Archive for the ‘Gene Therapy Research’ Category

London, November 1 (ANI): A team led by University of Colorado Boulder researchers has found that genetic similarities may help to explain why human birds of a feather flock together, but the full story of why people become friends "is contingent upon the social environment in which individuals interact with one another."

People are more likely to befriend genetically similar people when their environment is stratified, when disparate groups are discouraged from interacting, the study found.

When environments were more egalitarian, friends were less likely to share certain genes.

Scientists debate the extent to which genetics or environmental factors-"nature" or "nurture"-predict certain behaviours, said Jason Boardman, associate professor of sociology and faculty research associate with the Population Program in CU-Boulder's Institute of Behavioral Science.

"For all the social demographic outcomes we care about, whether it's fertility, marriage, migration, health, it's never nature or nurture. It's always nature and nurture. And most of the time it has a lot more to do with nurture," he said.

Boardman's team included Benjamin Domingue, research associate in the Population Program at IBS; and Jason Fletcher, associate professor of health policy at the Yale School of Public Health.

Early last year, PNAS published a study reporting evidence that certain shared genes might determine peoples' choice of friends. Time magazine dubbed this "friends with (genetic) benefits."

Boardman is a sociologist who spent five years studying genetics at CU-Boulder's Institute for Behavioral Genetics to bring insights of the social sciences to the natural sciences.

The research team used data from the National Longitudinal Study of Adolescent Health. Boardman's team focused on 1,503 pairs of friends in seventh through 12th grade in 41 schools. As with the earlier study, Boardman's group found that some pairs of friends shared certain genetic characteristics.

The team tested the evidence, arguing that if genes were the driving friendship factor, genetically based friendship should emerge most often and easily in schools with the least amount of social friction.

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Both genetics and environment influence peoples' choice of friends

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

Seattle Genetics, Inc. (SGEN) today announced that it has expanded its antibody-drug conjugate (ADC) collaboration with Abbott (ABT). Under the expanded deal, Abbott will pay an upfront fee of $25 million for rights to utilize Seattle Genetics auristatin-based ADC technology with antibodies to additional oncology targets. In addition, Seattle Genetics may receive up to $220 million in potential milestone payments per additional target upon achieving predetermined development and commercial objectives, as well as mid-to-high single-digit royalties on worldwide net sales of any resulting products under the multi-target collaboration.

ADCs have emerged as an important therapeutic approach to cancer, driven by the FDA approval of ADCETRIS, and encouraging data from numerous clinical and preclinical ADC programs in development by Seattle Genetics and our collaborators, said Natasha Hernday, Vice President, Corporate Development at Seattle Genetics. We are leading the field in ADC development, and this expanded collaboration with Abbott further validates our technology and approach in targeting and treating cancer.

Seattle Genetics and Abbott originally entered into an ADC collaboration in March 2011 under which Abbott paid an upfront fee of $8 million for rights to utilize Seattle Genetics ADC technology with antibodies to a single oncology target. Abbott is responsible for research, product development, manufacturing and commercialization of any ADC products under the expanded collaboration. In addition to the upfront payment and potential milestone payments and royalties, Seattle Genetics will receive annual maintenance fees and research support payments for assistance provided to Abbott under the collaboration.

ADCs are monoclonal antibodies that are designed to selectively deliver cytotoxic agents to tumor cells. With over a decade of experience and knowledge in ADC innovation, Seattle Genetics has developed proprietary technology employing synthetic cytotoxic agents, such as monomethyl auristatin E (MMAE) and monomethyl auristatin F (MMAF), and stable linker systems that attach these cytotoxic agents to the antibody. Seattle Genetics linker systems are designed to be stable in the bloodstream and release the potent cell-killing agent once inside targeted cancer cells. This approach is intended to spare non-targeted cells and thus reduce many of the toxic effects of traditional chemotherapy while enhancing antitumor activity. ADCETRIS (brentuximab vedotin) is the first drug approved utilizing Seattle Genetics ADC technology.

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 U.S. Food and Drug Administration granted accelerated approval of ADCETRIS in August 2011 for two indications. ADCETRIS is being developed in collaboration with Millennium: The Takeda Oncology Company. In addition, Seattle Genetics has three other clinical-stage ADC programs: SGN-75, ASG-5ME and ASG-22ME. 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. Across Seattle Genetics internal and collaborator programs, there are more than 15 ADCs in clinical development utilizing the companys technology. More information can be found at http://www.seattlegenetics.com.

Certain of the statements made in this press release are forward looking, such as those, among others, relating to the therapeutic potential and future clinical progress, regulatory approval and commercial launch of products utilizing Seattle Genetics ADC technology. Actual results or developments may differ materially from those projected or implied in these forward-looking statements. Factors that may cause such a difference include risks related to adverse clinical results as our product candidates or our collaborators product candidates move into and advance in clinical trials, risks inherent in early stage development and failure by Seattle Genetics to secure or maintain relationships with collaborators. More information about the risks and uncertainties faced by Seattle Genetics is contained in the Companys quarterly report on Form 10-Q for the quarter ended June 30, 2012 filed with the Securities and Exchange Commission. Seattle Genetics disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

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Seattle Genetics Expands Antibody-Drug Conjugate Collaboration with Abbott

CAMBRIDGE, Mass. & BOTHELL, Wash.--(BUSINESS WIRE)--

Millennium: The Takeda Oncology Company, a wholly owned subsidiary of Takeda Pharmaceutical Company Limited (TSE:4502), and Seattle Genetics, Inc. (SGEN) today announced the initiation of an international phase 3 clinical trial evaluating ADCETRIS (brentuximab vedotin) as part of a frontline combination chemotherapy regimen in patients with previously untreated advanced Hodgkin lymphoma (HL). The trial is being conducted under a Special Protocol Assessment (SPA) agreement from the U.S. Food and Drug Administration (FDA) and the trial also received scientific advice from the European Medicines Agency (EMA). ADCETRIS is an antibody-drug conjugate (ADC) directed to CD30, a defining marker of classical HL.

Millennium is pleased to announce the initiation of the phase 3 trial of ADCETRIS in patients with previously untreated advanced Hodgkin lymphoma. This is a key step in our efforts to explore the potential of this targeted therapy as part of a frontline treatment regimen, said Karen Ferrante, MD, Chief Medical Officer, Millennium.The trial is part of our ongoing development program to explore patient populations that may benefit from treatment with ADCETRIS in earlier lines of therapy and in other CD30-expressing malignancies.

There have been no new therapies approved for patients with newly diagnosed HL in many decades, representing a significant need to identify additional treatment options in this setting, said Thomas C. Reynolds, M.D., Ph.D., Chief Medical Officer, Seattle Genetics. We believe through this novel ADCETRIS-containing regimen we have the potential to redefine the treatment of frontline HL. This trial is also an important part of our development plan for ADCETRIS, and may serve as confirmatory to our U.S. accelerated approval in relapsed HL and systemic anaplastic large cell lymphoma.

Study Design

The randomized, open-label, phase 3 trial will investigate ADCETRIS+AVD1 versus ABVD2 as frontline therapy in patients with advanced classical HL. The primary endpoint is modified progression free survival (mPFS) per independent review facility assessment using the Revised Response Criteria for malignant lymphoma. Secondary endpoints include overall survival (OS), complete remission (CR) and safety. The multi-center trial will be conducted in North America, Europe, Latin America and Asia. The study will enroll approximately 1,040 eligible patients (approximately 520 patients per treatment arm) who have histologically-confirmed diagnosis of Stage III or IV classical HL who have not been previously treated with systemic chemotherapy or radiotherapy.

For more information, please visit http://www.clinicaltrials.gov.

About ADCETRIS

ADCETRIS (brentuximab vedotin) is an ADC comprising an anti-CD30 monoclonal antibody attached by a protease-cleavable linker to a microtubule disrupting agent, monomethyl auristatin E (MMAE), utilizing Seattle Genetics proprietary technology. The ADC employs a linker system that is designed to be stable in the bloodstream but to release MMAE upon internalization into CD30-expressing tumor cells.

ADCETRIS received accelerated approval from the U.S. Food and Drug Administration (FDA) in August 2011 for two indications: (1) the treatment of patients with Hodgkin lymphoma after failure of autologous stem cell transplant (ASCT) or after failure of at least two prior multi-agent chemotherapy regimens in patients who are not ASCT candidates, and (2) the treatment of patients with systemic anaplastic large cell lymphoma (sALCL) after failure of at least one prior multi-agent chemotherapy regimen. The indications for ADCETRIS are based on response rate. There are no data available demonstrating improvement in patient-reported outcomes or survival with ADCETRIS.

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Millennium and Seattle Genetics Initiate Global Phase 3 Clinical Trial of ADCETRIS® in Previously Untreated Advanced ...

AMES, IA--(Marketwire - Oct 31, 2012) - NewLink Genetics Corporation ( NASDAQ : NLNK ), a biopharmaceutical company focused on discovering, developing and commercializing cancer therapeutics, today reported consolidated financial results for the third quarter of 2012. NewLink updated the status of its clinical development programs including its HyperAcute Pancreas Immunotherapy Phase 3 trial (Immunotherapy for Pancreatic Resectable cancer Survival Study or "IMPRESS").

"As we approach the trigger event for the first interim analysis in our IMPRESS pivotal study, we have continued to execute our plan within the financial guidance we provided at the beginning of this year," commented Dr. Charles Link, Chairman and Chief Executive Officer of NewLink.

"In addition to our IMPRESS pivotal study for resected pancreatic patients, we have initiated a separate Phase 3 study for patients with locally advanced disease," said Dr. Nicholas Vahanian, NewLink's President and Chief Medical Officer. "If approved this new indication potentially doubles the number of patients who could be treated with algenpantucel-L. We are also expanding our HyperAcute immunotherapy platform by opening a major Phase 2B/3 study in non-small cell lung cancer."

The third quarter 2012 Financial Results

Financial Guidance

NewLink expects to end 2012 with $20 million to $23 million in cash, cash equivalents and marketable securities.

Significant recent events:

Upcoming Activities

NewLink expects to present at the following investor conferences:

About NewLink Genetics Corporation

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NewLink Genetics Corporation Reports Third Quarter 2012 Financial Results

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

Seattle Genetics, Inc. (SGEN) today announced that its collaborator, Millennium: The Takeda Oncology Company, a wholly owned subsidiary of Takeda Pharmaceutical Company Limited, announced that the European Commission has granted conditional marketing authorization for ADCETRIS (brentuximab vedotin). ADCETRIS was approved for two indications: (1) the treatment of adult patients with relapsed or refractory CD30-positive Hodgkin lymphoma (HL) following autologous stem cell transplant (ASCT) or following at least two prior therapies when ASCT or multi-agent chemotherapy is not a treatment option, and (2) for the treatment of adult patients with relapsed or refractory systemic anaplastic large cell lymphoma (sALCL). As a result, under the collaboration Seattle Genetics will receive two milestone payments from Millennium, one for each indication, totaling $25 million. ADCETRIS is an antibody-drug conjugate (ADC) directed to CD30.

The approval of ADCETRIS by the European Commission marks a significant milestone for the product and for the many relapsed or refractory HL and systemic ALCL patients in need of effective new treatment options in Europe, said Clay B. Siegall, Ph.D., President and Chief Executive Officer of Seattle Genetics. In addition to the U.S. and EU approvals of ADCETRIS, we are making regulatory progress for approval in Canada while Millennium and Takeda are pursuing regulatory approvals in other countries. Complementing these regulatory activities is a robust ADCETRIS clinical development program to support our goal of establishing it as the foundation of therapy for CD30-positive malignancies.

The conditional marketing authorization for ADCETRIS is valid in the 27 member states of the European Union (EU) as well as Norway, Liechtenstein and Iceland. Similar to accelerated approval regulations inthe United States, conditional marketing authorizations are granted in the EU to medicinal products that fulfill an unmet medical need with a positive benefit/risk assessmentand whose immediate availability would result in a significant public health benefit. Conditional marketing authorization by the European Commission includes obligations to provide additional clinical data at a later stage to confirm the positive benefit-risk assessment. The ADCETRIS Marketing Authorization Application was filed by Takeda Global Research & Development Centre (Europe) to the European Medicines Agency.

About ADCETRIS

ADCETRIS (brentuximab vedotin) is an ADC comprising an anti-CD30 monoclonal antibody attached by a protease-cleavable linker to a microtubule disrupting agent, monomethyl auristatin E (MMAE), utilizing Seattle Genetics proprietary technology. The ADC employs a linker system that is designed to be stable in the bloodstream but to release MMAE upon internalization into CD30-expressing tumor cells.

ADCETRIS received accelerated approval from the U.S. Food and Drug Administration (FDA) in August 2011 for relapsed HL and sALCL.

Seattle Genetics and Millennium are jointly developing ADCETRIS. Under the terms of the collaboration agreement, Seattle Genetics has U.S. and Canadian commercialization rights and the Takeda Group has rights to commercialize ADCETRIS in the rest of the world. Seattle Genetics and the Takeda Group are funding joint development costs for ADCETRIS on a 50:50 basis, except in Japan where the Takeda Group will be solely responsible for development costs. Seattle Genetics is entitled to royalties based on a percentage of Millennium's net sales in its territory at rates that range from the mid-teens to the mid-twenties based on sales volume, subject to offsets for royalties paid by Millennium to third parties.

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 FDA granted accelerated approval of ADCETRIS in August 2011 for two indications. ADCETRIS is being developed in collaboration with Millennium: The Takeda Oncology Company. In addition, Seattle Genetics has three other clinical-stage ADC programs: SGN-75, ASG-5ME and ASG-22ME. Seattle Genetics has collaborations for its ADC technology with a number of leading biotechnology and pharmaceutical companies, including Abbott, Bayer, Celldex Therapeutics, Daiichi Sankyo, Genentech, GlaxoSmithKline, Millennium, Pfizer and Progenics, as well as ADC co-development agreements with Agensys, an affiliate of Astellas, and Genmab. More information can be found at http://www.seattlegenetics.com.

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Seattle Genetics Announces ADCETRIS® Receives European Commission Conditional Marketing Authorization

David Levy CNN iReport Article - Gene Therapy: Cure for the Future?
Gene Therapy: Cure for the Future? Full Acticle - ireport.cnn.com David Levy on Facebook - http://www.facebook.com Animation Short Film: Gene Therapy in Thalassemia Major Patients - http://www.youtube.com Subscibe to my Video Blog Channel - http://www.youtube.com Subscibe to my Podcast Channel - http://www.youtube.com My Official Website - http://www.thalassemianme.org Website - http My Blog - thalassemianme.blogspot.sg Add me on Facebook - https Like me on Facebook - http://www.facebook.com Twitter - twitter.com Google + - plus.google.comFrom:SuperYoutub8Views:13 1ratingsTime:03:20More inPeople Blogs

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David Levy CNN iReport Article - Gene Therapy: Cure for the Future? - Video

Sickle Cell Anemia gene therp step
Dr lelbouch explain gene therapy in the case of sickle celle disease.From:dan mart wedoViews:12 0ratingsTime:01:49More inEducation

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Sickle Cell Anemia gene therp step - Video

[91VOA]Gene Therapy Used to Treat Cancer
English language lessons from the audio and transcript of the Voice of America. VOA Special English helps you learn English with lesson plans, grammar lessons, news and activities.From:91VOAChannelViews:1 0ratingsTime:04:14More inEducation

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[91VOA]Gene Therapy Used to Treat Cancer - Video

Dr. Shoukhrat Mitalipov explains his gene therapy method to prevent several childhood diseases
From:OHSUvideoViews:34 0ratingsTime:03:46More inNonprofits Activism

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Dr. Shoukhrat Mitalipov explains his gene therapy method to prevent several childhood diseases - Video

Subtitled -- Peter Weyland - Promotheus Speech - TED - 2023
TED 2023 Speech -- subtitled courtesy of Per Jacobsen engineeredreality.wordpress.com Peter Weyland has been a magnet for controversy since he announced his intent to build the first convincingly humanoid robotic system by the end of the decade. Whether challenging the ethical boundaries of medicine with nanotechnology or going toe to toe with the Vatican itself on the issue of gene-therapy sterilization, Sir Peter prides himself on his motto, "If we can, we must." After a three year media blackout, Weyland has finally emerged to reveal where he #39;s heading next. Wherever that may be, we will most certainly want to follow.From:Per JacobsenViews:14 0ratingsTime:03:09More inFilm Animation

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Subtitled -- Peter Weyland - Promotheus Speech - TED - 2023 - Video

Healthier Humans or Designer Babies. Should scientists be playing GOD ?
Healthier Humans or Designer Babies. Should scientists be playing GOD ? It is a procedure that could prevent children from inheriting rare, incurable diseases such as stroke, blindness, deafness, kidney failure and heart disease. Healthy embryos are being created using two women and one man. Scientists are trying to eliminate defects that affect an estimated one in 4000 children. But it is stirring the ethics debate. There are a lot of questions about the morality of having babies with three parents, the implications for the child #39;s descendants, and the prospect of babies-to-order. The research for the new technique is being conducted at Oregon Health and Science University in the US. The procedure still needs US federal government approval to be tested on humans. In 2009, scientists were able to create eggs with DNA transplants from Rhesus monkeys. Four developed into healthy animals. The team has now used the same technique to create human embryos, with about half having abnormalities. But they say some 20 per cent of the eggs did produce embryos that would have been suitable for transfer back into the mother. So is science creating so-called designer babies? And should scientists be allowed to interfere with nature? To discuss this on Inside Story with presenter Zami Zeidan are guests: Josephine Quintavalle, the director of Core-ethics, a public interest group focusing on the ethics of human reproduction; Laurie Zoloth, a professor in biotechs and medical humanity at ...From:Tsiyonut timesViews:16 1ratingsTime:25:02More inNews Politics

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Healthier Humans or Designer Babies. Should scientists be playing GOD ? - Video

Gene Therapy and Synthetic Biology
How the relatively young field of synthetic biology is changing the older field of gene therapy. Produced by UIUC iGEM 2012From:iGEMHPCollaborationViews:13 0ratingsTime:06:05More inEducation

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Gene Therapy and Synthetic Biology - Video

Scientists reveal gene-swapping technique. Should scientists be playing GOD ?
Scientists reveal gene-swapping technique. Should scientists be playing GOD ? It is a procedure that could prevent children from inheriting rare, incurable diseases such as stroke, blindness, deafness, kidney failure and heart disease. Healthy embryos are being created using two women and one man. Scientists are trying to eliminate defects that affect an estimated one in 4000 children. But it is stirring the ethics debate. There are a lot of questions about the morality of having babies with three parents, the implications for the child #39;s descendants, and the prospect of babies-to-order. The research for the new technique is being conducted at Oregon Health and Science University in the US. The procedure still needs US federal government approval to be tested on humans. In 2009, scientists were able to create eggs with DNA transplants from Rhesus monkeys. Four developed into healthy animals. The team has now used the same technique to create human embryos, with about half having abnormalities. But they say some 20 per cent of the eggs did produce embryos that would have been suitable for transfer back into the mother. So is science creating so-called designer babies? And should scientists be allowed to interfere with nature? To discuss this on Inside Story with presenter Zami Zeidan are guests: Josephine Quintavalle, the director of Core-ethics, a public interest group focusing on the ethics of human reproduction; Laurie Zoloth, a professor in biotechs and medical ...From:Conspirafied0Views:49 0ratingsTime:25:02More inNews Politics

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Scientists reveal gene-swapping technique. Should scientists be playing GOD ? - Video

27-Medical BiotechnologySG Part II.Gene Therapy, Tissue Engineering and Nanotechnology.mov
In theory, gene therapy offers a straight forward to the molecular treatment of various diseases, by using genes to prevent or treat disease by: Replacing a mutated gene(s) with a healthy copy; Inactivating/knocking out a mutated gene; or, Introducing new gene. Gene Therapy offers an approach to treating disease by either modifying the expressions of an individual #39;s genes or correction of abnormal genes. While this seems straight forward (ie replace or supply a healthy insulin gene in pancreatic cells of a diabetic) it has been difficult to actualize. Think here also about dual use problems (ie genetic doping for athletes, genetic modification enhancement for military personnel, and germline genetic engineering of humans with heritable genes). in this section we also review tissue engineering and some of the possibilities for nanotechnologies in medical devices. Nanosized GPS capable computers in our bloodstream?From:Albert KauschViews:5 0ratingsTime:37:43More inEducation

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27-Medical BiotechnologySG Part II.Gene Therapy, Tissue Engineering and Nanotechnology.mov - Video

Researchers Test New Gene Therapy Method in Human Cells... And It Works
Researchers at the university #39;s Oregon National Primate Research Center and the OHSU Department of Obstetrics Gynecology have successfully demonstrated their procedure in human cells. It #39;s believed that this research, along with other efforts, will pave the way for future clinical trials in human subjects. Full report available at Scicasts via: s.scicasts.com [Video produced by Oregon Health Science University.]From:scicastsViews:9 0ratingsTime:03:46More inScience Technology

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Researchers Test New Gene Therapy Method in Human Cells... And It Works - Video

Stopping the Clock
Original Article: Telomerase Gene Therapy in Adult and Old Mice Delays Aging and Increases Longevity Without Increasing Cancer Bernades de Jesus et al. EMBO Molecular Medicine, 2010. 4(8) p. 691-704. To view more exciting science videos visit http://www.yourekascience.comFrom:YourekaScienceViews:2 0ratingsTime:05:51More inScience Technology

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Stopping the Clock - Video

Mixed results have been obtained from ground-breaking studies of a "three parent" method of preventing inherited mitochondrial diseases.

Scientists testing the technique, which involves switching DNA in a donor egg, successfully produced human embryos, but over half contained abnormalities.

Despite this, the US researchers said they had demonstrated a viable way of preventing a mother passing devastating diseases onto her offspring. The technique, known as maternal spinal transfer (MST), is one of the procedures that could be allowed following a landmark change in the law under consideration in the UK.

A public consultation is now under way on the proposal, which would effectively permit the creation of IVF babies with three genetic parents - a mother, a father and a donor.

[Related: Microsoft set to unveil Windows 8]

Mitochondria are rod-like structures in cells that have their own DNA - separate from that in the nucleus - and act as powerhouses, pumping out energy. Only mothers pass on mitochondrial genes to sons and daughters.

Defects in mitochondrial DNA (mDNA) give rise to a range of serious and potentially life-threatening diseases, including a form of muscular dystrophy, and conditions leading to hearing and vision loss, heart problems and intestinal disorders.

The aim of mitochondrial replacement is to prevent embryos being created with abnormal mitochondria, thereby breaking the chain of inherited disease.

MST is carried out before fertilisation. A spindle-shaped structure containing a mother's nuclear DNA is first removed from one of her eggs. This is transplanted into a healthy donor egg - with normal mitochondria - which has had its own spindle removed, and then fertilised by the intended father's sperm.

The developing embryo and baby now has cells containing nuclear DNA from both its parents, and healthy mitochondrial DNA from the donor.

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'Three parent' gene therapy results

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

bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, announced today that the California Institute for Regenerative Medicine (CIRM) has approved an award to the company for $9.3 million for the first round of its new Strategic Partnership Awards initiative. The award is to support a Phase 1/2 study to evaluate the safety and efficacy of LentiGlobin, the companys development-stage program for the treatment of beta-thalassemia, which will be initiated in the United States in 2013.

We are very encouraged by the clinical data generated to date demonstrating the potential of LentiGlobin as a one-time transformative gene therapy for patients with beta-thalassemia, said David Davidson, M.D., chief medical officer, bluebird bio. bluebird bio has made significant advances in lentiviral vector design, transduction efficiency, and in our manufacturing process, enabling the production of gene-modified products that can be scaled and deployed for many different clinical indications. We are delighted that CIRM has chosen to recognize the importance of this innovative approach for the treatment of one of the most commonly inherited blood disorders, and we are excited to work with CIRM to continue the development of LentiGlobin in the U.S.

bluebird bios LentiGlobin product introduces a fully functional human beta-globin gene into the patient's own hematopoietic stem cells. These corrected stem cells ultimately produce fully functioning red blood cells. bluebird bio is currently conducting a Phase 1/2 trial examining the feasibility, safety and efficacy of LentiGlobin in the treatment of beta-thalassemia and sickle cell disease. Results of the first patient were reported in Nature in 2010, showing dramatic results, including stable expression of functional beta-globin resulting in transfusion independence which now extends for greater than four years following a single treatment.

This CIRM award is among the first awards under the agencys Strategic Partnership Awards initiative, which is designed to engage more effectively with industry and to increase outside investment in CIRM-funded stem cell research. The funding awards were made at the October 25, 2012 meeting of the stem cell agencys governing board, the Independent Citizens Oversight Committee (ICOC).

About CIRM

CIRM was established in November 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Act. The statewide ballot measure, which provided $3 billion in funding for stem cell research at California universities and research institutions, was overwhelmingly approved by voters, and called for the establishment of an entity to make grants and provide loans for stem cell research, research facilities, and other vital research opportunities. A list of grants and loans awarded to date may be seen here: http://www.cirm.ca.gov/for-researchers/researchfunding.

About bluebird bio

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

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bluebird bio Awarded $9.3 Million From the California Institute for Regenerative Medicine to Further Gene Therapy ...

Newswise BOSTONAspirin therapy can extend the life of colorectal cancer patients whose tumors carry a mutation in a key gene, but has no effect on patients who lack the mutation, Dana-Farber Cancer Institute scientists report in the Oct. 25 issue of the New England Journal of Medicine.

In a study involving more than 900 patients with colorectal cancer, the researchers found that, for patients whose tumors harbored a mutation in the gene PIK3CA, aspirin use produced a sharp jump in survival: five years after diagnosis, 97 percent of those taking aspirin were still alive, compared to 74 percent of those not using aspirin. By contrast, aspirin had no impact on five-year survival rates among patients without a PIK3CA mutation.

Our results suggest that aspirin can be particularly effective in prolonging survival among patients whose colorectal cancer tests positive for a mutation in PIK3CA, said the studys senior author, Shuji Ogino, MD, PhD, of Dana-Farber, Brigham and Womens Hospital, and the Harvard School of Public Health. For the first time, we have a genetic marker that can help doctors determine which colorectal cancers are likely to respond to a particular therapy. He cautions that the results need to be replicated by other researchers before they can be considered definitive.

While aspirin is often prescribed for colorectal cancer patients, doctors havent been able to predict which patients will actually benefit from the treatment. The new finding suggests that the survival benefit is limited to the 20 percent whose tumors have the PIK3CA mutation.

For the remaining patients, aspirin may still be used, but it is likely to be much less effective and can sometimes lead to gastrointestinal ulcers and stomach bleeding.

The study was prompted by previous research that suggested that aspirin blocks an enzyme called PTGS2 (cyclooxygenase-2), causing a slowdown in the signaling activity of another enzyme, PI3K. That led researchers to hypothesize that aspirin could be especially effective against colorectal cancers in which the PIK3CA gene which provides a subunit of PI3K is mutated.

To conduct the study, investigators obtained data on 964 patients with rectal or colon cancer from the Nurses Health Study and the Health Professionals Follow-up Study long-term tracking studies of the health of tens of thousands of people. The data included information on the patients use of aspirin after diagnosis and the presence or absence of PIK3CA mutations in their tumor tissue.

The study, which combines the study of disease-related genes and research into large populations of individuals, represents a new, hybrid field which Ogino has termed molecular pathology epidemiology. The field may help us bring together information from two frontiers of cancer research at both the molecular and population levels in ways that are beneficial to patients, he said.

The study was supported in part by grants from the National Institutes of Health (P01 CA87969, P01 CA55075, P50 CA127003, R01 CA149222, R01 CA137178, and R01 CA151993); the Bennett Family Fund for Targeted Therapies Research; the Entertainment Industry Foundation through the National Colorectal Cancer Research Alliance; the Frank Knox Memorial Fellowship at Harvard University; and a Damon Runyon Clinical Investigator Award.

The lead author of the study is Xiaoyun Liao, MD, PhD, of Dana-Farber. In addition to Ogino, the papers other corresponding author is Andrew Chan, MD, MPH, of Brigham and Womens and Massachusetts General Hospital. Co-authors include Paul Lochhead, MB, ChB, Reiko Nishihara, PhD, Aya Kuchiba, PhD, Mai Yamauchi, PhD, Yu Imamura, MD, PhD, Zhi Rong Qian, MD, PhD, Ruifang Sun, and Jeffrey Meyerhardt, MD, MPH, of Dana-Farber; Charles Fuchs, MD, MPH, of Dana-Farber and Brigham and Womens Hospital; Edward Giovannucci, MD, MPH, of the Harvard School of Public Health and Brigham and Womens; Teppei Morikawa, MD, PhD, of University of Tokyo Hospital; Yoshifumi Baba, MD, PhD, of Kumamoto University, Japan; Kaori Shima, DDS, PhD, of Kagoshima University, Japan; and Katsuhiko Nosho, MD, PhD, of Sapporo Medical University, Japan.

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Gene Mutation Identifies Colorectal Cancer Patients Who Live Longer with Aspirin Therapy

Researchers at Oregon Health & Science University say they have perfected a new gene therapy that could block the transmission of many inherited diseases from mother to child.

But the procedure could raise new ethical questions over genetically engineering offspring.

Already tested in monkeys, the technique replaces defective genetic material in a mother's egg cells before it is fertilized and implanted in the womb. In the new study, published in the journal Nature, researchers used 106 human egg cells obtained from volunteers.

If proven safe, the technique could be used to eliminate genetic dispositions toward diabetes, deafness and eye disorders, along with dementia, heart disease and neuropathy.

It would also give scientists the power to, for the first time, permanently alter the genetic material of future generations.

OHSU is already discussing the potential for human clinical trials with federal regulators, according to Shoukrat Mitalipov, the lead researcher.

Stay tuned for a fuller account later today.

-- Nick Budnick:

Twitter @nickbudnick

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Gene therapy procedure announced in Oregon could curb hereditary diseases, raise ethical questions

Public release date: 24-Oct-2012 [ | E-mail | Share ]

Contact: Jim Newman newmanj@ohsu.edu 503-494-8231 Oregon Health & Science University

PORTLAND, Ore. - Oregon Health & Science University's development of a new gene therapy method to prevent certain inherited diseases has reached a significant milestone. Researchers at the university's Oregon National Primate Research Center and the OHSU Department of Obstetrics & Gynecology have successfully demonstrated their procedure in human cells. It's believed that this research, along with other efforts, will pave the way for future clinical trials in human subjects.

The research results are online Wednesday, Oct. 24, in the highly respected journal Nature. Dr. Mitalipov also will present the results of his research at the American Society for Reproductive Medicine Conference in San Diego Oct. 24'.

The OHSU gene therapy method was initially devised through research in nonhuman primates led by Shoukhrat Mitalipov, Ph.D., associate scientist in the Division of Reproductive & Developmental Sciences at ONPRC, Oregon Stem Cell Center and OHSU School of Medicine departments of Obstetrics and Gynecology and Molecular and Medical Genetics.

The procedure was specifically developed to prevent diseases related to gene defects in the cell mitochondria. Mitalipov's previous work was published in the August 2009 edition of Nature. In the current study, Mitalipov, in collaboration with Paula Amato, M.D., associate professor of obstetrics and gynecology in the OHSU Center for Women's Health, demonstrated efficacy of this therapy in human gametes and embryos.

"Cell mitochondria contain genetic material just like the cell nucleus and these genes are passed from mother to infant," explained Mitalipov. "When certain mutations in mitochondrial DNA are present, a child can be born with severe conditions, including diabetes, deafness, eye disorders, gastrointestinal disorders, heart disease, dementia and several other neurological diseases. Because mitochondrial-based genetic diseases are passed from one generation to the next, the risk of disease is often quite clear. The goal of this research is to develop a therapy to prevent transmission of these disease-causing gene mutations."

To conduct this research, Mitalipov and his colleagues obtained 106 human egg cells from study volunteers recruited through OHSU's Division of Fertility and Reproductive Endocrinology. The researchers then used a method developed in previous nonhuman primate studies, to transfer the nucleus from one cell to another. In effect, the researchers "swapped out" the cell cytoplasm, which contains the mitochondria. The egg cells were then fertilized to determine whether the transfer was a success and whether the cells developed normally. Upon inspection, it was demonstrated that it was possible to successfully replace mitochondrial DNA using this method.

"Using this process, we have shown that mutated DNA from the mitochondria can be replaced with healthy copies in human cells," explained Mitalipov. "While the human cells in our study only allowed to develop to the embryonic stem cell stage, this research shows that this gene therapy method may well be a viable alternative for preventing devastating diseases passed from mother to infant."

The current Nature paper also expanded upon the previously reported nonhuman primate work by demonstrating that the method was possible using frozen egg cells. Mitochondria were replaced in a frozen/thawed monkey egg cell, resulting in the birth of a healthy baby monkey named Chrysta.

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OHSU researchers test new gene therapy method in human cells... and it works

ScienceDaily (Oct. 24, 2012) Oregon Health & Science University's development of a new gene therapy method to prevent certain inherited diseases has reached a significant milestone. Researchers at the university's Oregon National Primate Research Center and the OHSU Department of Obstetrics & Gynecology have successfully demonstrated their procedure in human cells. It's believed that this research, along with other efforts, will pave the way for future clinical trials in human subjects.

The research results are online Oct. 24, in the journal Nature. Dr. Mitalipov also will present the results of his research at the American Society for Reproductive Medicine Conference in San Diego Oct. 24

The OHSU gene therapy method was initially devised through research in nonhuman primates led by Shoukhrat Mitalipov, Ph.D., associate scientist in the Division of Reproductive & Developmental Sciences at ONPRC, Oregon Stem Cell Center and OHSU School of Medicine departments of Obstetrics and Gynecology and Molecular and Medical Genetics.

The procedure was specifically developed to prevent diseases related to gene defects in the cell mitochondria. Mitalipov's previous work was published in the August 2009 edition of Nature. In the current study, Mitalipov, in collaboration with Paula Amato, M.D., associate professor of obstetrics and gynecology in the OHSU Center for Women's Health, demonstrated efficacy of this therapy in human gametes and embryos.

"Cell mitochondria contain genetic material just like the cell nucleus and these genes are passed from mother to infant," explained Mitalipov. "When certain mutations in mitochondrial DNA are present, a child can be born with severe conditions, including diabetes, deafness, eye disorders, gastrointestinal disorders, heart disease, dementia and several other neurological diseases. Because mitochondrial-based genetic diseases are passed from one generation to the next, the risk of disease is often quite clear. The goal of this research is to develop a therapy to prevent transmission of these disease-causing gene mutations."

To conduct this research, Mitalipov and his colleagues obtained 106 human egg cells from study volunteers recruited through OHSU's Division of Fertility and Reproductive Endocrinology. The researchers then used a method developed in previous nonhuman primate studies, to transfer the nucleus from one cell to another. In effect, the researchers "swapped out" the cell cytoplasm, which contains the mitochondria. The egg cells were then fertilized to determine whether the transfer was a success and whether the cells developed normally. Upon inspection, it was demonstrated that it was possible to successfully replace mitochondrial DNA using this method.

"Using this process, we have shown that mutated DNA from the mitochondria can be replaced with healthy copies in human cells," explained Mitalipov. "While the human cells in our study were allowed to develop to the embryonic stem cell stage, this research shows that this gene therapy method may well be a viable alternative for preventing devastating diseases passed from mother to infant."

The current Nature paper also expanded upon the previously reported nonhuman primate work by demonstrating that the method was possible using frozen egg cells. Mitochondria were replaced in a frozen/thawed monkey egg cell, resulting in the birth of a healthy baby monkey named Chrysta.

The second portion of the study, which was completed at ONPRC, is also considered an important achievement because egg cells only remain viable for a short period of time after they are harvested from a donor. Therefore, for this therapy to be a viable option in the clinic, preservation through freezing likely is necessary so that both the donor cell and a mother's cell are viable at the time of the procedure.

While this form of therapy has yet to be approved in the United States, the United Kingdom is seriously considering its use for treating human patients at risk for mitochondria-based disease. It's believed that this most recent breakthrough, combined with earlier animal studies, will help inform that decision-making process.

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New gene therapy method tested in human cells ... and it works, researchers report

HORSHAM, Pa., Oct. 17, 2012 /PRNewswire/ --The 2012 U.S. Conference on Rare Diseases and Orphan Products will bring together the rare disease and orphan product communities to collaborate on and advance product research, drug development and access to treatments to help the 30 million Americans suffering from rare diseases. Sponsored by DIA and the National Organization for Rare Disorders (NORD), the conference will be held Oct. 22-24 at the Capital Hilton hotel in Washington, D.C.

Among the conference's influential speakers and moderators will be keynote speaker John J. Castellani, president and CEO of Pharmaceutical Research and Manufacturers of America, who will discuss special challenges in rare diseases, and Stephen C. Groft, director of the National Institutes of Health Office of Rare Diseases Research, who will moderate several panel discussions including one on gene therapy and genome sequencing.

Two influential journalists also will play a part in the sessions. Wall Street Journal reporter Thomas Burton will moderate a discussion about the environment surrounding investments in orphan products, and Steve Usdin, Washington editor of BioCentury and co-host of "BioCentury This Week," will moderate panel discussions about the ethical challenges of genetic testing and internationalization of the rare disease community.

Participants and other presenters will include representatives of patient organizations and drug and medical device companies, researchers, investors, thought leaders and regulatory officials.

Other conference sessions to be featured are Well-Designed and Well-Conducted Clinical Trials; Challenges of Reimbursement for the Rare Disease Patient; Comparative Effectiveness Research and Health Technology Assessments; and Role of Academic Centers in Orphan Product Development.

Another unique component of the 2012 conference will be an inspiring panel discussion on Oct. 22 featuring patients who have a rare disease and their advocates, who will talk about how nonprofit associations for rare diseases are playing a key role in the development of orphan products.

Visit http://www.diahome.org to learn more about the conference and register.

ABOUT DIA: DIA is a neutral, global, professional and member-driven association of nearly 18,000 professionals involved in the discovery, development and lifecycle management of pharmaceuticals, biotechnology, medical devices and related health care products. Through our international educational offerings and myriad networking opportunities, DIA provides a global forum for knowledge exchange that fosters the innovation of products, technologies and services to improve health and well-being worldwide. Headquarters are in Horsham, Pa., USA, with offices in Basel, Switzerland; Tokyo; Mumbai, India; and Beijing.

ABOUT NORD: NORD is a nonprofit organization representing U.S. patients and families affected by rare diseases. NORD was established in 1983 by leaders of rare disease patient organizations who worked together for the enactment of the Orphan Drug Act. Today, NORD provides programs and services that include advocacy, education of patients and medical professionals, support for research and patient assistance programs to help patients access lifesaving therapies.

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2012 U.S. Conference on Rare Diseases and Orphan Products: Shaping the Future Now

New research which has unravelled the barley genome could help produce better varieties of the crop - a vital part of the beer and whisky industries.

Scientists said the breakthrough is a critical step towards developing barley varieties able to cope with the demands of climate change.

The study, funded by the Biotechnology and Biological Sciences Research Council (BBSRC) and the Scottish Government, should also help in the fight against cereal crop diseases which cause millions of pounds of losses annually.

Researchers said barley is the second most important crop in UK agriculture, and malting barley, some 30% of the total, underpins the beer and whisky sector which is worth around 20 billion to the UK economy.

The UK team behind the international research was led by Professor Robbie Waugh, of Scotland's James Hutton Institute, who worked with researchers at the Genome Analysis Centre in Norwich.

He said: "Access to the assembled catalogue of gene sequences will streamline efforts to improve barley production through breeding for varieties better able to withstand pests and disease, and deal with adverse environmental conditions such as drought and heat stress.

"It will accelerate research in barley, and its close relative, wheat. Armed with this information, breeders and scientists will be much better placed to deal with the challenge of effectively addressing the food security agenda under the constraints of a rapidly changing environment."

As well as being used in the beer and whisky industry, barley is also a major component of the animal feed for the meat and dairy industries, while barley straw is used for animal bedding in the winter, and for frost protection in horticulture.

Unravelling the sequence of barley's DNA has proved a major challenge as the barley genome is almost twice the size of that of humans. Scientists from the International Barley Genome Sequencing Consortium (IBSC) managed to construct a high-resolution draft DNA sequence assembly which contains the majority of barley genes in linear order.

Published in the journal Nature, the sequence provides a detailed overview of the functional portions of the barley genome, revealing the order and structure of most of its 32,000 genes and a detailed analysis of where and when genes are switched on in different tissues and at different stages of development.

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Barley gene work 'can boost crops'

Submitted by The Life Sciences Report as part of our contributors program .

This interview was conducted by The Life Sciences Report (10/11/12)

The myth of technology, whether for smartphones or cancer treatments, is that the next big thing appears suddenly and magically. Casey Research Analyst Alex Daley sets the record straight in this exclusive interview with The Life Sciences Report . While the science of genetic medicine has accelerated the process of turning magical thinking into practical medicine, Daley cautions investors in biotech and medical device companies to be patient, and names companies with innovative technologies poised for explosive growth.

The Life Sciences Report: At Casey Research's "Navigating the Politicized Economy" summit, you talked about the difference between the speed of science and the speed of technology, and how quickly the time to market and cost of products in the life sciences space is decreasing. Can you provide some examples?

Alex Daley: Many technologies, like the touch-screen tablets and smartphones that now dominate the market, seem to come out of nowhere, perpetuating the myth of technology as almost magical. But you only have to look as far as the as-yet-unfulfilled promises of recent years to see the slow development curve that leads to explosive growth. This has been most noticeable in the advent of genetic medicine.

We all remember the sequencing of the human genome as a scientific milestone. Announced in 2000, just at the turn of the millennium, it was followed by much media fanfare about the dawn of genetic medicine. Every untreatable disease was going to be cured. Every person was going to receive medicine tailored to his or her unique makeup.

Yet, more than a decade later, that promise remains almost entirely unfulfilled. It's not that the science has stood still. Quite the opposite: It has been moving forward at blazing speed. The original human genome project, which sequenced a single person's genome to 92%?including everything but some particularly difficult areas?took 13 years and cost more than $3 billion ($3B). It was a monumental advancement, but not practical for everyday use.

Over the last decade the cost of genome sequencing has fallen far faster than many predicted. We've gone from taking 13 years to taking just about one day to sequence a whole genome. And the cost has fallen from billions to thousands of dollars. We've now sequenced tens of thousands of genomes for scientific research, and with the falling price that number is skyrocketing. We have built an amazing scientific base for study, and driven down costs to make it viable for mainstream use. All of that had to happen before genetic medicine could even begin to crawl forward?precisely what is happening now, with the advent of the first U.S. Food and Drug Administration (FDA)-approved antisense drug and other genetic milestones just being reached.

Just as the plasma TV (invented in the 1930s), the LED light (1960s), the industrial robot (also a child of the '60s), the touch-screen interface for computers (early 1980s) and other inventions we think of as thoroughly modern took decades to go from the lab into our everyday lives, it will take considerable time for genetic medicine to fully develop. But the pace is ever-increasing and advances happen at an astounding rate. The decrease in time needed for gene-sequencing, for instance, far outpaced the development of computer chips in terms of cost/speed, as in the famous Moore's law (predicting a doubling of circuit capacity every two years).

TLSR: What is the role of FDA in that race to market? Is it a speed bump, a safety crew or something else?

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Casey Analyst Forecasts Explosive Biotech Growth