincide with the Mount Shasta Police Departments Show & Shine car show in attempt to keep visitors in Mount Shasta the entire weekend.

Without the support of our community and countless other people I wouldn't be where I am today, Corben said via email from India last week. I can honestly say that without the help from my family, friends, this wonderful community and all who have so generously gone out of their way to help me, I would not be in the remarkable position that I am today. Thank you is nowhere near an adequate enough word to express my thanks.

Though he knows stem cell treatments are controversial, Corben said after each treatment he sees more function and sensation in his body for up to nine months after returning home.

So far on this trip I have gained new sensation and feeling in the back of my legs and hamstrings and additional feeling on my left foot, Corben said.

The results of a recent MRI also showed encouraging results, said Corben.

What we saw was the stem cells have been reducing the amount of scar tissue in my spinal cord at the injury site, Corben said. With the scar tissue being reduced, my nerves are given the opportunity to reestablish a connection. And we believe that is why I have been seeing continual recovery during and after these treatments.

Since his last visit in 2011, Corben said his walking has improved greatly, thanks to the help of his trainer back at home, Lisa Pigoni.

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Corben Brooks continues on road to recovery

Recommendation and review posted by sam

WINSTON-SALEM, N.C., March 26, 2012/PRNewswire/ -- Tengion, Inc. (NASDAQ: TNGN - News), a leader in regenerative medicine, today reported its financial results for the year ended December 31, 2011 and provided a business and clinical update detailed in a separate press release issued today, in which the Company reported that it has made significant advances in its two lead programs.

"We have made significant progress advancing our Neo-Urinary Conduit and Neo-Kidney Augment programs and I believe we have created strong momentum to build upon as we continue to execute on our aggressive development goals in 2012," stated John L. Miclot, President and Chief Executive Officer of Tengion. "A fourth patient has now been enrolled and implanted in our Neo-Urinary Conduit clinical trial and we have submitted a pre-Investigational New Drug filing to FDA for our Neo-Kidney Augment one quarter ahead of schedule."

Financial Update

For the year ended December 31, 2011, the Company reported an adjusted net loss of $24.4 million, or $1.13 per basic and diluted common share, compared with an adjusted net loss of $25.8 million, or $2.80 per basic and diluted common share, for the same period in 2010.

The decreased adjusted net loss for the 2011 period was primarily due to a decrease in interest expense of $1.2 million resulting from lower average debt balances outstanding in 2011, and a decrease in depreciation expense of $1.7 million resulting from both a change in the estimated useful life of leasehold improvements at the Company's leased facility in Winston-Salem, North Carolina and the impairment of the Company's leased facility in East Norriton, Pennsylvania. These decreases were offset in part by increased general and administrative expense of $1.2 million related to restructuring charges incurred during 2011 and an increase in research and development expense of $0.4 million primarily due to increased preclinical study costs associated with the Company's Neo-Kidney Augment program. The adjusted net loss per basic and diluted common share for the year ended December 31, 2011 was significantly affected by the issuance of common stock in connection with equity financings completed in April 2010 and March 2011.

For the quarter ended December 31, 2011, the Company reported an adjusted net loss of $6.2 million, or $0.26 per basic and diluted common share, compared with an adjusted net loss of $5.9 million, or $0.48 per basic and diluted common share, for the same period in 2010.

The increased adjusted net loss for the 2011 period was primarily due to an increase in research and development expense of $1.0 million related to preclinical studies associated with the Company's Neo-Kidney Augment program. In addition, general and administrative expense also increased $0.3 million primarily due to a restructuring charge. These increases were offset in part by a decrease in depreciation expense of $1.0 million related to a change in the estimated useful life of leasehold improvements at the Company's leased facility in Winston-Salem, North Carolina and the impairment of the property and equipment at the Company's leased facility in East Norriton, Pennsylvania. The adjusted net loss per basic and diluted common share for the fourth quarter ended December 31, 2011 was significantly affected by the issuance of common stock in connection with the equity financing completed in March 2011.

As of December 31, 2011, the Company held $15.3 million in cash, cash equivalents, and short-term investments. Based upon the Company's currently expected level of operating expenditures and debt repayments, the Company expects to be able to fund its operations to September 2012.

Recent Clinical Advancements

In a separate release issued today, the Company reported that it has made significant clinical advances in its two lead programs, as follows:

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Tengion Reports Fourth Quarter and Full Year 2011 Financial Results

Recommendation and review posted by sam

WINSTON-SALEM, N.C., March 26, 2012 /PRNewswire/ --Tengion, Inc. (NASDAQ: TNGN - News), a leader in regenerative medicine, today announced that it has made significant advances in its two lead programs. A fourth patient was implanted in the last several weeks in the ongoing initial clinical trial of the Company's lead product candidate, the Neo-Urinary Conduit. The Company also submitted a pre-Investigational New Drug (IND) filing to the U.S. Food and Drug Administration (FDA) for its lead preclinical program, the Neo-Kidney Augment. Tengion also announced its fourth quarter and full year 2011 financial results in a separate release issued today and will hold a conference call to discuss these updates today, March 26, 2012, at 5:00 p.m. EDT.

"The implanting of the Neo-Urinary Conduit into the fourth patient and the pre-IND filing for our Neo-Kidney Augment are significant achievements for Tengion and we are confident in our ability to execute on our ambitious objectives for these two programs going forward," stated John L. Miclot, President and Chief Executive Officer of Tengion. "We have gained substantial knowledge and insight from the first three patients in the ongoing trial for the Neo-Urinary Conduit and we believe that the surgical modifications implemented for the fourth patient will improve outcomes for current and future patients in the trial. In addition, now that we have submitted a pre-IND filing to the FDA for the Neo-Kidney Augment, we are preparing a proposed clinical trial design and timeline to continue our progress toward establishing clinical proof of concept."

Neo-Urinary Conduit Clinical Program UpdateTengion has now enrolled a fourth patient in the ongoing initial clinical trial of its lead product candidate, the Neo-Urinary Conduit, which is being evaluated in bladder cancer patients requiring a urinary diversion following bladder removal (cystectomy). The trial is designed to assess the safety and preliminary efficacy of the Neo-Urinary Conduit in up to 10 patients, as well as to translate the surgical procedure successfully used in preclinical animal models into clinical trials with human patients. The ongoing initial trial is being conducted at the University of Chicago Medical Center and at The Johns Hopkins Hospital in Baltimore, Maryland.

The goal set by the clinical investigators in this trial has been to optimize the surgical procedure and post-surgical care by incorporating the outcomes observed in each patient into the surgical approach for subsequent patients, as necessary. Data from the first three patients have allowed clinical investigators to make surgical modifications for the trial moving forward in an effort to address conduit patency and vascular supply. Complications that arose in the first three patients were resolved successfully and, following new surgeries to construct a urinary diversion using bowel tissue, all three patients have recovered well.

Upon implanting the fourth patient in the ongoing trial, Tengion and its clinical investigators believe they have translated the surgical technique, including the procedure to provide greater blood supply to the Neo-Urinary Conduit supporting regeneration of urinary tissue; the ureteral attachment to the Neo-Urinary Conduit using an established surgical procedure used in the current standard of care; and the procedure for the stoma incision and passage of the Neo-Urinary Conduit with its blood supply through the abdominal wall. The Company is also collaborating with post-operative care staff at the two trial sites to optimize stoma management for rapid patient recovery.

The Company plans to discuss with the Data Safety Monitoring Board a reduction in the timelines between future patient implants, which is currently 12 weeks. If granted and assuming appropriate safety data, the Company anticipates that it will complete implantation of up to 10 patients by the end of 2012.

Neo-Kidney Augment Preclinical Program UpdateTengion submitted a pre-IND filing to the FDA for its lead preclinical program, the Neo-Kidney Augment, one quarter ahead of schedule. The Neo-Kidney Augment is intended to prevent or delay the need for dialysis or kidney transplant by catalyzing the regeneration of functional kidney tissue in patients with advanced chronic kidney disease (CKD). Following the early submission of the pre-IND, Tengion's Board of Directors has authorized the Company to aggressively pursue the development of its Neo-Kidney Augment program and will retain the full team of employees involved in the Neo-Kidney Augment program.

Tengion has scheduled a meeting with the FDA to discuss the Company's proposed GLP animal study program to support an IND filing. Tengion is also exploring moving forward using the Advanced Therapy Medicinal Products (ATMP) pathway, an established regulatory route in Europe for advanced cell-based therapies. The Company expects to provide an update on its expectations for the clinical trial program in its first quarter 2012 financial results announcement in May.

About the Neo-Urinary ConduitThe Neo-Urinary Conduit is a combination of a patient's own cells and bioabsorbable scaffold that is intended to catalyze regeneration of a native bladder tissue conduit, passively transporting urine from the ureters through a stoma, or hole in the abdomen, into a standard ostomy bag. Standard of care for patients requiring a non-continent urinary diversion uses bowel tissue to construct a conduit for urine to exit from the body. There are over 20,000 urinary diversions performed annually in the United States and Europe. These patients are at risk for complications associated with the use of bowel tissue, as well as for those associated with the surgery to harvest the bowel tissue. The Neo-Urinary Conduit is the only product currently in development that aims to avoid the use of bowel tissue.

About the Neo-Kidney AugmentThe Neo-Kidney Augment is intended to prevent or delay the need for dialysis or kidney transplant by catalyzing the regeneration of functional kidney tissue in patients with advanced chronic kidney disease (CKD). This increase in functional kidney mass could thereby delay or prevent the need for dialysis or kidney transplant in patients with end stage renal disease (ESRD). According to the United States Renal Data System, more than $27 billion in Medicare costs each year are attributable to patients with ESRD and ESRD is associated with an approximate 20% mortality rate per year, with the average life expectancy of a patient initiating dialysis of approximately four years. Tengion scientists have published and presented positive data on the effect of the Company's Neo-Kidney Augment in four different preclinical models of CKD. Two of these preclinical models have been conducted for a sufficiently long period of time to demonstrate durability and an impact on survival.

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Tengion Announces Significant Advances in Neo-Urinary Conduit™ and Neo-Kidney Augment™ Programs

Recommendation and review posted by sam