Archive for the ‘Gene Therapy Research’ Category

RICHMOND, Calif., May 21, 2012 /PRNewswire/ --Sangamo BioSciences, Inc. (SGMO) announced today that data from clinical, preclinical and research-stage programs focused on the development of ZFP Therapeutics for HIV/AIDS, monogenic diseases and stem cell applications, were described in twelve presentations given by Sangamo scientists and collaborators at the 15th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). The meeting was held in Philadelphia from May 15-19, 2012.

"Sangamo's zinc finger DNA-binding protein (ZFP) technology is enabling development of new and improved gene and cell therapy approaches," said Geoff Nichol, M.B., Ch.B., Sangamo's executive vice president, research and development. "Our ZFP Nuclease (ZFN) technology provides an extremely efficient and precise process for editing any DNA sequence. This enables us to disrupt specific genes or to precisely add DNA sequences that allow a patient's own gene to be corrected and its proper function restored while preserving the natural regulation of the gene.

Sangamo has also developed technology that allows a therapeutic gene to be inserted into a specific 'safe harbor' site. Our ability to target changes to precise locations rather than randomly into the genome, avoids the challenges of traditional gene-addition approaches that can result in unintended mutations. The increased number of related presentations at this meeting demonstrates the growing adoption of ZFN-based gene editing by the field."

Presentations from Sangamo included preliminary clinical data from ongoing Phase 1 clinical trials in HIV/AIDS as well as data from preclinical and research-stage human therapeutic programs. Therapeutic areas included ZFP-based approaches for monogenic diseases such as hemophilia, hemoglobinopathies and Huntington's disease as well as adoptive T-cell therapies for oncology.

"Visibility of ZFPs in the scientific agenda at the ASGCT meeting illustrates the broad range of potential applications for ZFP Therapeutics," said Edward Lanphier, Sangamo's president and CEO. "Our technology can be used to modify any gene with singular specificity and high efficiency. As our technology functions at the DNA level, it can potentially be applied to any disease-related gene making it a versatile platform for the generation of novel therapeutic approaches for the treatment of unmet medical needs."

ZFP Therapeutics Featured at ASGCT Meeting

All abstracts for the meeting are available online at 2012 ASGCT Meeting Abstracts.

About Sangamo

Sangamo BioSciences, Inc. is focused on research and development of novel DNA-binding proteins for therapeutic gene regulation and genome editing. It has ongoing Phase 2 and Phase 1/2 clinical trials to evaluate the safety and efficacy of a novel ZFP Therapeutic for the treatment of HIV/AIDS.Sangamo's other therapeutic programs are focused on monogenic diseases, including hemophilia and hemoglobinopathies such as sickle cell anemia and beta-thalassemia, and a program in Parkinson's disease. Sangamo's core competencies enable the engineering of a class of DNA-binding proteins known as zinc finger DNA-binding proteins (ZFPs). Engineering of ZFPs that recognize a specific DNA sequence enables the creation of sequence-specific ZFP Nucleases (ZFNs) for gene modification and ZFP transcription factors (ZFP TFs) that can control gene expression and, consequently, cell function. Sangamo has entered into a strategic collaboration with Shire to develop therapeutics for hemophilia and other monogenic diseases and has established strategic partnerships with companies in non-therapeutic applications of its technology including Dow AgroSciences and Sigma-Aldrich Corporation. For more information about Sangamo, visit the company's website at http://www.sangamo.com.

ZFP Therapeutic is a registered trademark of Sangamo BioSciences, Inc. CompoZr is a registered trademark of Sigma-Aldrich Corporation.

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Sangamo BioSciences And Collaborators Highlight Widening Applications Of ZFP Therapeutics® In Presentations At Major ...

WEST BOYLSTON, Mass. & SOUTHBRIDGE, Mass.--(BUSINESS WIRE)--

TransCytos: NEVER BEEN DONE BEFORE

Enginasion and TransCytos announced today that their collaboration has resulted in a prototype transfection technology that is designed to have a dramatic and positive impact on the drug-research industry.

Enginasions product development partner, TransCytos, is developing a novel transfection instrument, Cytofector R1, based on a breakthrough, patent-pending hydrodynamic transfection technology. Transfection, the introduction of genetic material into living cells, is a fundamental and essential genetic engineering process in biomedical research, and drug and gene therapy development. It has revolutionized, worldwide, biotech and pharmaceutical R&D, including the research into such diseases as cancer, diabetes, arthritis, substance abuse, neurological disorders such as Parkinsons and Alzheimers, and also has applications in the study of anxiety, aging, and pain management. Furthermore, transfection is key in the production of recombinant human proteins such as insulin, hormones, antibiotics, and vaccines.

Frost & Sullivan estimates the 2010 transfection market at $350 million, with about 200 million transfections conducted per year. However, existing transfection technologies are limited to a small number of particular cell types just five cell lines make up as much as 50% of the market; in addition, low efficiency and cell viability, as well as very slow cell recovery, are slowing progress.

Because the new TransCytos transfection technology is gentle, highly effective, and does not physically damage cells, it is potentially capable of transfecting all cell types, says Dr. Otto Prohaska, CEO of TransCytos. Current transfection techniques represent a considerable bottleneck for biomedical and pharma R&D due to low efficiency, high variability, cellular toxicity, and the inability to introduce genetic material into many of the most important cell types relevant to major diseases. The majority of cells are hard or impossible to transfect, requiring lengthy, expensive procedures with low yield and poor reproducibility. Field testing of the Cytofector R1 prototype instrument showed (a) transfection of previously non-transfectable cells (e.g. neurons), and (b) better transfection efficiencies and expression of gene products in a shorter period of time, and at lower cost.

The TransCytos transfection process could contribute to a faster and more dependable path to drug discovery, a higher success rate for biotech and pharma, and better cures, added David Bonneau, CEO of Enginasion. The capability of transfecting primary cells effectively is expected to revolutionize progress in research, and especially in drug discovery, development, and production. Enginasion is very proud to be the product-development partner of TransCytos.

Click NEVER BEEN DONE BEFORE link (above) for further information about TransCytos.

About Enginasion

For more than two decades, Enginasion (formerly Industrial Automation Systems) has been an invaluable partner to companies in the Industrial, Medical Technology, Military and Pharmaceutical sectors that need a sophisticated yet affordable engineering resource to help them overcome critical hurdles related to automation, control electronics, embedded software, and/or integrated product development. Since 1995, the Company has been providing key elements of successful R&D projects, manufacturing processes, and new high-tech products at innovative companies in the Northeast U.S.

Originally posted here:
Enginasion Partners with TransCytos to Develop Breakthrough ‘Transfection’ Technology for Genetic Engineering ...

(RTTNews.com) - Viral Genetics Inc. (VRAL.PK) announced that a physician-initiated Investigational New Drug or P-IND application submitted to the FDA in late April, 2012, has cleared the FDA's screening process with the requirement for a regular IND application being waived, resulting in the company being able to begin the first of at least two proposed clinical trial sites to investigate a potential oncology treatment developed from Viral Genetics'Metabolic Disruption Technology or MDT, which is licensed exclusively to the Company.

Enrollment and treatment of patients is expected to commence upon completion of internal hospital Institutional Review Boards (IRBs), which are already underway. The UT Health Science Center portion of the study will commence when all approvals are finalized.

The company said the clinical trial will examine the safety and efficacy of one of Viral Genetics licensed MDT compounds in combination with an existing cancer drug, sorafenib (marketed as Nexavar) in the treatment of patients resistant or otherwise unsuitable for standard treatments for stage III or IV ovarian cancer and related carcinomas.

The Company expects to introduce various other drugs and drug combinations from both of its licensed MDT and Targeted Peptides Technology (TPT) platforms to the clinic throughout 2012 and in 2013 with the submission of several additional pre-IND, P-IND and regular sponsor IND applications.

For comments and feedback: contact editorial@rttnews.com

http://www.rttnews.com

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Viral Genetics' P-IND Clears FDA Process To Begin Clinical Trials In Humans

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

A physician-initiated Investigational New Drug (P-IND) application submitted to the FDA in late April, 2012, has cleared the FDAs screening process with the requirement for a regular IND application being waived, resulting in the company being able to begin the first of at least two proposed clinical trial sites to investigate a potential oncology treatment developed from Viral Genetics (Pinksheets:VRAL.PK - News) Metabolic Disruption Technology (MDT), which is licensed exclusively to the Company. The P-IND part of a larger, coordinated research effort was submitted by the first test site at the Cancer Therapy & Research Center (CTRC) at The University of Texas Health Science Center at San Antonio, which includes patient enrollment at Scott and White Hospital (S&W) in Temple, Texas. Additional test sites may also be added in the future.

Enrollment and treatment of patients is expected to commence upon completion of internal hospital Institutional Review Boards (IRBs), which are already underway. The UT Health Science Center portion of the study will commence when all approvals are finalized.

This clinical trial a milestone in the Companys transition from preclinical to clinical-stage will be the first for the intellectual property developed by Dr. M. Karen Newell Rogers, Viral Genetics Chief Scientist and licensed exclusively by the Company. It will examine the safety and efficacy of one of Viral Genetics licensed MDT compounds in combination with an existing cancer drug, sorafenib (marketed as Nexavar) in the treatment of patients resistant or otherwise unsuitable for standard treatments for stage III or IV ovarian cancer and related carcinomas.

The Primary Investigator on the trial is Tyler Curiel, M.D., MPH, a medical oncologist affiliated with the CTRC at the UT Health Science Center. Dr. Curiel is leading this study as he investigates the efficacy of combining two compounds in a cancer treatment that is hoped to cause the starvation of tumor cells and enhanced anti-tumor immunity, leading to the reduction of tumor size and reduced disease progression.

An internationally recognized expert and speaker in tumor immunology and in the humanities, Dr. Curiel has been a featured scientist on the NOVANow Science Television program on PBS, and is the recipient of numerous medical and civic service awards including the Mauveny Prize for Cancer Research from Tulane University, the STARS Research Enhancement Award from the University of Texas and the Health Care Hero Award from the San Antonio Business Journal. Dr. Curiel holds membership in a multitude of professional organizations and sits on the Editorial Boards of several publications including The Journal of Immunology and The Journal of Clinical Investigation.

Discussing Dr. Curiel's role as lead investigator and study chair, Viral Genetics' Chief Scientist, Dr. M. Karen Newell Rogers said, As one of the country's foremost ovarian cancer specialists, having Dr. Curiel as our primary investigator is an honor and we are grateful for the opportunity to validate MDT science as an approach to cancer therapy. MDT shows great promise across a broad spectrum of cancers, and other disease states. A successful clinical trial for this specific carcinoma using this combination approach could allow us to offer a new treatment option for many women and relief to their families.

The Company looked to start similar clinical trials utilizing MDT compounds on certain cancers at S&W in 2011, with funding for the research provided by an anonymous donor who gifted a $1.5 million grant to the Scott and White Foundation. This $1.5 million grant remains in place specifically to fund this research, but the scope of the project has been expanded to accommodate the participation of noted oncologist Dr. Tyler Curiel at the UT Health Science Center, and to include the UT Health Science Center in both preclinical studies and organization of the P-IND protocol.

We are very proud of Drs. Newell Rogers and Curiel, their teams, and this milestone accomplishment in the Company's growth plan. As we bring our R&D pipeline from the laboratory to the clinical environment, MDT will continue to be one of two foundations the other being TPT around which we believe we can create tremendous value for our shareholders, said Haig Keledjian, Viral Genetics' President. As important as this specific ovarian cancer study may be, it is important to note that it is just one step in a much larger planned research effort relating to MDT compounds as cancer therapies. While we have targeted ovarian cancer as our first indication, there are numerous other types of cancer we intend to study in the early stages of MDT research. There is much more to MDT than just cancer, and there is much more to Viral Genetics than just MDT. As a shareholder myself, I view this as one of multiple opportunities that help us move closer to licensing fees and revenues while potentially bringing relief to people suffering from debilitating illness.

Ovarian cancer is a particularly deadly form of cancer afflicting approximately 25,000 women each year in the United States. Amongst women, it is the most lethal of the gynecological cancers and the fifth leading cause of cancer death. Viral Genetics and Scott and White Foundation are sponsoring the study that is taking place at the UTHSCSA Cancer Therapy and Research Center and at Scott and White Hospital. The primary endpoints of the study are expected to relate to safety, although efficacy will also be studied in secondary endpoints, and to reflect a dose-escalation design.

Original post:
Viral Genetics’ P-IND Clears FDA To Commence Clinical Trials in Humans

SALT LAKE CITY, May 21, 2012 /PRNewswire/ --Intermountain Healthcare and Myriad Genetics today announced they have signed a collaborative research agreement. The purpose of this agreement is to perform research and validation studies on transformative molecular diagnostic tests being developed by Myriad in an effort to improve the care and treatments for patients at Intermountain and around the world. This collaboration highlights the shared purpose of Myriad and Intermountain in improving outcomes and the quality of life for patients.

The first project under this collaboration, PRO 008, is designed to further expand the utility of the Prolaris test by analyzing biopsy samples of 200 patients diagnosed with prostate cancer. This study will assess the ability of the Prolaris test to predict which men are at a heightened risk of biochemical recurrence and therefore should be given more aggressive therapy for their disease. The goal of this, and Myriad's other Prolaris studies underway in multiple centers in the United States and Europe, is to demonstrate the prognostic ability of the Prolaris test in assessing a patient's risk of biochemical recurrence of disease and death from disease.

"We are thrilled to be partnering with Myriad to further research across a number of diseases in an effort to improve patient care," said Brent Wallace, MD, Intermountain's Chief Medical Officer. "We look forward to embarking on our prostate cancer collaboration with Myriad and hope the findings from this study will help define the clinical benefit of the Prolaris test. This will assist in helping men diagnosed with prostate cancer to understand the aggressiveness of their disease and make better informed decisions about appropriate treatment."

"Intermountain is committed to improving patient outcomes which is in perfect alignment with the core mission of Myriad," said Peter Meldrum, President and Chief Executive Officer of Myriad Genetics."This research collaboration will have great potential to help patients by furthering research on molecular diagnostic tests which can assist healthcare providers to effectively guide treatment decisions and determine the risk of disease progression and recurrence."

About Intermountain Healthcare

Intermountain Healthcare, a nonprofit healthcare system based in Salt Lake City, Utah, serves the healthcare needs of Utah and southeastern Idaho residents. Its mission is to provideclinically excellent medical care at affordable rates in a healing environment. For more information visit intermountainhealthcare.org

About Myriad Genetics

Myriad Genetics, Inc., an internationally recognized leader in molecular diagnostics, is 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.

Safe Harbor Statement

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Intermountain Healthcare and Myriad Genetics Enter Into Research Collaboration Agreement

COLUMBIA, Md.--(BUSINESS WIRE)--

Osiris Therapeutics Inc. (NASDAQ:OSIR - News) announced today it has received market authorization from Health Canada to market its stem cell therapy Prochymal (remestemcel-L), for the treatment of acute graft-vs-host disease (GvHD) in children. The historic decision marks the worlds first regulatory approval of a manufactured stem cell product and the first therapy approved for GvHD a devastating complication of bone marrow transplantation that kills up to 80 percent of children affected, many within just weeks of diagnosis.

"I am very proud of the leadership role Canada has taken in advancing stem cell therapy and particularly gratified that this historic decision benefits children who would otherwise have little hope," said Andrew Daly, M.D., Clinical Associate Professor, Department of Medicine and Oncology at the University of Calgary, Canada and Principal Investigator in the phase 3 clinical program for Prochymal. "As a result of Health Canada's comprehensive review, physicians now have an off-the-shelf stem cell therapy in their arsenal to fight GvHD. Much like the introduction of antibiotics in the late 1920's, with stem cells we have now officially taken the first step into this new paradigm of medicine."

Prochymal was authorized under Health Canada's Notice of Compliance with conditions (NOC/c) pathway, which provides access to therapeutic products that address unmet medical conditions and which have demonstrated a favorable risk/benefit profile in clinical trials. Under the NOC/c pathway, the sponsor must agree to carry out confirmatory clinical testing.

Today is not only a great day for Osiris, but for everyone involved in the responsible development of stem cell therapies, said C. Randal Mills, Ph.D., President and Chief Executive Officer of Osiris. Most importantly, today is a great day for children and their families who bravely face this horrific disease. While today marks the first approval of a stem cell drug, now that the door has been opened, it will surely not be the last.

Health Canadas authorization was made following the recommendation of an independent expert advisory panel, commissioned to evaluate Prochymal's safety and efficacy. In Canada, Prochymal is now authorized for the management of acute GvHD in children who fail to respond to steroids. The approval was based on the results from clinical studies evaluating Prochymal in patients with severe refractory acute GvHD. Prochymal demonstrated a clinically meaningful response at 28 days post initiation of therapy in 61-64 percent of patients treated. Furthermore, treatment with Prochymal resulted in a statistically significant improvement in survival when compared to a historical control population of pediatric patients with refractory GvHD (p=0.028). The survival benefit was most pronounced in patients with the most severe forms of GvHD. As a condition of approval, the clinical benefit of Prochymal will be further evaluated in a case matched confirmatory trial and all patients receiving Prochymal will be encouraged to participate in a registry that will monitor the long-term effects of the therapy.

Refractory GvHD is not just deadly to the patients it afflicts, but is devastating for the family, friends, and caregivers who watch helplessly as the disease progresses, said Joanne Kurtzberg, MD, Head of the Pediatric Bone Marrow Transplant Program at Duke University and Lead Investigator for Prochymal. "I have personally seen Prochymal reverse the debilitating effects of severe GvHD in many of my patients and now, after nearly two decades of research, the data demonstrating consistently high response rates, a strong safety profile and improved survival clearly support the use of Prochymal in the management of refractory GvHD."

Prochymal is currently available in several countries, including the United States, under an Expanded Access Program (EAP). Prochymal will be commercially available in Canada later this year.

Today Osiris turns the promise of stem cell research into reality, delivering on decades of medical and scientific research, said Peter Friedli, Chairman and Co-founder of Osiris. It took 20 years of hard work and perseverance and I want to personally thank everyone involved for their dedication to this important mission.

In addition to the extensive intellectual property protection Osiris has around Prochymal, which includes 48 issued patents, Health Canada's decision will also provide Prochymal with regulatory exclusivity within the territory. Canada affords eight years of exclusivity to Innovative Drugs such as Prochymal, and an additional six-month extension is available since it addresses a pediatric population.

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World's First Approved Stem Cell Drug; Osiris Receives Marketing Clearance from Health Canada for Prochymal

Osiris Therapeutics Inc says Canadian health regulators have approved its treatment for acute graft-versus host disease in children, making it the first stem cell drug to be approved for a systemic disease anywhere in the world.

Osiris shares rose 14 percent to $6.00 in extended trading after the news was announced.

Graft versus host disease (GvHD) is a potentially deadly complication from a bone marrow transplant, when newly implanted cells attack the patient's body. Symptoms range from abdominal pain and skin rash to hair loss, hepatitis, lung and digestive tract disorders, jaundice and vomiting.

The disease kills up to 80 percent of children affected, Osiris said. To date there have been no approved treatments for the disease. Canadian authorities approved the therapy, Prochymal, for use in children who have failed to respond to steroids.

Prochymal was approved with the condition that Osiris carry out further testing after it reaches the market. C. Randal Mills, the company's chief executive, said in an interview that could take three to four years.

Some investment analysts have been skeptical about Prochymal's future. In 2009, two late-stage clinical trials failed to show the drug was more effective overall than a placebo in treating the disease, though it showed promise in certain subgroups of patients.

Since then, the company has mined data from all its clinical trials to show that in patients with severe refractory acute GvHD -- those who have more or less failed all other therapies -- Prochymal demonstrated a clinically meaningful response at 28 days after therapy began in 61-64 percent of patients.

In addition, treatment with Prochymal resulted in a statistically significant improvement in survival when compared with a historical control population of pediatric patients with refractory GvHD.

The Canadian authorities approved the drug on the basis of that data, the company said.

FDA SUBMISSION THIS YEAR

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Canada approves stem cell therapy

By Meg Tirrell - 2012-05-18T20:13:19Z

Osiris Therapeutics Inc. (OSIR) rose after the company said it won the worlds first approval for a stem- cell drug, gaining clearance in Canada to sell Prochymal for a disease that can attack patients who received bone-marrow transplants.

Osiris climbed 5.5 percent to $5.55 at 4 p.m. New York time. The shares have lost 24 percent in the last 12 months.

Prochymal was approved for the treatment of acute graft versus host disease in children for whom steroids havent worked, the Columbia, Maryland-based company said yesterday in a statement. Steroids have a 30 percent to 50 percent success rate, and severe GvHD can be fatal in 80 percent of cases, according to the company.

The therapy uses mesenchymal stem cells derived from bone marrow that can take on different forms to combat the immune reaction that causes patients to literally peel out of their skin and shed their intestinal lining, Osiris Chief Executive Officer Randal Mills said in a telephone interview. The disease has no equal.

The company hasnt sought approval for this indication in the U.S., where regulators asked for more data before considering whether to allow sales of the drug, Mills said. Prochymal is used in eight countries, including the U.S., on an expanded-access program basis, which allows patients to receive experimental medicines without participating in clinical trials.

This is the first regulatory approval of a stem-cell drug -- where the active ingredient of the drug is a stem cell -- in the world, Mills said. Its a huge deal for us and a huge deal for the entire field of stem-cell therapy.

Osiris shares declined from an all-time high of $28.56 in 2007 as the biotechnology company faced clinical setbacks, including two studies in 2009 that failed to show statistical improvement of Prochymal versus placebo.

The Canadian approval was based on data showing a clinically meaningful response 28 days after starting therapy for 61 percent to 64 percent of patients treated, Osiris said in the statement.

Prochymal may draw $16.7 million in revenue next year with Canadian approval, estimated Edward Tenthoff, an analyst with Piper Jaffray & Co., before the companys announcement. He said that while Prochymal would be the first stem-cell drug to receive approval, other regenerative products used for wound- healing that employ stem cells are already on the market, such as Carticel from Sanofis Genzyme unit.

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Osiris Wins Canadian Approval for First Stem-Cell Therapy

LONDON Scientists have mapped the complete genetic codes of 21 breast cancers and created a catalogue of the mutations that accumulate in breast cells, raising hopes that the disease may be able to be spotted earlier and treated more effectively in future.

The research, the first of its kind, untangles the genetic history of how cancer evolves, allowing scientists to identify mutational patterns that fuel the growth of breast tumors, and start to work out the processes behind them.

"These findings have implications for our understanding of how breast cancers develop over the decades before diagnosis in adults and might help to find possible targets for improved diagnosis or therapeutic intervention in the future," said Mike Stratton, who led the research team.

Breast cancer kills more than 450,000 women a year worldwide and is the most common cancer among women, accounting for 16 percent of all cases, according to the World Health Organization (WHO).

A study last year by the Institute for Health Metrics and Evaluation in the United States found that global breast cancer cases have more than doubled in just three decades, from 641,000 cases in 1980 to 1.6 million cases in 2010 - a pace that far exceeds global population growth.

"This is the first time we've been able to delve fully into breast cancer genomes in such a thorough way," said Peter Campbell, head of cancer genetics and genomics at the Wellcome Trust Sanger Institute in Cambridge, where the studies were led.

The work had given scientists "a full panoramic view of the cancer genome" and helped them identify "mutational patterns rather than individual mutations in specific genes", he added.

DNA mutations "We've known for many years now that all cancers are due to abnormalities of DNA...that occur in every single cell of the body over the course of a lifetime," said Stratton.

"But although we've known that, it's remarkable how rudimentary our knowledge is about what the processes are that cause these abnormalities, these mutations in our DNA."

Stratton's team sequenced the genomes of the 21 breast cancers and catalogued all the mutations. They found five major processes that cause one letter of code to be changed to another letter. Genetic code comes in four DNA letters, A,C,G and T.

Read the original here:
Gene changes behind breast cancer untangled

CRO adds TRAC to service portfolio for pre-clinical ADME-Tox studies

HELSINKI, FINLAND - Plexpress, developer of the innovative TRAC platform for high-throughput gene expression analysis, has announced it will partner with SBW to provide SBW's customers with access to Plexpress' novel technology. SBW, a contract research organisation serving the drug discovery market, will offer Plexpress' pre-validated TRAC Cytochrome P450 (CYP) family multiplex assays as part of its portfolio for investigating in vitro drug interactions and pre-clinical ADME-Tox profiles. Such a service is expected to be very popular, especially given a recent draft report from the FDA, which suggests that drug discovery researchers use CYP mRNA induction, rather than CYP activity, to assess drug metabolism and activity (1).

TRAC (Transcript Analysis with the aid of Affinity Capture) technology is a new approach to measuring gene expression changes that offers several benefits over qPCR and microarrays, including higher sample throughput and increased assay speed, while simultaneously reducing technical variation. For more information about how TRAC could boost the efficiency of your drug discovery pipeline, please visit http://www.plexpress.com. For more information about the new SBW service utilising TRAC, view the full release by visiting the SBW website or contact SBW directly at info@sbw.fi.

Reference: 1. FDA Guidance for Industry Report (Feb 2012): Drug Interaction Studies Study Design, Data Analysis, Implications for Dosing, and Labeling Recommendations

About Plexpress Plexpress was established as a spinoff from the Finnish Technology Research Center VTT in 2007. Including R&D at VTT, the company's TRAC technology has been developed continuously for nearly 10 years and it has been documented in over 15 publications. The technology was originally developed for demanding bioprocess applications, which required a very robust solution for gene expression analysis. Since starting operations in 2008, the company has carried out over 50 projects using the TRAC technology in the fields of pharmaceutical development and academic research.

For further information see: http://www.plexpress.com

About SBW SBW is an integrated contract research organization (CRO), located in Helsinki, Finland and has offices also in Germany and North-America. We operate under the brands SBW/Biodix, SBW/Toxis and SBW/novamass. We offer comprehensive services and platforms for drug discovery, functional food ingredients testing, REACH chemical safety assessment and related life science areas. Our services have more than 100 customers in aroung 20 countries in Europe, Asia and US. We offer services on a fee for service Cbasis as well as contracting early discovery projects on milestone reward basis. As a proof of our model, we bring our expertise and experience from more than 600 engagements with customers ranging from virtual Biotechs to several top 10 Pharmaceutical companies. We also promote industryacademia partnerships and are involved in many academic research projects aiming to provide novel innovation services. We are also authors in over 60 peer-reviewed research papers on drug research and technologies since 2005.

Dr Jari Rautio: CEO Plexpress t: +358 40 504 0355 e: jari.rautio@plexpress.com w: http://www.plexpress.com

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Plexpress and SBW Partner to Offer TRAC Gene Expression Analysis

Public release date: 17-May-2012 [ | E-mail | Share ]

Contact: Clea Desjardins clea.desjardins@concordia.ca 514-848-2424 x5068 Concordia University

Montreal, May 18, 2012 What do synthetic fuels, new treatments for malaria and genetic engineering have in common? In a word, biology. To examine the wide-reaching implications of this evolving discipline, Concordia University's Centre for Structural and Functional Genomics presents Building Biology: A Symposium on Synthetic Biology.

Held at Concordia's Loyola Campus on May 21, the symposium brings together the world's top researchers on the subject, including academics from Harvard, MIT, Berkeley and Johns Hopkins. Because this event is closely tied into the work of Concordia's cutting-edge genomics laboratory, the President and CEO of Genome Canada, Pierre Meulien, will give the welcoming address.

What: Building Biology: A Symposium for Synthetic Biology When: Monday, May 21, 8 a.m. to 6 p.m. Where: Concordia University, Loyola Campus, 7141 Sherbrooke Street West, SP S-110

The purpose of this symposium is to foster interaction between established synthetic biologists in Canada and North America. The event represents a cornerstone in the creation of a synthetic biology research hub for Canada while putting Concordia on the map as the prime location for research in synthetic biology.

Speakers include: Pierre Meulien (Genome Canada), Jay Keasling (University of California Berkeley), Jack Newman (Amyris Inc.), Peter Facchini (University of Calgary), Ron Weiss (MIT), Pamela Silver (Harvard University), Radhakrishnan Mahadevan (University of Toronto), Matthew Scott (University of Waterloo), Mads Kaern (University of Ottawa), Joel Bader (John Hopkins University) and Nathan Hillson (Joint BioEnergy Institute).

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This symposium is generously sponsored by Concordia University, Genome Quebec, CSFG, PhytoMetaSyn, CRIBIQ, DNA 2.0 and IDT.

Related links:

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Concordia welcomes world's best synthetic biology researchers

On my latest blog, ThinkCreeps posted a comment quoting my statement that "we do not know why natural genetic engineering systems are as successful as they have been in generating useful evolutionary novelties in the history of life." Then he goes on to answer, "Yes we do - the good ones spread quickly through the population."

If only things were that simple! Good novelties just appear, as if by magic, and then spread due to their selective advantages. ThinkCreeps apparently shares a common illusion in evolutionary thinking that natural selection is all we need in the way of basic principles to understand the evolutionary process.

All scientific views of evolution by descent with modification envision two separate and essential steps in the establishment of living organisms with novel features:

In the absence of detailed information about the mechanisms of variation, heritable differences were widely assumed to arise randomly and accidentally. After Mendelism was rediscovered at the start of the 20th century, Mendelian segregations were added as variation modes in the neo-Darwinian "Modern Synthesis." Nonetheless, the sources of new segregating alleles (genetic differences) were still assumed to be stochastic accidents.

It was even claimed by some neo-Darwinians that evolution could be ascribed to changes in allele frequencies in populations due to natural selection acting on their fitness contributions. Many thinkers did not notice that this argument neglected the large number of cases where evolutionary differences were accompanied by other kinds of heritable change, such as alterations in chromosome structure or number.

With the advent of molecular genetics and DNA sequencing in the second half of the 20th century, it became possible to study the mechanisms of genome change in detail. It is commonly assumed that genome alterations account for the vast majority of heritable variation in living organisms. Other kinds of heritable changes are known and may also play an important role in evolution. Non-DNA changes include inheritance of self-templated cell structures and protein conformations, such as prions.

The results of the molecular studies are clear. Heritable changes can occur at the genetic level, through alterations in DNA sequences and in the structures of cell DNA molecules, and at the epigenetic level, through alterations in the way DNA is modified chemically and complexed with RNA and proteins in stable chromatin configurations.

Genetic and epigenetic changes result from the actions of cell biochemical activities, not from accidents. This is a critical fundamental discovery of molecular genetics.

There are many different activities that work directly on DNA and bring about genetic changes, ranging from single nucleotide substitutions to major restructuring of chromosomes. DNA modules can move from one place to another in the genome, RNA molecules can be reverse transcribed into DNA and inserted into the genome, and broken DNA molecules can be rejoined in novel combinations. The genome sequence record provides a rapidly growing mountain of evidence showing how important such non-random events have been in evolutionary history.

There are also many distinct activities that modify DNA and chromatin structures leading to heritable epigenetic changes. Our knowledge of these chromatin remodeling processes is younger than our acquaintance with DNA changes, and they do not leave the same kind of trace in the genome sequence record. But we do know that epigenetic changes have a profound influence on genome restructuring activities, and the same ecological challenges and stresses lead to high levels of both epigenetic and genetic variability.

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James A. Shapiro: Variation and Selection: What's the Difference? What Are the Issues?

SAN DIEGO, May 18, 2012 /PRNewswire/ -- Sequenom, Inc. (SQNM), a life sciences company providing innovative genetic analysis solutions, today announced that a new publication from the large Women & Infants multi-center clinical study on the Sequenom Center for Molecular Medicine's (Sequenom CMM) MaterniT21 PLUS laboratory-developed test (LDT) has been published online in the peer-reviewed journal, Prenatal Diagnosis. Along with this week's publication, the Company announces that as of the week ended May 12, 2012, Sequenom CMM has processed more than 10,000 commercial MaterniT21 PLUS test samples in 2012.

The publication addresses the capability of the MaterniT21 PLUS LDT to accurately detect the presence of certain fetal trisomies in pregnant women carrying twins or triplets. The paper will appear in the journal's May issue and the full abstract can be found online at: http://onlinelibrary.wiley.com/doi/10.1002/pd.3892/abstract.

"The underlying biology and these positive study data provide evidence that this type of DNA-testing can be reliably employed as a clinical management option for women expecting twins or triplets who are at increased risk for fetal chromosome anomalies," said Allan Bombard, M.D., Laboratory Director for Sequenom Center for Molecular Medicine.

The published results are derived from the large international, multi-center study conducted at 27 prenatal diagnostic centers worldwide, with previous publications on trisomy 21 and trisomies 18 and 13 in Genetics in Medicine. Participating sites collected and processed maternal plasma samples from 4,664 pregnant women in the late first and early second trimester who were at increased risk for fetal aneuploidy. Blinded samples from pregnancies with trisomy 21, trisomy 18, and trisomy 13 as well as those with other abnormal karyotypes were tested.

In the same multi-center study, maternal plasma samples were tested from 25 twin and two triplet pregnancies. Of the twin pregnancies, there were no trisomies in 17 pregnancies (known as euploid), trisomy 21 in seven (two cases of trisomy 21 in both fetal twins, five cases of trisomy 21 in one fetal twin only), and trisomy 13 in one (in one fetal twin). There were two triplet pregnancies, neither of which had trisomies. The MaterniT21 PLUS technology correctly classified the eight twin pregnancies with trisomy 21 or trisomy 13, the 17 twin euploid pregnancies and both triplet euploid pregnancies.

"We know that, in the U.S., pregnant women carrying twins or higher multiples are becoming more common due to the use of assisted reproductive technologies and acknowledge that more of these women have increased risks for fetal aneuploidy, such as advanced maternal age," said Harry F. Hixson, Jr., Ph.D., Chairman and CEO, Sequenom, Inc. "This published data provides valuable evidence to specialists that Sequenom CMM's MaterniT21 PLUS LDT can provide reliable detection of certain fetal trisomies in twins, just as in single pregnancies."

The research was led by Jacob Canick, PhD, and Glenn Palomaki, PhD, of the Division of Medical Screening and Special Testing in the Department of Pathology and Laboratory Medicine at Women & Infants Hospital and The Warren Alpert Medical School of Brown University. The study also included scientists at Sequenom Center for Molecular Medicine, San Diego, CA.

As of the week ended May 12, Sequenom CMM has processed more than 10,000 MaterniT21 PLUS tests in 2012. Due to the successful rate of adoption, the Company recently announced that it has increased its internal goal to 40,000 MaterniT21 PLUS tests billed in 2012, up from the original internal goal of 25,000 tests billed for the year. As of the last week in April, the 52-week run rate had increased to more than 45,000 tests.

The MaterniT21 PLUS LDT is available solely through Sequenom CMM as a testing service to physicians. To learn more about the test, please visit 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.

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Study In Prenatal Diagnosis Finds Sequenom CMM's MaterniT21™ PLUS Lab-developed Test Accurately Detects Fetal ...

Public release date: 17-May-2012 [ | E-mail | Share ]

Contact: Jane E. Rubinstein jrubinstein@rubenstein.com 212-843-8287 IntegraGen

Toronto, CANADA (May 17, 2012) By focusing on the identification of common genetic variants, researchers have identified 57 single nucleotide polymorphisms (SNPs) that predictwith a high degree of certainty--the risk that siblings of children with Autism Spectrum Disorder (ASD) will also develop the condition. The findings were presented at the International Meeting for Autism Research.

ASD is among the most common form of severe developmental disability with prevalence rates up to 1 in 88 children. Boys are greater than four times more likely to be diagnosed with ASD, while recurrence risks for the sibling of a child with ASD are estimated at 18.7%. Since multiple studies have shown that early assessment and intervention offer significantly improved long-term outcomes, early identification of children at risk of ASD has become a key goal.

Though many recent studies demonstrate that autism has a genetic basis, the inheritance pattern of ASD in most families is highly complex. While genetic testing for autism has been limited to the identification of copy number variants (CNVs), autism-associated CNVs are found only in approximately 10% of children with ASD.

Researchers seeking an alternative approach to identify biomarkers for autism have focused on a number of common genetic variants--or SNPs --that have been shown to be related to the risk of ASD. While individual SNPs do not cause ASD, recent studies have shown that the presence of a combination of autism-associated SNPs can predict with a high degree of certainty whether a child will develop ASD.

"By looking at a combination of gender-specific, risk-associated, genetic common variants, we were able to identify siblings of children with ASD who have a significantly increased risk of developing autism," says lead author Francois Liebaert, MD, Vice President of Research and Development for IntegraGen, SA, Evry, France, "Earlier identification of siblings of children with autism at increased risk may lead to faster referrals, earlier diagnosis, earlier intervention and better prognosis. We also hope to replicate these findings in families that do not have a child with autism."

These findings build upon earlier identification of eight autism-related SNPs that occur in males and females (Autism risk assessment in siblings of affected children using sex-specific genetic scores) published the February 17, 2011 edition of Molecular Autism.

To determine which SNPs were associated with autism, researchers applied techniques that have been used to analyze other complex diseases. By combining statistical results from genome wide association studies (GWAS) with biological information from multiple sources including databases and scientific literature, the researchers were able to identify and prioritize the SNPs, and develop gender-specific genetic scores to predict the risk of autism.

The study comprised greater than 1,100 families which have more than one child diagnosed with ASD, referred to as multiplex families, including nearly 2,000 affected and 600 unaffected siblings. The male to female ratio for affected children was close to 4.2:1. The discovery cohort included 545 families from the Autism Speaks Autism Genetic Resource Exchange Repository (AGRE). The findings were then replicated in a population comprising 627 families including 339 families from a separate AGRE collection and DNA samples from 288 independent families collected at the University of Washington, Seattle and currently maintained at the University of Pennsylvania.

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Common genetic variants identify autism risk in high risk siblings of children with ASD

Public release date: 17-May-2012 [ | E-mail | Share ]

Contact: Raymond MacDougall macdougallr@mail.nih.gov 301-402-0911 NIH/National Human Genome Research Institute

People have increasing opportunities to participate in genetic testing that can indicate their range of risk for developing a disease. Receiving these results does not appreciably drive up or diminish test recipients' demand for potentially costly follow-up health services, according to a study performed by researchers at the National Institutes of Health and colleagues at other institutions.

The study in the May 17, 2012 early online issue of Genetics in Medicine was done by investigators with the Multiplex Initiative, a multi-center collaborative initiative involving investigators from the National Institutes of Health's Intramural Research Program, Group Health Cooperative in Seattle, and the Henry Ford Health System in Detroit.

The tests are available from a growing number of commercial producers, and health care providers have been uncertain whether people who received information only about risk would follow up by demanding diagnostic testing to monitor for predicted illnesses.

The study is the first to use electronic health records -- rather than self-reported behavior -- to measure the impact of genetic testing on the subsequent consumption of health services by commercially insured, healthy adults. Self reports, which can be affected by memory lapses and other problems, tend to be less accurate.

"We need to understand the impact of genomic discoveries on the health care system if these powerful technologies are going to improve human health," said Dan Kastner, M.D., Ph.D., scientific director and head of the National Human Genome Research Institute's (NHGRI) Division of Intramural Research. "We are still learning how to integrate new genomic discoveries into clinical care effectively and efficiently."

"There are a lot of unanswered questions about how genetic test results can be used to guide people towards making positive lifestyle and health behavior changes," said Colleen McBride, Ph.D., chief of NHGRI's Social and Behavioral Research Branch. "This study goes a long way towards bringing data to these debates and shows that people are not likely to make inappropriate demands of health delivery systems if they are properly informed about the limitations of genetic tests."

Genetic tests, such as those used in this study, can detect common variants of genes associated with modest alterations in the chances of developing particular diseases. The term multiplex refers to simultaneously performing multiple genetic tests on a single blood sample.

The study included 217 healthy people between the ages of 25 and 40 who elected to participate in genetic susceptibility testing offered by their health plan. The researchers analyzed health care usage by the participants in the 12 months before genetic testing and the 12 months following the testing. They also compared the test group's behavior with a group of about 400 similar plan members who declined the testing offer.

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NIH-led study finds genetic test results do not trigger increased use of health services

Medical experts feared personal genetic test results might drive overuse of expensive medical care

The study in the May 17, 2012 early online issue of Genetics in Medicine was done by investigators with the Multiplex Initiative, a multi-center collaborative initiative involving investigators from the National Institutes of Health's Intramural Research Program, Group Health Cooperative in Seattle, and the Henry Ford Health System in Detroit.

The tests are available from a growing number of commercial producers, and health care providers have been uncertain whether people who received information only about risk would follow up by demanding diagnostic testing to monitor for predicted illnesses.

The study is the first to use electronic health records -- rather than self-reported behavior -- to measure the impact of genetic testing on the subsequent consumption of health services by commercially insured, healthy adults. Self reports, which can be affected by memory lapses and other problems, tend to be less accurate.

"We need to understand the impact of genomic discoveries on the health care system if these powerful technologies are going to improve human health," said Dan Kastner, M.D., Ph.D., scientific director and head of the National Human Genome Research Institute's (NHGRI) Division of Intramural Research. "We are still learning how to integrate new genomic discoveries into clinical care effectively and efficiently."

"There are a lot of unanswered questions about how genetic test results can be used to guide people towards making positive lifestyle and health behavior changes," said Colleen McBride, Ph.D., chief of NHGRI's Social and Behavioral Research Branch. "This study goes a long way towards bringing data to these debates and shows that people are not likely to make inappropriate demands of health delivery systems if they are properly informed about the limitations of genetic tests."

Genetic tests, such as those used in this study, can detect common variants of genes associated with modest alterations in the chances of developing particular diseases. The term multiplex refers to simultaneously performing multiple genetic tests on a single blood sample.

The study included 217 healthy people between the ages of 25 and 40 who elected to participate in genetic susceptibility testing offered by their health plan. The researchers analyzed health care usage by the participants in the 12 months before genetic testing and the 12 months following the testing. They also compared the test group's behavior with a group of about 400 similar plan members who declined the testing offer.

The researchers counted the number of physician visits and laboratory tests or procedures the people received, particularly those services associated with four of the eight conditions tested by the multiplex panel. Most of the procedures or screening tests that were counted are not among those currently recommended for people in this age group who don't have symptoms. The researchers found that participants in genetic testing did not change their overall use of health care services compared with those not tested.

All of the individuals who elected to undergo the multiplex test carried at least one at-risk genetic marker, with the majority carrying an average of nine at-risk variants. The tests performed for the Multiplex Initiative include a set of genetic variants reliably associated with an increase in disease risk and for which some corrective health behavior has been shown to prevent illness.

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Genetic Test Does Not Trigger Increased Use of Health Services

May 18, 2012

Connie K. Ho for RedOrbit.com

Recently, genetic tests have been on the rise and are offered by more and more commercial producers. While there are many opportunities for people to participate in genetic testing, this doesnt necessarily mean that people are following up on those services. These are the results found in a study that is published in the May 17 issue of Genetics in Medicine.

The study was a collaborative project by the Multiplex Initiative, which includes researchers from the National Institutes of Healths Intramural Research Program, the Group Health Cooperative, and the Henry Ford Health System. Researchers analyzed variants of genes related to diseases such as colorectal cancer, coronary heart disease, high blood cholesterol, hypertension, lung cancer, melanoma, osteoporosis, and type 2 diabetes. The study allowed researchers to better understand patients health care needs.

Our study was a best-case scenario, because we chose 15 genes reliably associated with relatively small risks for eight common diseases that health behaviors can affect, remarked lead author Dr. Robert J. Reid in a prepared statement. We hope that testing positive activates patients to make behavior changes that could lower their risk, such as quitting smoking without causing them to make many extra visits to their doctors.

The report was one of the first studies that looked at electronic health records, as opposed to self-reported behavior, to quantify the impact of genetic testing on health services chosen by adults.

We need to understand the impact of genomic discoveries on the health care system if these powerful technologies are going to improve human health, explained Dr. Dan Kastner, scientific director and head of the National Human Genome Research Institutes (NHGRI) Division of Intramural Research, in a statement. We are still learning how to integrate new genomic discoveries into clinical care effectively and efficiently.

Genetic tests are also important because they can find variants of genes related to the changes in the risk of developing a particular disease.

Understanding personalized genetic information is important because it is becoming more readily available and we need to figure out how to integrate it effectively and efficiently into the clinical care we provide, noted coauthor Dr. Eric B. Larson in a prepared statement.

The scientists hope to continue to research consumer interest of genetic testing and how that affects patients long-term health care goals.

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Few Use Follow Up Services After Genetic Testing

Public release date: 17-May-2012 [ | E-mail | Share ]

Contact: Rebecca Hughes hughes.r@ghc.org 206-287-2055 Group Health Research Institute

SEATTLEPeople have more and more chances to participate in genetic testing that can indicate their range of risk for developing a disease. Receiving these results does not appreciably drive up or diminishtest recipients' demand for potentially costly follow-up health services, according to a new study in the May 17, 2012 early online issue of Genetics in Medicine.

The study was done by researchers with the Multiplex Initiative, a multi-center collaborative initiative involving investigators from the National Institutes of Health's Intramural Research Program, Group Health Cooperative in Seattle, and the Henry Ford Health System in Detroit.

The tests are available from a growing number of commercial producers, and health care providers have been uncertain whether people who received information only about risk would follow up by demanding diagnostic testing to check for predicted illnesses.

The study is the first to use electronic health recordsrather than self-reported behaviorto measure the impact of genetic testing on the subsequent use of health services by commercially insured, healthy adults. Self-reports, which can be affected by memory lapses and other problems, tend to be less accurate.

"Our study was a best-case scenario, because we chose 15 genes reliably associated with relatively small risks for eight common diseases that health behaviors can affect," said the study's first author Robert J. Reid, MD, PhD. Dr. Reid is Group Health's associate medical director of research translation and an associate investigator at Group Health Research Institute. Those diseases were type 2 diabetes, coronary heart disease, high blood cholesterol, hypertension, osteoporosis, lung cancer, colorectal cancer, and melanoma. "We hope that testing positive activates patients to make behavior changes that could lower their risk, such as quitting smoking," he added, "without causing them to make many extra visits to their doctors."

"Understanding personalized genetic information is important because it is becoming more readily available and we need to figure out how to integrate it effectively and efficiently into the clinical care we provide," said coauthor Eric B. Larson, MD, MPH, Group Health Cooperative's vice president for research and Group Health Research Institute's executive director.

"There are a lot of unanswered questions about how genetic test results can be used to guide people toward making positive lifestyle and health behavior changes," said Colleen McBride, PhD, chief of the Social and Behavioral Research Branch at the National Human Genome Research Institute (NHGRI). "This study goes a long way toward bringing data to these debates and shows that people are not likely to make inappropriate demands of health delivery systems if they are properly informed about the limitations of genetic tests."

Genetic tests, such as those used in this study, can detect common variants of genes associated with modest changes in the chances of developing particular diseases. "Multiplex" means simultaneously performing many genetic tests on one blood sample.

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Genetic testing may not trigger more use of health services

NEW YORK--(BUSINESS WIRE)--

Pfizer Inc announced today that the Phase 3 INTORSECT (B1771003) study, evaluating TORISEL (temsirolimus) in patients with advanced renal cell carcinoma (RCC) whose disease had progressed on or after SUTENT (sunitinib malate) therapy, did not meet the primary endpoint of prolonging progression free survival (PFS) when compared to sorafenib. Although PFS was numerically higher in patients treated with temsirolimus, the difference was not statistically significant. Overall survival, a secondary endpoint in the study, showed statistical significance favoring patients randomized to the sorafenib arm. Adverse events in this study were consistent with the known safety profiles for both drugs. Full efficacy and safety data from this study will be presented at an upcoming major medical congress.

Approximately 270,000 people worldwide are diagnosed with renal cell cancer every year with about 20 percent having advanced disease at the time of diagnosis.1 Between 40 and 65 percent of patients in the U.S. who progress following first-line therapy go on to receive a second-line treatment.2,3,4

This trial advances our knowledge about TORISEL in RCC. TORISEL remains an important drug for treatment of advanced kidney cancer based on its pivotal study in first-line patients with poor prognostic risk, said Dr. Mace Rothenberg, senior vice president of clinical development and medical affairs for Pfizers Oncology Business Unit. TORISEL continues to be an important part of Pfizers portfolio of therapies for advanced kidney cancer.

About TORISEL (temsirolimus)

TORISEL is approved in the US and other countries for the treatment of advanced RCC. TORISEL is approved in the European Union for the first-line treatment of patients with advanced renal cell carcinoma (RCC) who have at least three of six prognostic risk factors. In a pivotal Phase 3 study, TORISEL demonstrated median overall survival (OS) in previously untreated patients of 10.9 months in patients with advanced RCC with poor prognostic risk, compared with 7.3 months for interferon-alpha (IFN-).

TORISEL is the only intravenous mammalian target of rapamycin (mTOR) inhibitor approved for the treatment of advanced RCC. TORISEL remains the only treatment to show a significant improvement in OS in treatment-nave poor risk patients with advanced RCC.5

Based on preclinical studies, TORISEL inhibits the activity of mTOR, an intracellular protein implicated in multiple growth-related cellular functions including proliferation, growth and survival. The inhibition of mTOR also reduces levels of certain growth factors, such as vascular endothelial growth factor (VEGF), which are overexpressed in solid tumors like kidney cancer and are thought to play a crucial role in angiogenesis, the process by which tumors acquire blood vessels, nutrients and oxygen needed for growth.

Important TORISEL (temsirolimus) Safety Information

TORISEL is contraindicated in patients with bilirubin >1.5 x ULN and should be used with caution when treating patients with mild hepatic impairment (bilirubin >1 1.5 x ULN or AST > ULN but bilirubin ULN). If TORISEL must be given to patients with mild hepatic impairment, reduce the dose of TORISEL to 15 mg/week. In a phase 1 study, the overall frequency of grade 3 adverse reactions and deaths, including deaths due to progressive disease, was greater in patients with baseline bilirubin > 1.5 x ULN.

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Pfizer Provides Topline Results From Phase 3 Study Of Torisel® As Second-Line Treatment In Advanced Renal Cell ...

LEUVEN, BELGIUM--(Marketwire -05/15/12)- TiGenix NV (TIG) a leader in the field of cell therapy, today gave a business update and announced the financial results for the first quarter ending March 31, 2012.

Business highlights

Financial highlights

"In the first quarter 2012 we continued to aggressively push our commercial efforts forward," said Eduardo Bravo, CEO of TiGenix. "As a result sales of ChondroCelect are developing in line with the improved traction we observed in the second part of last year. At the same time we are moving ahead of schedule with most of our clinical adipose stem cell programs. We closed the quarter with almost EUR 17 million cash on hand, which is sufficient to execute on our business plan and reach key inflection points."

Business update

ChondroCelect sales increase continues apaceThe Company reports net sales growth for the quarter of 123% compared with the same period of last year, and of 62% compared to Q4, 2011, a positive trend reflecting the uptake in Belgium, where we benefit from national reimbursement. In the Netherlands one of the leading private healthcare insurance companies has made treatment with ChondroCelect compulsory for its insured, and no longer reimburses non-ATMP treatments. Similarly, one of the large private insurers in the UK has expressed its intention to routinely reimburse ChondroCelect going forward. Discussions to obtain full national reimbursement keep advancing in the Netherlands, France, Spain and Germany.

Positive outcome of ChondroCelect compassionate use program published in leading journalPositive outcome data from the ChondroCelect compassionate use program (CUP), involving 43 orthopedic centers in 7 European countries, treating 370 patients with ChondroCelect over the span of four years, were published in advance online in Cartilage, the official journal of the International Cartilage Repair Society. The data show that the implantation of ChondroCelect results in a positive benefit/risk ratio when used in an unselected, heterogeneous population, irrespective of the follow-up period, lesion size and type of lesion treated. In addition, the CUP study significantly expands the data set used to obtain approval for ChondroCelect from the European Medicines Agency in 2009, increasing eight-fold, from 43 to 334, the number of patients with long-term follow up data. To date almost 700 patients have been treated with ChondroCelect.

ADMIRE-CD Phase III trial (Cx601) in complex perianal fistula on schedule The ADMIRE-CD (Adipose Derived Mesenchymal stem cells for Induction of REmission in perianal fistulizing Crohn's Disease) Phase III protocol was submitted to Ethics Committees or Health Authorities in all 8 participating countries, and to date approvals have been received in four of those countries already.

Cx611 Phase IIa in RA passes last safety hurdleOn April 17, upon review of the safety data of the first three patients of the third cohort of the company's Phase IIa clinical trial in rheumatoid arthritis (Cx611), TiGenix received the go-ahead from the independent Safety Monitoring Board to recruit and dose the remaining patients of this cohort. This fact is of major importance. In RA it ensures that the product will not be held back by any dose-limiting factors and that we will be able to move forward with the optimal treatment dose. Of almost equal importance is that, if required, we can expand the dosing range in other indications that we are exploring as well. With 6 months of follow-up, the current RA trial in 53 patients is expected to report meaningful results in H1 2013.

Last patient treated in Cx621 Phase I clinical trialAll 10 healthy volunteers have been recruited and treated in the Phase I study of Cx621. Cx621 investigates the safety and feasibility of intra-lymphatic administration of stem cells. Intra-lymphatic administration of (all) stem cells is patented by TiGenix. The final report of this trial will be available at the end of June.

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TiGenix Reports Business & Financial Results for the First Quarter 2012

JERUSALEM--(BUSINESS WIRE)--

Gamida Cell announced today that it has closed an internal E financing round of $10 million. All major shareholders participated.

The financing will be used to support the global commercialization of the companys lead cell therapy product, StemEx, in development as an alternative therapeutic treatment for patients with blood cancers, such as leukemia and lymphoma, who can be cured by bone marrow transplantation but do not have a matched bone marrow donor. The company is currently seeking a strategic partner to join in the global commercialization of StemEx.

The financing will also support the continued development of the companys pipeline of products, primarily the NiCord clinical trial for sickle cell disease and thalassemia.

Mr. Reuven Krupik, chairman of the board of Gamida Cell said, The investors were unanimous in their decision to reinvest, understanding the importance of bringing StemEx to market as well as maintaining the companys leadership role in the stem cell industry. Gamida Cell is a game changer.

The international, multi-center, pivotal registration, Phase III clinical trial of StemEx completed enrollment in February 2012. Clinical outcome is expected in Q4/2012. The market launch of StemEx is planned for 2013. StemEx is likely to be the first allogeneic stem cell product in the market. StemEx is being developed by the Gamida Cell-TEVA joint venture.

Dr. Yael Margolin, president and chief executive officer of Gamida Cell said, With the continued support of our shareholders and the analysis of the clinical results of the StemEx trial just around the corner, we are now focused on submitting the BLA.

StemEx is a graft of an expanded population of stem/progenitor cells, derived from part of a single unit of umbilical cord blood and transplanted by IV administration along with the remaining, non-manipulated cells from the same unit. Competing products in development use two units. As the average cost of a cord blood unit in the U.S. is $40K, StemEx is expected to be a significantly less expensive treatment option. StemEx is also expected to be available in the market several years before any of the competing products.

About Gamida Cell

Gamida Cell is a world leader in stem cell population expansion technologies and stem cell therapy products for transplantation and regenerative medicine. The companys pipeline of stem cell therapy products are in development to treat a wide range of conditions including blood cancers, solid tumors, non-malignant hematological diseases such as hemoglobinopathies, neutropenia and acute radiation syndrome, autoimmune diseases and metabolic diseases as well as conditions that can be helped by regenerative medicine. Gamida Cells therapeutic candidates contain populations of adult stem cells, selected from non-controversial sources such as umbilical cord blood, bone marrow and peripheral blood, which are expanded in culture. Gamida Cells current shareholders include: Elbit Imaging, Clal Biotechnology Industries, Israel Healthcare Venture, Teva Pharmaceutical Industries, Amgen, Denali Ventures and Auriga Ventures. For more information, please visit: http://www.gamida-cell.com.

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Gamida Cell Closes $10 Million E Financing Round Earmarked to Support the Global Commercialization of the Company’s ...

Barbara Netter, co-founder of the Alliance for Cancer Gene Therapy, receives the first-ever ACGT Partnership Award from Savio Woo, chairman of the Alliance's scientific council and professor and founding chair of the Department of Gene and Cell Medicine at Mt. Sinai School of Medicine.

Photo credit: Courtesy of Elaine Ubia

GREENWICH, Conn. The Greenwich-based Alliance for Cancer Gene Therapy celebrated its 10th anniversary of funding cancer gene therapy research with a gala benefit last month.

Thirty of the nations top cancer research scientists and more than 450 Alliance supporters gathered on April 19 to mark the progress made in the field. The event raised more than $1.4 million, all of which will go directly to funding new grants and clinical trails.

The alliance also honored the life of its visionary co-founder, Edward Netter, who died last year, by presenting the first ever ACGT Partnership award to Netter's wife, Barbara, who co-founded the alliance with her husband.

Ed epitomized the innovative thinker and problem solver, and he felt it important to examine the status quo, Barbara Netter said in a prepared statement. He had the perseverance to pursue a better way. Our progress, our recent breakthrough, is proving to be a fitting tribute and legacy to Ed, who worked tirelessly and passionately to this end. Ed Netter was a key figure in accelerating research and bringing us to this point.

Prior to the evenings festivities many of the nations leading cancer researchers held a scientific symposium, where they discussed the alliances progress as a leader in the field, as well as how to bridge the gap between the lab and bringing science to patients.

Robert Bazell, the Emmy award-winning chief science and health correspondent for NBC News, was the keynote speaker. Dr. Dennis Clegg, professor, Department of Molecular, Cellular and Developmental Biology, University of California, Santa Barbara, California, was the featured scientist at the dinner.

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Greenwich Cancer Research Group Raises $1.4M

Public release date: 14-May-2012 [ | E-mail | Share ]

Contact: Juan J. Gomez juanj.gomez@cnio.es 34-917-328-000-4060 Centro Nacional de Investigaciones Oncologicas (CNIO)

A number of studies have shown that it is possible to lengthen the average life of individuals of many species, including mammals, by acting on specific genes. To date, however, this has meant altering the animals' genes permanently from the embryonic stage an approach impracticable in humans. Researchers at the Spanish National Cancer Research Centre (CNIO), led by its director Mara Blasco, have proved that mouse lifespan can be extended by the application in adult life of a single treatment acting directly on the animal's genes. And they have done so using gene therapy, a strategy never before employed to combat ageing. The therapy has been found to be safe and effective in mice.

The results are published today in the journal EMBO Molecular Medicine. The CNIO team, in collaboration with Eduard Ayuso and Ftima Bosch of the Centre of Animal Biotechnology and Gene Therapy at the Universitat Autnoma de Barcelona (UAB), treated adult (one-year-old) and aged (two-year-old) mice, with the gene therapy delivering a "rejuvenating" effect in both cases, according to the authors.

Mice treated at the age of one lived longer by 24% on average, and those treated at the age of two, by 13%. The therapy, furthermore, produced an appreciable improvement in the animals' health, delaying the onset of age-related diseases like osteoporosis and insulin resistance and achieving improved readings on ageing indicators like neuromuscular coordination.

The gene therapy utilised consisted of treating the animals with a DNA-modified virus, the viral genes having been replaced by those of the telomerase enzyme, with a key role in ageing. Telomerase repairs the extremes of chromosomes, known as telomeres, and in doing so slows the cell's and therefore the body's biological clock. When the animal is infected, the virus acts as a vehicle depositing the telomerase gene in the cells.

This study "shows that it is possible to develop a telomerase-based anti-ageing gene therapy without increasing the incidence of cancer", the authors affirm. "Aged organisms accumulate damage in their DNA due to telomere shortening, [this study] finds that a gene therapy based on telomerase production can repair or delay this kind of damage", they add.

'Resetting' the biological clock

Telomeres are the caps that protect the end of chromosomes, but they cannot do so indefinitely: each time the cell divides the telomeres get shorter, until they are so short that they lose all functionality. The cell, as a result, stops dividing and ages or dies. Telomerase gets round this by preventing telomeres from shortening or even rebuilding them. What it does, in essence, is stop or reset the cell's biological clock.

But in most cells the telomerase gene is only active before birth; the cells of an adult organism, with few exceptions, have no telomerase. The exceptions in question are adult stem cells and cancer cells, which divide limitlessly and are therefore immortal in fact several studies have shown that telomerase expression is the key to the immortality of tumour cells.

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CNIO scientists successfully test the first gene therapy against aging-associated decline

ScienceDaily (May 15, 2012) A novel mechanism for anxiety behaviors, including a previously unrecognized inhibitory brain signal, may inspire new strategies for treating psychiatric disorders, University of Chicago researchers report.

By testing the controversial role of a gene called Glo1 in anxiety, scientists uncovered a new inhibitory factor in the brain: the metabolic by-product methylglyoxal. The system offers a tantalizing new target for drugs designed to treat conditions such as anxiety disorder, epilepsy, and sleep disorders.

The study, published in the Journal of Clinical Investigation, found that animals with multiple copies of the Glo1 gene were more likely to exhibit anxiety-like behavior in laboratory tests. Further experiments showed that Glo1 increased anxiety-like behavior by lowering levels of methylglyoxal (MG). Conversely, inhibiting Glo1 or raising MG levels reduced anxiety behaviors.

"Animals transgenic for Glo1 had different levels of anxiety-like behavior, and more copies made them more anxious," said Abraham Palmer, PhD, assistant professor of human genetics at the University of Chicago Medicine and senior author of the study. "We showed that Glo1 was causally related to anxiety-like behavior, rather than merely correlated."

In 2005, a comparison of different mouse strains found a link between anxiety-like behaviors and Glo1, the gene encoding the metabolic enzyme glyoxylase 1. However, subsequent studies questioned the link, and the lack of an obvious connection between glyoxylase 1 and brain function or behavior made some scientists skeptical.

"When people discover a gene, they're always most comfortable when they discover something they already knew," Palmer said. "The alarming thing here was there was a discovery of something that nobody knew, and therefore it seemed less likely to actually be correct."

A 2009 study from Palmer's laboratory suggested that differences in Glo1 expression between mouse strains were due to copy number variants, where the segment of the genome containing the gene is repeated multiple times. To test this hypothesis, lead author Margaret Distler inserted two, eight or ten copies of the Glo1 gene into mouse lines. She then ran experiments such as the open field test, in which researchers measure how much time a mouse spends in the center of an arena versus along the walls, to detect changes in anxiety behavior.

The results confirmed a causative role for Glo1 copy number variants, as mice with more copies of the Glo1 gene exhibited higher anxiety-like behavior in their experiments.

"It's the first study to show that it's the copy number variant that has the potential to change Glo1 expression and behavior," said Distler, an MD/PhD student in the Pritzker School of Medicine's Medical Scientist Training Program. "Our study was a physiological representation of what it means to increase Glo1 expression for anxiety."

The researchers then set about answering the mystery of how Glo1 expression influences anxiety-like behaviors. The primary function of glyoxylase 1 is to metabolize and lower cellular levels of methylglyoxal, a waste product of glycolysis. Distler produced the opposite effect by injecting MG to artificially increase its levels in the brain, finding that raising MG levels quickly reduced anxiety symptoms in mice.

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Mystery gene reveals new mechanism for anxiety disorders

Diane Cook's 65th birthday was a milestone for her, but not because of her age. That was the day she was diagnosed with Parkinson's disease.

"I was stunned," she said, adding that even though she had had symptoms for four years before being diagnosed, the news was still a surprise.

That day, she could not muster the courage to learn more about what was in store for her. But the next day she pored over the Internet to learn about the disease. She quickly discovered that it would continue to get worse, and that there was no cure.

Parkinson's disease, often associated with boxer Muhammad Ali and actor Michael J. Fox, affects 1 million Americans, according to the National Parkinson Foundation.

While the exact causes largely remain a mystery, doctors know that the condition arises from the degeneration of a specific area of the brain involved in movement. As a result, those with Parkinson's experience tremors, rigidity, slowness in moving, and difficulty with balancing and walking.

Parkinson's eventually leads to mood disorders and dementia. The complications associated with the condition are the 14th largest cause of death in the United States, according to the U.S. Centers for Disease Control and Prevention.

Not only is there no cure for Parkinson's, but many patients have no way of knowing how quickly their symptoms will progress.

"We all worry about how rapidly we'll lose our abilities," Cook said. "The uncertainty is very frustrating."

A new study from UCLA may help. Researchers have found two variants on a gene already known to be associated with Parkinson's that may be able to predict how quickly patients with the condition will deteriorate. The study found that patients with one particular variant were four times as likely to have rapid decline of motor function. Those patients having both of the variants studied were even more likely to see their disease progress more quickly.

The information is important, as patients who have more severe motor disease tend to die sooner.

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Gene Predicts Parkinson's Progression