Archive for the ‘Gene Therapy Research’ Category

April 18, 2013

Brett Smith for redOrbit.com Your Universe Online

Officials and scientists across the United States are debating the role of genetic engineering in agriculture. While some see genetically modified foods as a sign of technological progress and a way to maximize profits, others see it as a potential hazard with unknown effects for the public health.

In Oregon, state legislators recently heard testimony on several bills that would affect genetic engineering in the agricultural industry. These bills would require labels on all agriculture GMOs, as well as ban the importation genetically altered fish.

We have a right to know whats in our food, Scott Bates, of the advocacy group GMO Free Oregon, told the states Agriculture and Natural Resources Committee. He added the technology has not proven to be free of risk for consumers.

Currently, Oregon does not have state restrictions on growing GMOs, generally leaving the US Department of Agriculture in charge of deciding whether genetic engineering in an agricultural product is safe for the farmers field and the store shelf.

Other states are taking notice of the genetic engineering farms are now embracing as a new way to improve crop yields. In Hawaii, some lawmakers and scientists are at odds over how to proceed with respect to agricultural genetic engineering, with some scientists campaigning against the techniques.

I compare GMOs in a way to steroids because steroids are the easy way to go. It makes it easier for people in sports to enjoy but theres side effects, University of Hawaii agriculture professor Hector Valenzuela told local news station KHNL.

In pushing for mandatory labels for GMOs, Valenzuela cited potential secondary impacts, including the contamination of non-GMO farms with modified seeds and increased pesticide spraying that many GMOs require.

So in terms of what adverse effects, you have to ask questions also of the potential side effects of consuming all of those pesticides, Valenzuela said. For a major importer like Hawaii, the debate over genetic engineering in agriculture poses a unique conundrum, since the islands limited space places a premium on maximizing yield.

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Minor Skirmishes In The Great Debate Over Genetic Engineering In Agriculture

Melbourne, A[r 19 : A new University of Melbourne and Austin Health led study has revealed further insight into fainting showing the phenomenon may be genetic and, in some families, only one gene may be responsible.

However, a predisposition to certain triggers, such as emotional distress or the sight of blood, may not be inherited a new study has found.

Fainting, also called vasovagal syncope, is a brief loss of consciousness when your body reacts to certain triggers. It affects at least one out of four people.

The study, published in the prestigious journal Neurology today and led by Professor Samuel Berkovic from the University of Melbourne's Department of Medicine, based at Austin Health, shows fainting may run in families while triggers may not.

"Our study strengthens the evidence that fainting may be commonly genetic," Professor Berkovic said. "Our hope is to uncover the mystery of this phenomenon so that we can recognise the risk or reduce the occurrence in people as fainting may be a safety issue," he said.

Researchers interviewed 44 families with a history of fainting and reviewed their medical records. Of those, six families had a large number of affected people, suggesting that a single gene was running through the family.

The first family consisted of 30 affected people over three generations with an average fainting onset of eight to nine years.

The other families were made up of four to 14 affected family members. Affected family members reported typical triggers, such as the sight of blood, injury, medical procedures, prolonged standing, pain and frightening thoughts. However, the triggers varied greatly within the families.

Genotyping of the largest family showed significant links to a specific region on chromosome 15, known as 15q26. Linkage to this region was excluded in two medium-sized families but not in the two smaller families.

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Fainting may be genetic: Study

Public release date: 19-Apr-2013 [ | E-mail | Share ]

Contact: William Allstetter allstetterw@njhealth.org 303-398-1002 National Jewish Health

A new genome-wide association study of more than 6,000 people has identified seven new genetic regions associated with pulmonary fibrosis. In findings published online in Nature Genetics on April 14, 2013, researchers at National Jewish Health, the University of Colorado and several other institutions found a number of genes associated with host defense, cell-cell adhesion and DNA repair, which provide clues to possible mechanisms underlying this currently untreatable disease.

"This research gives us several new targets for investigation of pulmonary fibrosis ," said David Schwartz, MD, senior author on the paper, Professor of Medicine at National Jewish Health and Chair of Medicine the University of Colorado School of Medicine. "We believe that there are several relatively common genetic risk factors, which combine with repeated lung injury to cause this devastating lung disease."

Pulmonary fibrosis is a potentially deadly scarring of lung tissue. Although there are a number of known contributors to its development, most cases have no known cause. Without an approved medical therapy, patients with the most common form, idiopathic pulmonary fibrosis, survive an average of only two to three years after diagnosis.

"Pulmonary fibrosis has resisted our attempts to find a clearly beneficial treatment," said co-author Kevin K. Brown, MD, Vice Chair of Medicine at National Jewish Health. "This study gives us new insights into how the disease develops. By better understanding this, we can better focus future therapies."

Researchers from more than 20 institutions, led by National Jewish Health and the University of Colorado, confirmed three previously reported genetic risk factors and identified seven new ones, which together account for about one-third of the disease risk.

The team's findings confirmed the risk associated with specific changes in MUC5B, a gene that produces a protein in mucus. Researchers believe variations in this gene may lead to pulmonary fibrosis by interfering with mucosal defense, repair of lung alveoli or direct toxicity to cells.

The researchers also found stronger evidence for the role of telomeres, a protective section of DNA located at the tips of chromosomes. Shorter telomeres are associated with a reduced ability to divide and premature cell death. Previously, two rare genetic mutations had been associated with some forms of pulmonary fibrosis. The research team found common variants in and near those two genes, and a common variant in another gene.

The researchers also identified three genes associated with connections that hold adjoining cells together, known as cell-cell adhesion. Impaired cell-cell adhesion can lead to lost tissue integrity.

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Genome study suggests new strategies for understanding and treating pulmonary fibrosis

Genetic testing isn't the best way to predict who will develop Type 2 diabetes, a new study suggests.

An international team of researchers arrived at this conclusion after tracking health outcomes for more than 15,000 people on four different continents over and three-year period.

The researchers looked at whether 16 genetic markers that are believed to be related to Type 2 diabetes in Caucasians played a role in the development of the disease for people of other ethnicities, particularly, South Asians and self-identified Latinos.

The association between the genetic factor and diabetes was generally quite consistent across the ethnic groups, said Dr. Sonia Anand, a professor of medicine and epidemiology at McMaster University in Hamilton, Ont. and the study's principal investigator.

After making this discovery, the researchers, she said, wanted to find out whether testing for these variants is an effective way to predict whether an individual will develop Type 2 diabetes.

We said, 'How important is this genetic information? Is it better than the information we already use to predict diabetes, and that type of information includes things such as family history of diabetes? What the person's body weight is, do they have fat around the middle and things of that nature?'

Poring over the data they gathered from study participants, the researchers discovered that other clinical factors were better predictors of who would end up developing the condition.

At this point in time, we don't see that genetic information is useful to determine who will develop diabetes versus who will not in a large population, said Anand.

It's probably just as effective for people to determine, their ethnicity, their fasting blood sugar and their family history.

However, Anand didn't fully dismiss the value of genetic testing. She's working on a project to determine how the practice could help convince people who are risk of developing diabetes to adopt a healthy diet and exercise more.

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Genetic testing not useful for predicting Type 2 diabetes, study suggests

Minecraft - Bee Genetics (S05 E132)
Minecraft Season 5 let #39;s play continues on this heavily modded world. Today I focus on beekeeping and in particular genetic machine to help make the bees I w...

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Minecraft - Bee Genetics (S05 E132) - Video

Gene-patenting case reaches US Supreme Court
The US Supreme Court heard the most high-profile genetics case in history on Monday, as justices considered whether private firms should be allowed to patent...

By: AFP

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Gene-patenting case reaches US Supreme Court - Video

Supreme Court Tackles Case of Patent Law, Human Genetics
The Supreme Court heard arguments in a case on whether a biotech company can patent a gene associated with cancer. Jeffrey Brown gets details from National L...

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Supreme Court Tackles Case of Patent Law, Human Genetics - Video

SALT LAKE CITY, April 18, 2013 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (MYGN) today announced that it will issue financial results for the third fiscal quarter 2013 following the close of market on Tuesday, May 7, 2013.

The Company will also host a conference call on Tuesday, May 7, 2013 at 4:30 P.M. Eastern to review the financial results. Participating on the call will be: Peter Meldrum, President and Chief Executive Officer, Mark Capone, President of Myriad Genetic Laboratories, Inc. and Jim Evans, Chief Financial Officer.

To listen to the call, interested parties within the U.S. may dial 800-617-7643 or +1 303-223-2680 for international callers. All callers will be asked to reference reservation number 21654923.

The conference call will also be available through a live webcast at http://www.myriad.com.

A replay of the call will be available two hours after the end of the call for seven days and may be accessed by dialing 800-633-8284 within the U.S. or +1 402-977-9140 for international callers, and entering reservation number 21654923.

About Myriad Genetics

Myriad Genetics is a leading molecular diagnostic company dedicated to making a difference in patient's lives through the discovery and commercialization of transformative tests to assess a person's risk of developing disease, guide treatment decisions and assess risk of disease progression and recurrence. Myriad's portfolio of molecular diagnostic tests are based on an understanding of the role genes play in human disease and were developed with a commitment to improving an individual's decision making process for monitoring and treating disease. Myriad is focused on strategic directives to introduce new products, including companion diagnostics, as well as expanding internationally. 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-F, MYGN-G

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Myriad Genetics to Announce Third Fiscal Quarter 2013 Results on Tuesday, May 7, 2013

The genetics lab at King Faisal Specialist Hospital and Research Center (KFSHRC) has recently received samples of Adam Oliver Syndrome (AOS) patients to conduct more studies on genes responsible for the disease, local media reported. These have been sent by medical researchers in Egypt, Spain and the United States.

The KFSHRC lab has gained recognition and reputation in the last few years because of outstanding genetics studies carried out at these labs.

Two years ago, the KFSHRC developmental genetics lab identified the first genes related to the Adam Oliver Syndrome. The result was widely published in scientific magazines and periodicals. This medical breakthrough has sparked researchers at medical centers in the three countries to seek medical consultancy services at KFSHRC facilities, according to local media.

Dr. Fowzan Al-Kiraie, senior genetics researcher and head of the lab, said AOS leads to partial loss of the scalp and shrinking of limbs. Identification of genetics leading to the disease will help the infected families benefit from the modern technologies so as to avoid syndrome-infected newborns, he said.

He said the identification of the AOS genes will open new avenues to understand the natural evolution of human limbs. He added that researches carried out on mouse embryos indicated that the genes clearly appeared in the beginning of limbs formation and, therefore, more studies were needed to understand the functional aspects of the genes.

The research was funded by King Abdulaziz City for Science and Technology (KACST) and the Harvard Foundation for Medical Research in Dubai. The outcome of the research was published in the April issue of the American Journal of Human Genetics.

According to medical data, one of the specific features of AOS is cutis aplasia congenita (missing hair and/or skin) affecting the posterior part of the skull, with or without an underlying defect of the cranial bone. There may also be varying degrees of terminal transverse defects of either the upper extremities, lower extremities, or both.

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Genetics lab at KFSHRC attracts attention of global researchers

SAN DIEGO, April 19, 2013 /PRNewswire/ -- Ceregene, Inc. today announced the top-line data from its double-blind, randomized, controlled Phase 2b clinical study of CERE-120 (AAV-neurturin), a gene therapy product designed to deliver the neurotrophic factor neurturin, for Parkinson's disease. The trial did not demonstrate statistically significant efficacy on the primary endpoint (UPDRS-motor off). However, one of the "key secondary endpoints" (Diary-off score), as defined and prespecified in the Statistical Analysis Plan, did produce statistically significant benefit. The trial also provided further evidence for the safety of CERE-120 and the dosing methods employed. A marked placebo effect was observed in this trial in that both the sham-surgery-control patients and the CERE-120 treated patients showed significant improvement following their surgery.

Fifty-one (51) patients with moderately advanced Parkinson's disease who could not be satisfactorily controlled with conventional Parkinson's medication were enrolled in the study at 11 leading clinical sites throughout the U.S. Approximately half of the patients received CERE-120 while the other half received sham (placebo) surgery as a control. Patients were monitored for 15-24 months to assess safety and changes in Parkinson's disease symptoms, using multiple endpoints such as the Unified Parkinson's Disease Rating Scale (UPDRS), Daily Diaries that assess motor function throughout the day, and PDQ-39 (a measure of quality of life), among others. Ceregene continues to analyze the data from this trial to gain as much information as possible.

Jeffrey M. Ostrove, Ph.D., president and chief executive officer of Ceregene, Inc. stated, "We are disappointed that we did not achieve broader statistical significance in this small clinical trial, perhaps due in part to the marked placebo effect noted above. That said, we at Ceregene want to acknowledge the courage of all of the patients enrolled in this very important study and their family members. We also would like to acknowledge the Michael J. Fox Foundation for Parkinson's Research for their long-term support as we continued the development of this novel neurotrophic factor-based treatment with the potential to improve symptoms and also slow disease progression."

Raymond T. Bartus, Ph.D., executive vice president and chief scientific officer of Ceregene stated: "While we did not achieve the degree of efficacy we had hoped for in this trial, we are proud that our efforts have helped to establish that gene therapy can provide the enabling technology to safely deliver stable, long-term bioactive protein to targeted sites deep in the human brain and that the Parkinson's disease brain is able to show a positive response to neurotrophic factor stimulation. Hopefully, the information and insight we achieved and shared with the biomedical community will aid in the continuing effort to develop more effective therapies for many of these tragic and dehumanizing neurodegenerative diseases."

C. Warren Olanow, M.D., Chairman Emeritus of the Department of Neurology and Professor of Neuroscience at the Mount Sinai School of Medicine in New York and a clinical advisor to Ceregene noted, "This was an extremely well-conceived and designed study. It is unfortunate for patients that broader benefits from this extremely promising therapy could not be demonstrated in this clinical trial. These results illustrate how difficult it is to establish clinical efficacy with entirely novel therapeutic approaches in complicated neurological diseases. The Ceregene team, its scientific advisors and the participating clinical sites are to be congratulated for that effort and the flawless execution of this difficult scientific study."

Ceregene established a leadership position in the fields of gene therapy and neurotrophic factors for the treatment of neurodegenerative diseases. A total of over 100 patients have been safely dosed in two clinical programs: CERE-120 (AAV-NRTN) for Parkinson's disease and CERE-110 (AAV-NGF) for Alzheimer's disease. A randomized, controlled Phase 2 study of CERE-110 for Alzheimer's is continuing. It is fully enrolled and financially supported in large part by a grant from the NIH, with top line data expected by late 2014. In addition to the Parkinson's and Alzheimer's programs, Ceregene has conducted extensive preclinical work with CERE-120 for Huntington's disease, as well as another gene therapy/neurotrophic factor product (AAV-NT4) for blinding ocular diseases (such as Retinitis Pigmentosa, macular degeneration, diabetic retinopathy and glaucoma) and yet another (AAV-IGF1) for Lou Gehrig's disease (ALS). Ceregene is currently looking at strategic alternatives to advance its AAV gene therapy platform.

About CERE-120 and its Application to Treating Parkinson's Disease CERE-120 is composed of a harmless adeno-associated virus (AAV) vector carrying the gene for neurturin, a naturally occurring protein known to repair damaged and dying dopamine-secreting neurons, keeping them alive and restoring function. Neurturin is a member of the same protein family as glial cell line-derived neurotrophic factor (GDNF). The two molecules have similar pharmacological properties and both have been shown to benefit the midbrain dopamine neurons that degenerate in Parkinson's disease. Degeneration of these neurons is responsible for the major motor impairments of Parkinson's disease. CERE-120 is delivered by stereotactic injection to the terminal fields (i.e., the ends of the degenerating neurons), located in an area of the brain called the putamen, as well as the cell bodies for these same neurons, located in a different area of the brain, called the substantia nigra. Once CERE-120 is delivered to the brain, it provides stable, controlled and highly targeted neurturin expression for years following a single injection, confirmed in both animal and human studies.

About Parkinson's Disease Parkinson's disease is a progressive movement disorder that affects a million people in the United States. Its main symptoms, stiffness, tremors and slowed movements and gait, are caused by a loss of dopamine-containing nerve cells in the substantia nigra, which project their axons to the putamen. Dopamine is a neurotransmitter involved in controlling movement and coordination, so Parkinson's patients exhibit a progressive inability to initiate and control physical movements. There is currently no treatment that can reverse the degeneration of these neurons, let alone cure Parkinson's disease.

About Ceregene Ceregene, Inc. is a San Diego-based biotechnology company focused on the development of nervous system growth factors (neurotrophic factors) as treatments for neurodegenerative and retinal disorders using gene transfer for their delivery. The company has established a leadership position in the fields of gene therapy and neurotrophic factors, having launched 6 separate clinical trials in Parkinson's and Alzheimer's disease, enrolling a total of nearly 200 patients, over 100 of whom have been administered the gene therapy products, some several years ago, with no safety serious issues. Additionally, the company has published 2 dozen peer-reviewed scientific papers describing novel nonclinical and clinical findings. Ceregene's clinical program for Alzheimer's disease involves CERE-110, an AAV2-based vector expressing nerve growth factor (NGF). A fully enrolled multi-center, controlled Phase 2 study with CERE-110 is ongoing, conducted in collaboration with the Alzheimer's Disease Cooperative Study and partially funded by a grant from the National Institutes of Health (NIH). Ceregene was launched in January 2001. The company's investors include Alta Partners, MPM Capital, Hamilton BioVentures, Investor Growth Capital, California Technology Partners and BioSante Pharmaceuticals (BPAX).

About The Michael J. Fox Foundation for Parkinson's Research As the world's largest private funder of Parkinson's research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson's disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson's patients, business leaders, clinical trial participants, donors and volunteers. In addition to funding more than $325 million in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson's research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; increases the flow of participants into Parkinson's disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson's awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world.

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Ceregene Reports Data From Parkinson's Disease Phase 2b Study

Public release date: 19-Apr-2013 [ | E-mail | Share ]

Contact: Katie Delach katie.delach@uphs.upenn.edu 215-349-5964 University of Pennsylvania School of Medicine

PHILADELPHIA A gene therapy study focused on finding a cure for a rare congenital blinding disease has been recognized as one of the ten most outstanding clinical research projects of the year by the Clinical Research Forum (CRF). The study, led by Jean Bennett, MD, Phd, F.M. Kirby professor of Ophthalmology at the University of Pennsylvania School of Medicine, and carried out in collaboration with Penn Medicine's Albert M. Maguire, MD, and Katherine A. High, MD at The Children's Hospital of Philadelphia (CHOP), has been presented with the Distinguished Clinical Research Achievement Award, the second highest given in the CRF's Annual Top 10 Clinical Research Achievement Awards. CRF award winners are cited as the most compelling examples of scientific innovation that results from the nation's investment in clinical research that can benefit human health and welfare.

The results of the most recent phase of the study for Leber's Congenital Amaurosis (LCA) at CHOP have led to the first Phase 3 gene therapy study in the United States and the first Phase 3 gene therapy study in the world for a non-lethal disorder. The team of researchers hopes that the studies could lead to the first approved gene therapy product in the United States.

"The data from our study has already been used to develop additional clinical trials for other blinding diseases," said Bennett. "There are two things that I think are really going to be important from this work: one, that we'll move forward with this particular disease and get approval for the drug that we've been developing, and two, that this could ultimately lead to approved treatments for other currently untreatable conditions."

Published in 2012, the winning studies are the latest in a long tradition of notable health advances that have occurred through clinical research such as eliminating polio, reducing the mortality of AIDS, and improving cancer survival rates that were propelled by combined investment in basic science and clinical research.

"These patients, once rendered blind by LCA, have had their lives transformed and their vision restored by this team's efforts to further gene therapy research," said Joan O'Brien, MD, chair of the department of Ophthalmology at the Scheie Eye Institute at Penn Medicine. "The groundbreaking work sets the stage for the treatment of numerous other blinding conditions, but is also a shining example of what scientists with NIH resources can accomplish for the betterment of humanity."

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The Clinical Research Forum is an organization comprised of the nation's most prestigious and acclaimed academic medical centers and healthcare systems whose goal is to sustain and expand a cadre of talented, well-trained clinical investigators at all stage of career development, and support nurturing environments and comprehensive research capabilities within academic institutions. Its mission is to provide leadership to the national clinical and translational research enterprise and promote understanding and support for clinical research and its impact on health.

Penn Medicine is one of the world's leading academic medical centers, dedicated to the related missions of medical education, biomedical research, and excellence in patient care. Penn Medicine consists of the Raymond and Ruth Perelman School of Medicine at the University of Pennsylvania (founded in 1765 as the nation's first medical school) and the University of Pennsylvania Health System, which together form a $4.3 billion enterprise.

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Penn receives prestigious national award for breakthrough in gene therapy

PHILADELPHIA A gene therapy study focused on finding a cure for a rare congenital blinding disease has been recognized as one of the ten most outstanding clinical research projects of the year by the Clinical Research Forum (CRF). The study, led by Jean Bennett, MD, Phd, F.M. Kirby professor ofOphthalmologyat the University of Pennsylvania School of Medicine, and carried out in collaboration with Penn Medicines Albert M. Maguire, MD, and Katherine A. High, MD at The Childrens Hospital of Philadelphia (CHOP), has been presented with the Distinguished Clinical Research Achievement Award, the second highest given in the CRFs Annual Top 10 Clinical Research Achievement Awards. CRF award winners are cited as the most compelling examples of scientific innovation that results from the nations investment in clinical research that can benefit human health and welfare.

The results of the most recent phase of the study for Lebers Congenital Amaurosis (LCA) at CHOP have led to the first Phase 3 gene therapy study in the United States and the first Phase 3 gene therapy study in the world for a non-lethal disorder. The team of researchers hopes that the studies could lead to the first approved gene therapy product in the United States.

The data from our study has already been used to develop additional clinical trials for other blinding diseases, said Bennett. There are two things that I think are really going to be important from this work: one, that well move forward with this particular disease and get approval for the drug that weve been developing, and two, that this could ultimately lead to approved treatments for other currently untreatable conditions.

Published in 2012, the winning studies are the latest in a long tradition of notable health advances that have occurred through clinical research such as eliminating polio, reducing the mortality of AIDS, and improving cancer survival rates that were propelled by combined investment in basic science and clinical research.

These patients, once rendered blind by LCA, have had their lives transformed and their vision restored by this teams efforts to further gene therapy research, said Joan OBrien, MD, chair of the department of Ophthalmology at the Scheie Eye Institute at Penn Medicine. The groundbreaking work sets the stage for the treatment of numerous other blinding conditions, but is also a shining example of what scientists with NIH resources can accomplish for the betterment of humanity.

The Clinical Research Forum is an organization comprised of the nations most prestigious and acclaimed academic medical centers and healthcare systems whose goal is to sustain and expand a cadre of talented, well-trained clinical investigators at all stage of career development, and support nurturing environments and comprehensive research capabilities within academic institutions. Its mission is to provide leadership to the national clinical and translational research enterprise and promote understanding and support for clinical research and its impact on health.

###

Penn Medicine is one of the world's leading academic medical centers, dedicated to the related missions of medical education, biomedical research, and excellence in patient care. Penn Medicine consists of the Raymond and Ruth Perelman School of Medicine at the University of Pennsylvania (founded in 1765 as the nation's first medical school) and the University of Pennsylvania Health System, which together form a $4.3 billion enterprise.

The Perelman School of Medicine has been ranked among the top five medical schools in the United States for the past 16 years, according to U.S. News & World Report's survey of research-oriented medical schools. The School is consistently among the nation's top recipients of funding from the National Institutes of Health, with $398 million awarded in the 2012 fiscal year.

The University of Pennsylvania Health System's patient care facilities include: The Hospital of the University of Pennsylvania -- recognized as one of the nation's top "Honor Roll" hospitals by U.S. News & World Report; Penn Presbyterian Medical Center; and Pennsylvania Hospital -- the nation's first hospital, founded in 1751. Penn Medicine also includes additional patient care facilities and services throughout the Philadelphia region.

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Penn Researcher Receives Prestigious National Clinical Research Award for Breakthrough in Gene Therapy

Apr. 17, 2013 Researchers at UT Southwestern Medical Center have identified a specific gene that regulates the heart's ability to regenerate after injuries.

The function of the gene, called Meis1, in the heart was not known previously. The findings of the UTSW investigation are available online in Nature.

"We found that the activity of the Meis1 gene increases significantly in heart cells soon after birth, right around the time heart muscle cells stop dividing. Based on this observation we asked a simple question: If the Meis1 gene is deleted from the heart, will heart cells continue to divide through adulthood? The answer is 'yes'," said Dr. Hesham Sadek, assistant professor of internal medicine in the division of cardiology, and senior author of the study.

In 2011, Dr. Sadek's laboratory showed that the newborn mammalian heart is capable of a vigorous, regenerative response to injury through division of its own cells. As the newborn develops, the heart rapidly loses the ability to regenerate and to repair injuries such as heart attacks.

The research team demonstrated that deletion of Meis1 extended the proliferation period in the hearts of newborn mice, and also re-activated the regenerative process in the adult mouse heart without harmful effect on cardiac functions. This new finding demonstrates that Meis1 is a key factor in the regeneration process, and the understanding of the gene's function may lead to new therapeutic options for adult heart regeneration. The findings also provide a possible alternative to current adult heart regeneration research, which focuses on the use of stem cells to replace damaged heart cells.

"Meis1 is a transcription factor, which acts like a software program that has the ability to control the function of other genes. In this case, we found that Meis1 controls several genes that normally act as brakes on cell division," Dr. Sadek said. "As such, Meis1 could possibly be used as an on/off switch for making adult heart cells divide. If done successfully, this ability could introduce a new era in treatment for heart failure."

According to the American Heart Association, almost 6 million people in the U.S. have heart failure, which occurs when the heart cannot pump enough blood and oxygen to support other organs. Heart disease is the leading cause of death for both men and women in the country, according to the Centers for Disease Control and Prevention.

The study received support from the American Heart Association, the Gilead Research Scholars Program in Cardiovascular Disease, the Foundation for Heart Failure Research, and the National Institutes of Health.

The co-first authors of the study are Dr. Ahmed I. Mahmoud, who is now a postdoctoral fellow at Harvard University; Dr. Fatih Kocabas, who is now a postdoctoral fellow at North American College; and Dr. Shalini A. Muralidhar, a postdoctoral research fellow II of internal medicine. Other researchers at UT Southwestern involved in the study are Wataru Kimura, a visiting senior researcher of internal medicine; Ahmed Koura, now a medical student at Ain Shams University in Egypt; Dr. Enzo Porrello, research fellow and faculty member at the University of Queensland in Australia; and Suwannee Thet, a research associate of internal medicine.

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Gene regulates heart's ability to regenerate after injuries

London, April 18 (ANI): Researchers at UT Southwestern Medical Center have reported discovery of a specific gene that regulates the heart's ability to regenerate after injuries.

Scientists led by Dr. Hesham Sadek have demonstrated that the gene Meis1 regulates the regenerative capability of newborn hearts.

The function of the gene, called Meis1, in the heart was not known previously.

"We found that the activity of the Meis1 gene increases significantly in heart cells soon after birth, right around the time heart muscle cells stop dividing," said Dr. Hesham Sadek, assistant professor of internal medicine in the division of cardiology, and senior author of the study.

"Based on this observation we asked a simple question: If the Meis1 gene is deleted from the heart, will heart cells continue to divide through adulthood? The answer is 'yes'," asserted Dr. Sadek.

In 2011, Dr. Sadek's laboratory showed that the newborn mammalian heart is capable of a vigorous, regenerative response to injury through division of its own cells. As the newborn develops, the heart rapidly loses the ability to regenerate and to repair injuries such as heart attacks.

The research team demonstrated that deletion of Meis1 extended the proliferation period in the hearts of newborn mice, and also re-activated the regenerative process in the adult mouse heart without harmful effect on cardiac functions.

This new finding demonstrates that Meis1 is a key factor in the regeneration process, and the understanding of the gene's function may lead to new therapeutic options for adult heart regeneration. The findings also provide a possible alternative to current adult heart regeneration research, which focuses on the use of stem cells to replace damaged heart cells.

"Meis1 is a transcription factor, which acts like a software program that has the ability to control the function of other genes. In this case, we found that Meis1 controls several genes that normally act as brakes on cell division," Dr. Sadek said.

"As such, Meis1 could possibly be used as an on/off switch for making adult heart cells divide. If done successfully, this ability could introduce a new era in treatment for heart failure," he noted.

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Gene that regulates heart's regenerative ability found

The business of DNA is undergoing a revolution. We can already get our genes scanned for the bargain-basement price of $99. Soon well be able to have entire genomes sequenced for less than the cost of a MacBook Air. Thats huge considering that not so long ago it cost billions of dollars to map a single genome.

But the ability of scientists, doctors and companies to tell us how our genes might impact our health now hinges on a case being debated by the U.S. Supreme Court, theinfamously drawn-out Association for Molecular Pathology v. Myriad Genetics. The casedates back to a 2009 lawsuit filed by the American Civil Liberties Union andcenters on the question of whether genes those snippets of DNA that encode the proteins inside cells can be patented.The ACLU and the scientists, advocacy organizations and patients it represents say no because a gene is something that exists in nature, just like a leaf, an oxygen molecule or gold. Myriad Geneticstold the Courtits inventors had created a new, never-before-seen molecule.

Some of the justices dont seem to buy Myriads argument, sparking some hope in the genomics and personalized medicine communities that when the Court finally decides the case probably in late June theyd have reason to celebrate.

What were arguing about is really the future of medicine and either accelerating it or slowing it down, says Dietrich Stephan, the CEO of SV Bio and founder of Navigenics. [Gene patenting] turned into this quagmire that was holding the field back.

We need to be able to instantaneously deliver that information to [patients] and their doctors and not go through all these crazy hoops of paying the license holder.

The real value and potential for innovation, experts say, comes after genes or gene mutations are identified, and that genetic insight is then applied to a new process, method or algorithm. In most cases, those new processes, methods or algorithms would still be patentable, but not the underlying genetic code.

Diagnostic and therapeutic companies would compete on the quality and price of their tests instead of having a monopoly on the entire pipeline, as Myriad does now with its patents on BRCA1 and BRCA2, two genes associated with early-onset breast and ovarian cancers. Women who want to be tested for these genes have to take Myriads test, which can cost several thousand dollars.

As it stands now, the patent-happy U.S. Patent and Trademark Office has doled out thousands of gene patents since the early 1980s, many of which cover human genes. Myriad Genetics is not the only organization that holds these kinds of patents. 23andMe, Incyte Genomics, Genentech, Harvard, MIT and the University of California are but a few of the institutions that have held or continue to hold gene patents. Some of the patents dont require licensing fees and are pretty open. Others are locked up tight. Run afoul of the wrong gene patent, and you can expect a lawyer to get involved.

Myriad has sent researcherscease-and-desist ordersand Harvard and MIT have filed lawsuits. All of which hampers the progress of scientific research, says Rochelle Dreyfuss, a professor at the New York University School of Law who specializes in intellectual property law and science. In order to do a full sequence for a person, you might have to get permission from all of them, says Dreyfuss.Thats a lot of different people, and any one person could just say no.

That defeats the purpose and potential of whole-genome sequencing, and in a way, robs patients of their right to know their own genetics, a point the Association for Molecular Pathology has made in the past, says Dreyfuss. Their argument is that people have a First Amendment right to know the information inside their own bodies, but the courts have never ruled on that issue, she said.

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Health Science | Gene Patents Are Sabotaging the Future of Medicine

SEATTLE--(BUSINESS WIRE)--

NanoString Technologies, Inc., a privately held provider of life science tools for translational research and molecular diagnostic products, today announced that it has secured an option to an exclusive worldwide license for a 186-gene signature that could be used to determine the prognosis of patients diagnosed with the most common type of liver cancer, hepatocellular carcinoma (HCC), or with hepatitis C-related early-stage cirrhosis. HCC is the third leading cause of cancer mortality worldwide and Hepatitis C cirrhosis-related HCC is the most rapidly increasing cause of cancer related death in the United States.

NanoString secured the option to an exclusive worldwide license from The Broad Institute, a leading non-profit research institute of genomic medicine in Cambridge, Massachusetts, acting on behalf of the inventors institutions. The HCC gene signature was invented by Todd Golub, MD, Chief Scientific Officer of the Broad Institute and a group of researchers from the Dana-Farber Cancer Institute, the Icahn Sinai School of Medicine at Mount Sinai, Massachusetts General Hospital, Massachusetts Institute of Technology, The Hospital Clnic of Barcelona, Institut d'Investigacions Biomdiques August Pi i Sunyer (IDIBAPS), Instituci Catalana de Recarca i Estudis Avanats (ICREA), andCentro de Investigacin Biomdica en Red de Enfermedades Hepticas y Digestivas (CIBERehd).

During the period in which the option can be exercised, NanoString plans to assess the feasibility of developing an in vitro diagnostic assay based on the HCC gene signature for use on the nCounter Analysis System.

A paper in the New England Journal of Medicine in 2008 by Hoshida, et al, described the HCC gene signature in connection with a method for conducting gene expression analysis on RNA extracted from liver tissue adjacent to HCC tumors. Using this method, the authors discovered a 186-gene signature that identifies those HCC patients who have a poor prognosis because of a high rate of recurrence after primary treatment. This gene signature was highly correlated with survival in a training set of 82 Japanese patients and was validated in an independent set of 225 patients from the United States and Europe. A paper recently published online in Gastroenterology by Hoshida et al, demonstrated that this same 186-gene signature also identifies those patients with hepatitis C-related early-stage cirrhosis who have a poor prognosis because of their high rate of developing HCC.

Josep M. Llovet, MD, Professor of Medicine and Director of the HCC Program, Liver Diseases, Mount Sinai, Professor ICREA at IDIBAPS-Hospital Clnic, and a co-inventor of the HCC gene signature, commented: By identifying those HCC patients who are at the greatest risk of recurrence, doctors may choose to monitor these patients more regularly or to enter them into clinical trials in the adjuvant setting to reduce the risk of HCC recurrence. The signature also identifies patients with hepatitis C-related early-stage cirrhosis at high risk of developing HCC who are good candidates for entry into surveillance programs or clinical trials of new agents in the chemo-preventive setting.

Yujin Hoshida, M.D., Ph.D., who led the discovery of the signature while a postdoctoral fellow in Todd Golub's laboratory at the Broad Institute and is on the faculty at Mount Sinai, commented: This platform could provide the multiplexed gene expression capabilities needed for clinical diagnostic use of this HCC gene signature, especially given the potential for a large scale global surveillance testing opportunity. We are excited to take this next step in translating the liver cancer gene signature we have applied in research into a clinical diagnostic available to patients worldwide.

NanoString is dedicated to helping patients and physicians by providing information that can inform major clinical decisions to improve patient care, said Brad Gray, President and CEO of NanoString. On the heels of launching our Prosigna breast cancer assay in the European Union and Israel, we are excited at the prospect of once again partnering with a customer of our Life Sciences business to translate their discovery into a diagnostic product.

About Hepatocellular Carcinoma

Hepatocellular carcinoma (HCC), the most common form of liver cancer, is an increasingly prevalent clinical problem and is the third most common cause of cancer-related death globally. The incidence rates of HCC in the United States have historically been lower than in many countries. However, in recent decades, HCC age-adjusted incidence rates have doubled and primary liver cancer mortality rates have increased faster than mortality rates for any other leading cause of cancer. HCC develops from advanced fibrosis of the liver, or cirrhosis, which is estimated to affect one to two percent of the worlds population. The prognosis for patients with advanced HCC is poor, with a reported five-year survival rate of approximately 10 percent due to the high rate of recurrence after initial treatment of the primary tumor. Like many cancers, patient outcomes are better when the disease is detected and treated at an early stage.

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NanoString Technologies Secures Option From The Broad Institute for Exclusive Worldwide License of Liver Cancer Gene ...

SAN DIEGO, April 16, 2013 /PRNewswire/ -- Sequenom, Inc. (SQNM), a life sciences company providing innovative genetic analysis solutions, today announced that earlier this month, its wholly owned subsidiary, the Sequenom Center for Molecular Medicine (Sequenom CMM), surpassed 100,000 MaterniT21 PLUS test samples processed since the launch of its laboratory developed test (LDT) in October of 2011. The MaterniT21 PLUS test was the first commercial non-invasive prenatal test (NIPT) of its kind for the detection of a genetic chromosomal anomaly known as Trisomy 21, the most common cause of Down syndrome.

"There has been a significant clinical benefit of incorporating NIPT technology, such as the MaterniT21 PLUS test, into my practice, and I expect to see this technology utilized as a best practice tool among women at high risk of fetal aneuploidy," said Arnold Cohen, MD, Chairman, Department of Obstetrics and Gynecology, Albert Einstein Medical Center in Philadelphia. "The MaterniT21 PLUS test has enabled me to provide more accurate prenatal test results in a timely manner. It's reliability and ease of use is of great value to my patients and their families."

The MaterniT21 PLUS test has shown high accuracy, precision and sensitivity, and it is offered by Sequenom CMM as outlined by The American College of Obstetricians and Gynecologists Committee on Genetics and the Society for Maternal-Fetal Medicine Publications Committee joint recommendation supporting NIPT in pregnant women at high risk of carrying a fetus with fetal aneuploidy.

"This milestone represents the overwhelmingly positive response we have received from the obstetrics community to the MaterniT21 PLUS test, and we believe the physician loyalty and interest in our offerings validate the superior attributes of our testing services and support," said William Welch, President and COO, Sequenom, Inc. "With the expansion of our testing capacity and continued investment in our team dedicated solely to providing physicians with education and support for the MaterniT21 PLUS test, we expect that Sequenom CMM will surpass the next milestone of accessioning 150,000 test samples in 2013."

In response to the rapid volume growth and medical community adoption of the MaterniT21 PLUS test, Sequenom CMM is in the process of increasing its testing capacity by completing the validation and licensure of an additional location in North Carolina. This additional location is expected to be operational in the second half of 2013 and will increase the total NIPT capacity from the current 200,000 test samples per year to a minimum of 300,000 test samples per year. At the close of December 2012, the annualized run rate for the MaterniT21 PLUS test was more than 120,000 test samples, an average of more than 2,300 samples weekly.

The MaterniT21 PLUS LDT analyzes the relative amount of 21, 18, 13, as well as X and Y chromosomal material in cell-free DNA. The test is intended for use in pregnant women at increased risk for fetal aneuploidy and can be used as early as 10 weeks' gestation.Estimates suggest there are about 750,000 pregnancies at high risk for fetal aneuploidy each year in the United States. The MaterniT21 PLUS test is available exclusively through the Sequenom CMM as a testing service provided to physicians. To learn more about the test, please visit http://www.Sequenomcmm.com.

About SequenomSequenom, Inc. (SQNM) is a life sciences company committed to improving healthcare through revolutionary genetic analysis solutions. Sequenom develops innovative technology, products and diagnostic tests that target and serve discovery and clinical research, and molecular diagnostics markets. The company was founded in 1994 and is headquartered in San Diego, California. Sequenom maintains a Web site at http://www.sequenom.com to which Sequenom regularly posts copies of its press releases as well as additional information about Sequenom. Interested persons can subscribe on the Sequenom Web site to email alerts or RSS feeds that are sent automatically when Sequenom issues press releases, files its reports with the Securities and Exchange Commission or posts certain other information to the Web site.

SEQUENOM, MaterniT21 and MaterniT21 PLUS are trademarks of Sequenom, Inc. All other trademarks and service marks are the property of their respective owners.

About Sequenom Center for Molecular MedicineSequenom Center for Molecular Medicine (Sequenom CMM) is a CAP accredited and CLIA-certified molecular diagnostics reference laboratory currently with two locations dedicated to the development and commercialization of laboratory developed tests for prenatal and eye conditions and diseases. Utilizing innovative proprietary technologies, Sequenom CMM provides test results that can be used by health care professionals in managing patient care. Testing services are available only upon request by physicians. Sequenom CMM works closely with key opinion leaders and experts in obstetrics, retinal care and genetics. Sequenom CMM scientists use a variety of sophisticated and cutting-edge methodologies in the development and validation of tests. Sequenom CMM is changing the landscape in genetic diagnostics. Visit http://www.sequenomcmm.com for more information on laboratory testing services.

Forward-Looking Statements Except for the historical information contained herein, the matters set forth in this press release, including statements regarding increasing demand from for Sequenom CMM's prenatal tests, growing Sequenom CMM's market presence, sales expansion and establishing its leadership position, the Company's 2013 expectations regarding test volumes, capacity expansion including the opening, build-out, and licensure of a third laboratory location in North Carolina for Sequenom CMM, the Company's commitment to improving healthcare through revolutionary genetic analysis solutions, and Sequenom CMM's dedication to the development and commercialization of laboratory developed tests for prenatal and eye conditions and diseases, and changing the landscape in genetic diagnostics, are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially, including the risks and uncertainties associated with market demand for and acceptance and use of technology and tests such as the MaterniT21 PLUS test, reliance upon the collaborative efforts of other parties such as, without limitation, healthcare providers, international distributors and licensees, the Company or third parties obtaining or maintaining regulatory approvals that impact the Company's business, government regulation particularly with respect to diagnostic products and laboratory developed tests, publication processes, the performance of designed product enhancements, the Company's ability to develop and commercialize technologies and products, particularly new technologies such as noninvasive prenatal diagnostics, laboratory developed tests, and genetic analysis platforms, the Company's financial position, the Company's ability to manage its existing cash resources or raise additional cash resources, competition, intellectual property protection and intellectual property rights of others, litigation involving the Company, and other risks detailed from time to time in the Company's most recently filed Quarterly Report on Form 10-Q for the quarter ended September 30, 2012, its most recently filed reports on Form 8-K, and its most recently filed Annual Report on Form 10-K, and other documents subsequently filed with or furnished to the Securities and Exchange Commission. These forward-looking statements are based on current information that may change and you are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. All forward-looking statements are qualified in their entirety by this cautionary statement, and the Company undertakes no obligation to revise or update any forward-looking statement to reflect events or circumstances after the issuance of this press release.

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Sequenom CMM Achieves Milestone Of 100,000 MaterniT21™ Plus Tests Processed

A cancer patient holds the IV tubes during chemotherapy.

STORY HIGHLIGHTS

(TIME.com) -- While more effective chemotherapy agents have improved cancer survival, not all patients benefit from the drugs.

Now, a team of researchers from Academia Sinica and the National Taiwan University College of Medicine say they have identified an eight gene "signature" that predicts how long cancer patients might survive without relapse after undergoing chemotherapy.

Predicting how well patients respond to chemotherapy drugs, the toxic agents that can decimate not just tumor cells but healthy ones as well, involves a complicated calculation of how active tumor genes are, how likely cancer cells are to spread to other sites, and how effective the chemotherapy drugs are in targeting and destroying abnormally growing cells.

To find the genetic markers in cancer cells that might provide clues to their response to chemotherapy, the researchers relied upon the the National Cancer Institute's panel of 60 human cancer cell lines, known as NCI-60, which represent leukemia, melanoma, lung, colon, ovarian, renal, breast and prostate cancers, among others.

From this panel, they identified 633 genes that were associated with a cancer cell's ability to spread, or invade other healthy tissues. They then compared how these genes reacted to 99 different anti-cancer drugs and found eight genes that showed a strong correlation to an elevated response to five chemotherapy agents -- dasatinib, docetaxel, erlotinib, everolimus and paclitaxel.

TIME.com: Cancer and foggy thinking: Is chemotherapy really the cause?

The eight genes seemed to provide patients with a better chance of responding to chemotherapy, but to confirm the link, the researchers also turned to published studies of cancer patients to see if their panel could predict longer survival.

And indeed, lung and breast cancer patients in these trials with the eight gene signature showed a lower risk of recurrence and longer relapse-free survival than patients without the genetic markers.

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Genetic profile can predict post-chemo cancer survival

INDIAN WELLS, Calif. & CAMBRIDGE, Mass.--(BUSINESS WIRE)--

Good Start Genetics, Inc.,an innovative molecular diagnostics company that has developed the new gold standard in carrier screening, presented clinical data at this weeks annual meeting of the Pacific Coast Reproductive Society (PCRS). These data, highlighted in two poster presentations, demonstrate that the companys next-generation sequencing (NGS) based carrier screening platform detects pathogenic (i.e., disease-causing) mutations that would have been missed by conventional technologies.

The results presented this week continue to demonstrate that our proprietary approach using NGS can accurately detect far more disease-causing mutations than traditional carrier screening technologies used in the IVF setting, said Don Hardison, president and CEO of Good Start Genetics. We have taken a rigorous approach to the design and validation of the Good Start technology platform, and we are seeing terrific results in IVF centers across the country. We are pleased to be partnering with the IVF community to detect carriers that would have been missed using other technologies.

The first of two abstracts is titled, Carrier Screening of 4,200 IVF Patients Utilizing Next Generation DNA Sequencing Detects Common, Rare and Otherwise Undetectable Mutations Across Several Diseases. The poster presented at the meeting provides updated data regarding the detection of numerous rare and novel pathogenic mutations among 10 diseases. In this study, using NGS technology in a cohort of over 8,000 subjects, researchers showed that more than 26% of the carriers identified would have been missed had traditional tests been used. The authors conclude that without the NGS technology platform, IVF couples would be at increased risk of having a child with one of these genetic disorders. The data were presented in abstract number P65 (lead author, Stephanie Hallam, PhD).

The second abstract is titled, Carrier Screening for Cystic Fibrosis Among IVF Patients Utilizing Next Generation DNA Sequencing Detects Common, Rare and Otherwise Undetectable Mutations. In this study of nearly 8,500 patients screened for cystic fibrosis (CF) in a clinical setting, investigators report the detection of numerous rare pathogenic (i.e., disease-causing) CF mutations that would have been missed using traditional screening assays. In the conclusion, the authors note that there are significant advantages to NGS over traditional genotyping approaches, including the ability to detect more CF carriers, and across ethnicities, than ever before possible. The data were presented in abstract number P66 (lead author, Stephanie Hallam, PhD).

Since its national launch in April 2012, Good Start Genetics high-complexity, CLIA- and CAP-accredited laboratory has processed tens of thousands of test orders. The GoodStart Select carrier screening service detects both common disease-causing mutations in carriers across all 23 diseases recommended for testing by major medical societies, as well as rare pathogenic mutations that would go undetected by laboratories using older, traditional genotyping-based technologies. For example, most traditional genotyping-based technologies detect about 100 pathogenic mutations for CF, while GoodStart Select detects more than 550 pathogenic mutations.

The Pacific Coast Reproductive Society annual meeting is a premier forum for scientific discussion toward the advancement of the field of reproductive medicine. The meeting is attended by global thought leaders in the reproductive health community, including reproductive endocrinologists, embryologists and allied health professionals from across the country. The data presented by Good Start Genetics at this meeting build on data presented last month at the American College of Medical Genetics and Genomics (ACMG) annual meeting in Phoenix, Ariz.

About Good Start Genetics, Inc.

Good Start Genetics has developed the new gold standard in carrier screening by making testing for the most comprehensive set of known and novel disease-causing mutations accessible for routine clinical practice. After years of development and rigorous validation, Good Start Genetics has harnessed the power of next-generation sequencing and other best-in-class technologies to provide highly accurate, actionable and affordable tests for all disorders recommended for genetic testing by ACOG, ACMG and other key medical societies. For these reasons, fertility specialists and their patients can have a high degree of confidence in their carrier screening results, and no longer have to compromise accuracy for price. For more information, please visit http://www.goodstartgenetics.com.

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Good Start Genetics Reports Clinical Data Showing Its Carrier Screening Technology Detects Mutations in Cystic ...

NASHVILLE, Tenn.--(BUSINESS WIRE)--

Insight Genetics, a molecular diagnostics company focused on advancing precision medicine in cancer care, today announced that its Quality Management System has received ISO 9001:2008 certification. This milestone achievement, issued on March 12, 2013, represents an international consensus on good management practices and quality assurance.

Insight Genetics is dedicated to providing the highest quality services to our customers and partners, said Eric Dahlhauser, Chairman and CEO of Insight Genetics. This certification demonstrates the rigorous quality standards we uphold in our lab and underscores our commitment to physicians caring for those with cancer and to drug developers advancing treatments available to these patients.

ISO 9001:2008 is an internationally recognized standard that requires organizations to demonstrate the ability to consistently provide products and services that meet high quality requirements and continually enhance customer satisfaction through the effective application of its management system. Insight Genetics quality program, led by Regulatory Scientist Rachel Skelton, Ph.D., and Vice President of R&D David Hout, Ph.D., completed the extensive management system audit conducted by National Quality Assurance. The audit meticulously evaluated Insight Genetics quality system that supports its molecular diagnostic services and early assay development for cancer biomarkers.

About Insight Genetics

Insight Genetics is dedicated to improving the lives of cancer patients through advanced molecular diagnostics for precision cancer care. The company specializes in the discovery, development and commercialization of companion diagnostics that detect specific cancer biomarkers and assist in the effective diagnosis and treatment of cancer. Insight partners with leading academic researchers, pharmaceutical and biotechnology companies, clinical reference laboratories, and IVD kit manufacturers to create new standards of care for cancer patients worldwide.www.insightgenetics.com

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Insight Genetics Receives ISO 9001:2008 Certification

BOTHELL, Wash.--(BUSINESS WIRE)--

Seattle Genetics, Inc. (SGEN) announced today that it will report its first quarter 2013 financial results on Tuesday, May 7, 2013, after the close of financial markets. Following the announcement, company management will host a conference call and webcast discussion of the results and provide a general corporate update. Access to the event can be obtained as follows:

LIVE access on Tuesday, May 7, 2013 1:30 p.m. Pacific Time / 4:30 p.m. Eastern Time

REPLAY access

About Seattle Genetics

Seattle Genetics is a biotechnology company focused on the development and commercialization of monoclonal antibody-based therapies for the treatment of cancer. The companys lead program, ADCETRIS (brentuximab vedotin), received accelerated approval from the U.S. Food and Drug Administration in August 2011 and approval with conditions from Health Canada in February 2013 for two indications. In addition, under a collaboration with Millennium: The Takeda Oncology Company, ADCETRIS received conditional marketing authorization from the European Commission in October 2012. Seattle Genetics also has four other clinical-stage ADC programs: SGN-75, ASG-5ME, ASG-22ME and SGN-CD19A. Seattle Genetics has collaborations for its ADC technology with a number of leading biotechnology and pharmaceutical companies, including Abbott, Agensys (an affiliate of Astellas), Bayer, Celldex Therapeutics, Daiichi Sankyo, Genentech, GlaxoSmithKline, Millennium, Pfizer and Progenics, as well as ADC co-development agreements with Agensys and Genmab. More information can be found at http://www.seattlegenetics.com.

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Seattle Genetics to Host Conference Call and Webcast Discussion of First Quarter 2013 Financial Results on May 7, 2013

Damaged or weakened heart muscle that cant pump enough blood to the rest of the body causes a host of problems that fall under the classification of heart failure. Researchers at the University of Washington have discovered a gene-based method they think can restore strength to those muscles and are progressing toward the clinic

The startup they co-founded, BEAT BioTherapeutics Corp., just announced a $2.5 million seed round from CET Capital Partners and the W Fund, which invests in university spinouts in Washington. According to a U.S. Securities and Exchange Commission filing, that funding is coming in the form of debt.

BEAT says it will use the funds to move its therapy into clinical development. That therapy was built on researchers discovery that a small increase in a naturally occurring molecule called dATP appeared to boost the pumping power of the heart muscles, both in speed and force. In animal models, the treatment increases dATP levels in the heart and has shown to restore wall motion and improve overall cardiac performance, the company says.

That technology was spun into a company in 2008 with grants from the National Institutes of Health. Seattle-based BEAT is now led by Michael Kranda, a former chief operating officer of Immunex and CEO of Vaccinogen.

Celladon Corp. and Belgium-based Cardio3 Biosciences are among the companies with heart failure gene therapies already in clinical trials.

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Gene therapy to restore muscle power to the heart nets UW spinoff $2.5 million

Public release date: 16-Apr-2013 [ | E-mail | Share ]

Contact: Andreas M. Zeiher zeiher@em.uni-frankfurt.de The JAMA Network Journals

Treatment that consisted of shock wave (procedure using high-dose ultrasound)-mediated preconditioning of the target heart tissue prior to administration of bone marrow-derived mononuclear cells was associated with significant, albeit modest improvement in left ventricular ejection fraction (a measure of how well the left ventricle of the heart pumps with each contraction) after 4 months in patients with chronic postinfarction heart failure, according to a study in the April 17 issue of JAMA. The results, which require confirmation in larger trials, demonstrate the potential for this type of therapy to reduce adverse clinical events in these chronically ill patients.

"Regenerative therapies have emerged as a promising novel approach to improve heart function and prevent the development of end-stage heart failure. Application of various cell types including bone marrow-, heart tissue- or adipose tissue-derived cell populations were shown to improve cardiac functional recovery. In patients with acute myocardial infarction [heart attack], recent meta-analyses suggested a moderate but sustained enhancement of left ventricular function and improved clinical outcome following administration of bone marrow-derived mononuclear cells (BMCs)," according to background information in the article. "Extracorporeal shock wave treatment has been experimentally shown to increase homing factors in the target tissue, resulting in enhanced retention of applied BMCs."

Birgit Assmus, M.D., of Goethe University Frankfurt, Germany, and colleagues conducted a study to test the hypothesis that shock wave-facilitated cell therapy could improve the efficacy of intracoronary application of autologous (the donor and recipient are the same person) BMCs in patients with chronic postinfarction heart failure. The randomized, placebo-controlled trial was conducted between 2006 and 2011. The interventions included low-dose (n=42), high-dose (n=40), or placebo (n=21) shock wave pretreatment targeted to the left ventricular anterior wall. Twenty-four hours later, patients receiving shock wave pretreatment were randomized to receive intracoronary infusion of BMCs or placebo, and patients receiving placebo shock wave received intracoronary infusion of BMCs.

The researchers found that the primary end point (change in LVEF from baseline to 4 months ) was significantly improved in the shock wave + BMCs group (absolute change in LVEF, 3.2 percent), compared with the shock wave + placebo infusion group (1.0 percent). Regional wall thickening improved significantly in the shock wave + BMCs group (3.6 percent) but not in the shock wave + placebo infusion group (0.5 percent). Overall occurrence of major adverse cardiac events was significantly less frequent in the shock wave + BMCs group (n=32 events) compared with the placebo shock wave + BMCs (n=18) and shock wave + placebo infusion (n=61) groups.

"The results demonstrate that shock wave-facilitated infusion of BMCs beneficially affects global and regional left ventricular contractile function [improved pump function of the heart] and may reduce adverse clinical events in these chronically ill patients," the authors write. "However, the observed beneficial effects on clinical outcome require confirmation in larger clinical end point trials."

###

(JAMA. 2013;309(15):1622-1631; Available pre-embargo to the media at http://media.jamanetwork.com)

Editor's Note: Please see the article for additional information, including other authors, author contributions and affiliations, financial disclosures, funding and support, etc.

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Outcomes for treating heart failure with cell therapy, high-dose ultrasound

By Margaret Steele HealthDay Reporter

MONDAY, April 15 (HealthDay News) -- The question of whether human genes can be patented is at the center of a case to be heard Monday by the U.S. Supreme Court.

The court's decision could have a profound effect on medical research in the country, efforts to fight diseases such as breast and ovarian cancer, and the multi-billion dollar medical and biotechnology industry, experts say.

The U.S. Patent and Trademark Office has been granting patents on human genes for more than 30 years, according to the Associated Press. The current case stems from a 2009 lawsuit filed by the American Civil Liberties Union on behalf of breast cancer patients and health professionals challenging the validity of Myriad Genetics' patents on two genes associated with breast and ovarian cancer risk.

BRCA1 and BRCA2 gene mutations are linked to increased risk of breast and ovarian cancer, and Myriad sells the only BRCA gene test, which gives them a monopoly on a highly profitable business, critics say.

Opponents also say that allowing companies to patent human genes or parts of human genes will hinder potentially lifesaving research to fight diseases such as breast cancer.

"What that means is that no other researcher or doctor can develop an additional test, therapy or conduct research on these genes," Karuna Jagger, executive director of Breast Cancer Action, told the AP.

Myriad has the patent on a specific method of isolation and identification of specific BRCA mutations, explained Dr. Iuliana Shapira, director of cancer genetics at North Shore-LIJ Cancer Institute in Lake Success, N.Y.

The way Shapira sees it, patents allow a temporary legal monopoly over the use of an invention, but genes are "not invented" by human intelligence and therefore cannot be patented.

On the other hand, if scientists "edit" the gene, for example removing parts of it or introducing some parts and thus creating "a synthetic gene" -- something that does not exist in nature and has specific functions -- that type of "synthetic gene" is patentable, Shapira said.

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Human Gene Patent Case Goes to Supreme Court

15 April 2013 Last updated at 16:13 ET

The US Supreme Court has heard arguments questioning whether the human genome can be claimed as intellectual property.

The case relates to a lawsuit filed by the American Civil Liberties Union in 2009, and centres on whether companies should be able to patent genes.

US authorities have been awarding patents on genes to universities and medical companies for almost 30 years.

The case may have far-reaching repercussions for future gene research.

Currently, researchers and private companies work to isolate genes in order to use them in tests for gene-related illnesses, and in emerging gene therapies.

According to researchers at Weill Cornell Medical College in the US, patents now cover some 40% of the human genome.

The ACLU lawsuit, filed in conjunction with the Public Patent Foundation, relates to seven patents on two human genes held by US firm Myriad Genetics.

The genes are linked to breast and ovarian cancer, and Myriad has developed a test to look for mutations in these genes that may increase the risk of developing cancer.

The company argues that the genes patented were "isolated" by them, making them products of human ingenuity and therefore patentable.

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US court hears gene patents case