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

Good Start Genetics, Inc.,an innovative molecular diagnostics company that has developed the new gold standard in carrier screening, today announced that it has closed a non-dilutive loan facility for up to $28 million of capital from Capital Royalty L.P. Good Start Genetics will use the proceeds to support its long-term corporate growth initiatives for the companys next-generation sequencing (NGS) based carrier screening platform.

Our investment in Good Start Genetics is consistent with our focus on providing flexible financing solutions for innovative companies with commercial technologies, said Charles Tate, chairman and founder of Capital Royalty L.P. We are excited about the significant long-term growth potential of Good Start given the combination of its unique next-generation sequencing based technology, applicability of GoodStart Select in large and growing markets, and very capable management team.

Good Start Genetics is a leading provider of carrier screening for the in vitro fertilization (IVF) market. Since its April 2012 commercial launch targeting the 460 IVF centers in the United States, Good Start Genetics high-complexity, CLIA- and CAP-accredited laboratory has processed tens of thousands of test orders. The GoodStart Select carrier screening service provides testing for all 23 diseases recommended by major medical societies and detects both common disease-causing mutations, as well as rare pathogenic mutations that would go undetected by laboratories using older, traditional genotyping-based technologies.

Were proud to have the support of Capital Royalty through this investment and under very attractive, non-dilutive terms, said Don Hardison, president and chief executive officer of Good Start Genetics. These funds further position us to continue growing our NGS-based GoodStart Select carrier screening presence within the IVF community, while evaluating potential opportunities to expand our reach into other areas, including global carrier screening markets. We are now in a strong financial position with sufficient capital to take us far beyond our projected 2013 profitability and cash flow operating goals.

About Good Start Genetics, Inc.

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

About Capital Royalty L.P.

Capital Royalty L.P. is a market pioneer and innovator in healthcare investing focused on intellectual property investments in approved products through structures including royalty bonds, secured debt, revenue interests and traditional royalty monetizations. Capital Royalty works directly with leading healthcare companies, research institutions and inventors to provide customized solutions to meet their unique financing needs. The value of each investment is based on the future revenue of commercialized biopharmaceutical products and medical technologies. Capital Royalty is actively making investments through its managed investment funds.

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Good Start Genetics Announces $28 Million Financing from Capital Royalty

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Seattle Genetics, Inc. (SGEN) reported first quarter 2013 net loss per share of 14 cents, narrower than the Zacks Consensus Estimate of a loss of 19 cents, but wider than the year-ago loss of 11 cents per share.

First quarter revenues were $57.3 million, compared with $48.2 million in the year-ago quarter. Revenues surpassed the Zacks Consensus Estimate of $54 million.

Net revenues for the first quarter included Adcetris revenues (down 1.7% to $33.9 million), collaboration and license agreement revenues (up 52.8% to $21 million) and royalty revenues. With Adcetris generating $33.9 million of revenues in this quarter, Seattle Genetics is on track to achieve its guidance of $130 million to $140 million by the end of 2013.

Research and development expenses increased 24% year over year to $47.7 million. Selling, general and administrative expenses fell 1.3% year over year to $21.9 million.

Pipeline Update

Seattle Genetics is making efforts to expand Adcetris label. In Jan 2013, a global phase III study (ECHELON-2) was initiated on Adcetris. In this study, Adcetris plus chemotherapy will be evaluated for the front-line treatment of CD30-positive mature T-cell lymphomas (:MTCL) including patients with systemic anaplastic large cell lymphoma (sALCL) and other types of peripheral T-cell lymphomas.

Seattle Genetics intends to submit a supplemental biologics license application (sBLA) in the first half of 2013 for the use of Adcetris in the retreatment of patients and for extended duration of use beyond 16 cycles of therapy. Adcetris is approved for the treatment of relapsed or refractory Hodgkin lymphoma (HL) and sALCL.

By mid-2013, Seattle Genetics will initiate a phase I/II study of Adcetris in combination with bendamustine for second-line HL patients.

In the second half of 2013, Seattle Genetics will initiate a phase II frontline study of Adcetris in combination with Roche Holding / Biogen Idecs (RHHBY / BIIB) Rituxan and standard chemotherapy (R-CHOP) for patients with diffuse large B-cell lymphoma.

Seattle Genetics has collaborations with various companies for the development of antibody-drug conjugates (ADCs). ADC collaborators are progressing on various programs.

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Narrower-than-Expected Loss at Seattle Genetics

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May 8, 2013 A gene associated with both protection against bacterial infection and excessive blood clotting could offer new insights into treatment strategies for deep-vein thrombosis -- the formation of a harmful clot in a deep vein. The gene produces an enzyme that, if inhibited via a specific drug therapy, could offer hope to patients prone to deep-vein clots, such as those that sometimes form in the legs during lengthy airplane flights or during recuperation after major surgery.

The research, which was led by Yanming Wang, a Penn State University associate professor of biochemistry and molecular biology, and Denisa Wagner, senior author with decades of research on thrombosis at the Boston Children's Hospital and the Harvard University Medical School, will be published in in the Online Early Edition of the journal Proceedings of the National Academy of Sciences during the week ending 10 May 2013.

The team's new findings are an extension of previous research by Wang and other scientists. In earlier studies, Wang and his colleagues had revealed that a gene in mice called Pad4 (peptidylarginine deiminase 4) produces an enzyme that plays an important role in protecting the body from infection. The researchers discovered that cells with a functioning PAD4 enzyme are able to build around themselves a protective, bacteria-killing web that is dubbed a NET (neutrophil extracellular trap).

Now, in their new research, team members have studied the PAD4 enzyme's role in clotting. Wang explained that, as a part of its NET-producing duties, PAD4 regulates the formation of chromatin -- the condensed form of DNA that the cell remodels to form chromosomes. "PAD4 decondenses chromatin by loosening up the interaction between DNA and special proteins called histones. The resulting chromatin threads then combine with protein fibers, blood platelets, and other materials to become, not only the bacteria-killing NET, but also the fluffy, scattered ball that comprises a blood clot." Wang added that, in some individuals, blood clots tend to form within deep veins. These clots can then travel to the heart, causing cardiac arrest, or to the lungs, causing breathing problems.

In one of their experiments, team members compared mice with a normally functioning Pad4 gene to mice with a defective gene. They found that, when veins were constricted, genetically normal mice -- those able to produce the PAD4 enzyme -- formed clots as expected. However, genetically mutated mice -- those unable to produce the enzyme -- did not form clots normally. In fact, the scientists noted a two-fold difference in clot formation between genetically normal and genetically abnormal mice at six hours after the procedure. After 48 hours, the difference had reached 10-fold. "We noted some clotting activity in these genetically abnormal mice, but the clots were not as bulky and were not maintained over time," Wang said. "Clearly, the PAD4 enzyme plays a critical role in the formation of a blood clot, as well as in the formation of a bacteria-fighting NET."

In another experiment, the research team transferred infection-combatting white blood cells -- called neutrophils -- from normal mice to genetically mutated mice. First author Kim Martinod, a graduate student in the Immunology Graduate Program at the Harvard University Medical School, found that, in response to vein constriction, these "rescued" mice now could function normally, forming clots as efficiently as mice with a functioning Pad4 gene, demonstrating that the Pad4 gene did produce a functioning PAD4 enzyme in these white blood cells to regulate blood clotting.

"PAD4, which is also called PADI4 in humans, is a necessary enzyme involved in multiple disorders," Wang explained. "On the one hand, it plays an integral part in the body's defense system, as we showed in earlier work: It is necessary in the production of the protective, bacteria-killing NET. On the other hand, our earlier work also showed that this enzyme acts to silence tumor-suppressor genes. Now, in our new research, we are starting to see that its overactivity also may be part of the reason that some individuals suffer from deep-vein clotting." Wang added that patients prone to deep-vein thrombosis might benefit from drugs that target the PAD4 enzyme. "In future research, specific drug therapies could be developed and tested with the goal of targeting this enzyme," Wang said. "If we could find a way to dial back the enzyme's clot-forming effects, we might be able to offer new hope to patients suffering from clotting disorders and deep-vein thrombosis."

In addition to Wang, Wagner, and Martinod, other scientists who contributed to this research include Jing Hu from Penn State; Melanie Demers, Tobias A. Fuchs, Siu Ling Wong, and Alexander Brill from the Harvard University Medical School and Boston Children's Hospital; and Maureen Gallant from Boston Children's Hospital.

The research was funded by the National Heart, Lung, and Blood Institute of the National Institutes of Health and the National Cancer Institute.

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Gene offers clues to new treatments for a harmful blood clotting disorder

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Spinal Cord Injury: Kevin Oldt Walks in KAFO Leg Braces
Spinal cord injury patient Kevin Oldt walks in KAFO leg braces at Good Shepherd Rehabilitation Network. http://www.GoodShepherdRehab.org.

By: GoodShepherdRehab

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Spinal Cord Injury: Kevin Oldt Walks in KAFO Leg Braces - Video

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Bonita Springs, Florida (PRWEB) May 07, 2013

The Alliance for the Advancement of Stem Cell Therapy and Research monetarily supports some of the treatments provided by Intercellular Sciences. The Alliance is a non-profit organization dedicated to promoting research and treatment of life altering diseases with adult stem cells.

Howie Linderman, founding board member of The Alliance, says, The Alliance is very proud to be a part of helping people obtain this breakthrough treatment. When I had my stem cell treatment in 2008, the process was much more complex. This new protocol will change the way the world views cardiac problems and we are excited to participate.

To become a supporting member, please visit http://www.thestemcellalliance.org

John C. and Eddie W. have had Dilated Cardiomyopathy for many years and as time has gone on their heart muscle has continued to weaken. The deterioration of their hearts function deteriorated to the point that walking across the room was impossible without shortness of breath. Eddie had become so bad that he was restricted to a wheelchair. Modern science has not been able to stop the progression of this devastating disease. All the medications and defibrillators can do is treat the symptoms. Both men were facing a very grim prognosis. In March they each underwent a treatment using their own stem cells to regenerate the heart muscle. Within several weeks they were both feeling better and able to do much more activity than before. Eddie was out of his wheel chair. John went back to bowling.

John C. (age 62, Dickson, TN), has had Cardiomyopathy since he was a young man. First diagnosed while in college he has had to adjust his life style to adapt to his weak heart function. His doctors recommended a defibrillator a few years ago and he has been dependent on it since. His symptoms became rapidly progressive in the last couple of months. He had to stop many of his activities including cardiac rehab. It had become just too difficult for him. Johns ejection fraction (the percent of pumping of the heart) had dropped to under 10%. His doctors were not optimistic. He and his wife Karen refused to accept such a grim fate and searched for alternative treatment. They soon found an option: adult stem cell therapy. I have much more energy now, said John after his treatment. Im back to walking the perimeter of my 6 acre property and Im back at cardiac rehab. I have even started bowling again which I had stopped. Karen says that the change has been Amazing! An echocardiogram done 3 weeks after his treatment showed an increase in his ejection fraction of 50%. It is expected that his heart function will continue to improve for up to 6 months.

Eddie W. (age 41, Americus, GA) has battled heart disease for the last 14 years. He went through several years with just mild symptoms but, the last year has been devastating. His heart function deteriorated to such a degree that he has been hospitalized twice recently for severe swelling and shortness of breath. The doctors told his wife Leslie to get his affairs in order. While he was in the hospital this last time his family searched for other options. Coming across adult stem cell treatment for heart disease they took a leap of faith and made arrangements for treatment that has to be done outside the United States. Eddie was so weak that he could only get around being pushed in a wheelchair. He also had a pump attached that delivered a medication called Primacor to help his heart. Leslie had to fill the pump once a day with this life sustaining medicine. Eddie was treated in March. The treatment was a success. Within one week the pump was discontinued. Within two weeks he was getting around without the wheelchair. We hosted a church function and Eddie was able to keep up the whole time. Leslie says He could barely get out of his wheelchair before and now to be hosting a party, I cant believe it. Im so grateful. My boys have their father back. Eddies treatment was in part supported by The Alliance for The Advancement of Stem Cell Therapy and Research.

Zannos G. Grekos, MD, MAAC, FACC, chief science officer of Intercellular Sciences and a Florida-based pioneer in the field of adult stem cell therapy explains how, in this groundbreaking treatment, cardiologists insert a catheter into the patients heart similar to a heart cath. In 20 minutes, about 30 separate injections of Regenocytes (activated stem cells) are introduced into the damaged part of the heart. The process of tissue repair begins almost immediately.

The activation provides a key step in the process, Dr. Zannos Grekos explains. The lab extracts the stem cells, concentrates and activates them into over a billion of regenerative adult stem cells while educating them to assist the specific organ that needs regeneration. These cells we call Regenocytes.

Patients remain in the hospital overnight for observation, and are typically discharged the next day. Patients are scheduled for regular subsequent visits after the stem cell treatment to monitor their progress and measure their results.

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The Alliance for the Advancement of Adult Stem Cell Therapy and Research Recently Has Proven Stem Cells To Be ...

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A new drug treatment that can switch off a key gene involved in skin cancer has been developed and tested in a world-first trial on Sydney patients.

The first-ever human trials of the drug, called Dz13, have shown it not only shrank the basal-cell carcinoma (BCC) skin cancers, but seemed to encourage the body's own immune system to fight them as well.

In a study published in the Lancet medical journal on Tuesday, Levon Khachigian from the University of NSW showed that for the first time Dz13 appears to be both safe and effective in humans, and could potentially be used in a number of cancers and other conditions.

[Turning off the gene] sends the tumour into a death spiral, which then triggers the body's own natural inflammatory and immune system to go into battle and shrink the tumour, he said.

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An international research team that included scientists from institutions across NSW were involved with developing and trialling the drug, funded by the Cancer Institute NSW, the Cancer Council, and the National Health and Medical Research Council.

Professor Khachigian said Dz13 was enormously promising because it targeted the "c-jun" gene we all have, which is overactive in skin cancers, as well as other conditions including macular degeneration and diabetic retinopathy.

It's a pivotal growth gene, or survival gene, he said.

Usually it stays relatively inactive, but when it is switched on it triggers the production of a protein that supports unhealthy cell growth.

We think it is a bit of a lynchpin protein, he said. We don't really know why it gets switched on, except in BCCs, where we know sunlight turns it on."

Link:
Skin cancer drug switches off gene

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May 6, 2013 UCLA life scientists have identified a gene previously implicated in Parkinson's disease that can delay the onset of aging and extend the healthy life span of fruit flies. The research, they say, could have important implications for aging and disease in humans.

The gene, called parkin, serves at least two vital functions: It marks damaged proteins so that cells can discard them before they become toxic, and it is believed to play a key role in the removal of damaged mitochondria from cells.

"Aging is a major risk factor for the development and progression of many neurodegenerative diseases," said David Walker, an associate professor of integrative biology and physiology at UCLA and senior author of the research. "We think that our findings shed light on the molecular mechanisms that connect these processes."

In the research, published today in the early online edition of the journal Proceedings of the National Academy of Sciences, Walker and his colleagues show that parkin can modulate the aging process in fruit flies, which typically live less than two months. The researchers increased parkin levels in the cells of the flies and found that this extended their life span by more than 25 percent, compared with a control group that did not receive additional parkin.

"In the control group, the flies are all dead by Day 50," Walker said. "In the group with parkin overexpressed, almost half of the population is still alive after 50 days. We have manipulated only one of their roughly 15,000 genes, and yet the consequences for the organism are profound."

"Just by increasing the levels of parkin, they live substantially longer while remaining healthy, active and fertile," said Anil Rana, a postdoctoral scholar in Walker's laboratory and lead author of the research. "That is what we want to achieve in aging research -- not only to increase their life span but to increase their health span as well."

Treatments to increase parkin expression may delay the onset and progression of Parkinson's disease and other age-related diseases, the biologists believe. (If parkin sounds related to Parkinson's, it is. While the vast majority of people with the disease get it in older age, some who are born with a mutation in the parkin gene develop early-onset, Parkinson's-like symptoms.)

"Our research may be telling us that parkin could be an important therapeutic target for neurodegenerative diseases and perhaps other diseases of aging," Walker said. "Instead of studying the diseases of aging one by one -- Parkinson's disease, Alzheimer's disease, cancer, stroke, cardiovascular disease, diabetes -- we believe it may be possible to intervene in the aging process and delay the onset of many of these diseases. We are not there yet, and it can, of course, take many years, but that is our goal."

'The garbage men in our cells go on strike'

To function properly, proteins must fold correctly, and they fold in complex ways. As we age, our cells accumulate damaged or misfolded proteins. When proteins fold incorrectly, the cellular machinery can sometimes repair them. When it cannot, parkin enables cells to discard the damaged proteins, said Walker, a member of UCLA's Molecular Biology Institute.

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Boosting 'cellular garbage disposal' can delay the aging process

Recommendation and review posted by Bethany Smith

American researchers found that the gene, which has previously been implicated in Parkinsons Disease, extended the healthy lifespan of fruit flies by more than 25 per cent.

They said that the research could have important implications for ageing and disease in humans.

The gene, called parkin, serves at least two functions: marking damaged proteins so that cells can discard them before they become toxic and removing damaged mitochondria from cells.

David Walker, an associate professor of integrative biology and physiology at University of California, Los Angeles, said: Ageing is a major risk factor for the development and progression of many neurodegenerative diseases. We think that our findings shed light on the molecular mechanisms that connect these processes.

In the research, published in the journal Proceedings of the National Academy of Sciences, Dr Walker and his colleagues demonstrated how parkin modulated the ageing process in fruit flies, which typically live less than two months. The researchers increased parkin levels in the cells of the flies and found that this extended their lifespan by more than 25 per cent, compared with a control group.

Dr Walker added: In the control group, the flies are all dead by Day 50. In the group with parkin overexpressed, almost half of the population is still alive after 50 days. We have manipulated only one of their roughly 15,000 genes, and yet the consequences for the organism are profound.

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Dorian Gray gene could add decades to life

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Previous studies have suggested that protein build up within cells may play an important role in aging.

The fountain of youth might be more like a trash shredder of youth.

Scientists at UCLA have found a single gene that, when stimulated to be overexpressed, extends the healthy life span of fruit flies by more than 25 percent.

[ALSO:Gene Mutation Linked to Migraines, Researchers Say]

The gene, called parkin, plays an important role in disposing of damaged proteins within a cell. Previous studies have suggested that protein build up within cells may play an important role in aging. In fruit flies, and potentially in humans, parkin "marks" damaged proteins and instructs the cell to dispose of them.

By stimulating parkin expression, thereby boosting the power of the "cellular garbage disposal," David Walker, lead author of the study, was able to keep a group of fruit flies alive much longer than normal.

"In the control group, the flies are all dead by day 50," Walker said in a statement. "In the group with parkin overexpressed, almost half of the population is still alive after 50 days. We have manipulated only one of their roughly 15,000 genes, and yet the consequences for the organism are profound."

[READ:Girls With Autism May Need Different Treatments Than Boys]

According to the study, published Monday in Proceedings of the National Academy of Sciences, overexpression of parkin led to "a significant increase in longevity without any physiological tradeoffs. Fruit flies altered to overexpress parkin remained "healthy, active and fertile" much longer than a control group.

Walker said that parkin has previously been linked to Parkinson's Disease in humans, but his finding suggests that parkin might play a role in other age-related diseases as well. Previous fruit fly studies have found that removing parkin leads to earlier death.

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Single Gene May Extend Lifespan by 25 Percent

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Public release date: 6-May-2013 [ | E-mail | Share ]

Contact: Michael C. Purdy purdym@wustl.edu 314-286-0122 Washington University School of Medicine

Scientists' picture of how a gene strongly linked to Alzheimer's disease harms the brain may have to be revised, researchers at Washington University School of Medicine in St. Louis have found.

People with harmful forms of the APOE gene have up to 12 times the risk of developing Alzheimer's disease compared with those who have other variations of the gene.

Many researchers believe that the memory loss and cognitive problems of Alzheimer's result from the buildup over many years of brain amyloid plaques. The plaques are made mostly of a sticky substance called amyloid beta.

For years, researchers have thought that the APOE gene increases Alzheimer's risk by producing a protein that binds to amyloid beta. Scientists thought that this bond could make it easier for plaques to form.

But in a new study now available online in the Proceedings of the National Academy of Sciences, Washington University researchers show that APOE and amyloid beta don't bind together in cerebrospinal fluid and in fluids present outside cells grown in dishes. This means they are unlikely to bind together in the fluids circulating in the brain. The cerebrospinal fluid was taken from people who were cognitively normal but have forms of APOE that increase the risk of Alzheimer's.

"This is the first time we've looked at naturally produced APOE and amyloid beta to see if and how much they bind together, and we found that they have very little interaction in the fluids bathing the brain," said David M. Holtzman, MD, the Andrew B. and Gretchen P. Jones Professor and head of neurology. "This suggests that we may need to rethink any therapeutic strategies that target APOE to slow amyloid plaque accumulation and Alzheimer's."

According to Holtzman, leading Alzheimer's researchers recently agreed that targeting APOE is a promising approach both for improving treatments for Alzheimer's. But to do that, scientists must first fully understand how the harmful forms of APOE increase risk of the disease.

"APOE is a major player in Alzheimer's, there's no question about that," said Philip Verghese, PhD, a postdoctoral research associate. "We did some additional studies in mice and cell cultures that suggested the APOE protein may be blocking a pathway that normally helps degrade amyloid beta."

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New perspective needed for role of major Alzheimer's gene

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Los Angeles, May 7 (IANS) Scientists have zero-ed in on a gene linked to Parkinson's disease that plays a role in delaying ageing in fruit flies, says a US study.

The University of California, Los Angeles (UCLA) life scientists have identified a gene previously implicated in Parkinson's disease that can delay the onset of ageing and extend the healthy life span of fruit flies.

The research, they say, could have important implications for ageing and disease in humans.

The gene, called parkin, serves at least two vital functions: It marks damaged proteins so that cells can discard them before they become toxic, and it is believed to play a key role in the removal of damaged mitochondria from cells.

"Ageing is a major risk factor for the development and progression of many neurodegenerative diseases," said David Walker, an associate professor of integrative biology and physiology at UCLA and senior author of the research. "We think that our findings shed light on the molecular mechanisms that connect these processes."

In the research, published Tuesday in the early online edition of the journal Proceedings of the National Academy of Sciences, Walker and his colleagues show that parkin can modulate the ageing process in fruit flies, which typically live less than two months.

The researchers increased parkin levels in the cells of the flies and found that this extended their life span by more than 25 percent, compared with a control group that did not receive additional parkin.

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'Parkinson's gene could extend lifespan'

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Posted with photos, video at: http://www.washington.edu/news/2013/05/06/new-device-can-extract-human-dna-with-full-genetic-data-in-minutes/

Newswise Take a swab of saliva from your mouth and within minutes your DNA could be ready for analysis and genome sequencing with the help of a new device.

University of Washington engineers and NanoFacture, a Bellevue, Wash., company, have created a device that can extract human DNA from fluid samples in a simpler, more efficient and environmentally friendly way than conventional methods.

The device will give hospitals and research labs a much easier way to separate DNA from human fluid samples, which will help with genome sequencing, disease diagnosis and forensic investigations.

"It's very complex to extract DNA," said Jae-Hyun Chung, a UW associate professor of mechanical engineering who led the research. "When you think of the current procedure, the equivalent is like collecting human hairs using a construction crane."

This technology aims to clear those hurdles. The small, box-shaped kit now is ready for manufacturing, then eventual distribution to hospitals and clinics. NanoFacture, a UW spinout company, signed a contract with Korean manufacturer KNR Systems last month at a ceremony in Olympia, Wash.

The UW, led by Chung, spearheaded the research and invention of the technology, and still manages the intellectual property.

Separating DNA from bodily fluids is a cumbersome process that's become a bottleneck as scientists make advances in genome sequencing, particularly for disease prevention and treatment. The market for DNA preparation alone is about $3 billion each year.

Conventional methods use a centrifuge to spin and separate DNA molecules or strain them from a fluid sample with a micro-filter, but these processes take 20 to 30 minutes to complete and can require excessive toxic chemicals.

UW engineers designed microscopic probes that dip into a fluid sample saliva, sputum or blood and apply an electric field within the liquid. That draws particles to concentrate around the surface of the tiny probe. Larger particles hit the tip and swerve away, but DNA-sized molecules stick to the probe and are trapped on the surface. It takes two or three minutes to separate and purify DNA using this technology.

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New Device Can Extract Human DNA with Full Genetic Data in Minutes

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Senator Durbin Defends Rallying with Communists and Anarchists
Senator Durbin Defends Rallying with Communists and Anarchists.

By: Rebel Pundit

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Senator Durbin Defends Rallying with Communists and Anarchists - Video

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May 7, 2013 UCLA researchers led by Drs. Peiyee Lee and Richard Gatti at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research have used induced pluripotent stem cells (iPSC) to advance disease-in-a-dish modeling of a rare genetic disorder, Ataxia Telangiectasia (A-T). Their discovery shows the positive effects of drugs that may lead to effective new treatments for the neurodegenerative disease. iPSC are made from patient skin cells rather than from embryos and can become any type of cells, including brain cells, in the laboratory.

The study appears online ahead of print today in the journal Nature Communications.

Patients with A-T begin life with neurological deficits that become devastating through progressive loss of function in a part of the brain called the cerebellum, which leads to severe difficulty with movement and coordination. A-T patients also suffer frequent infections due to their weakened immune systems and have increased cancer risk. A-T is caused by lost function in a gene, ATM, which normally repairs damaged DNA in the cells and preserves normal function.

Laboratory mouse models are commonly used to study A-T; however, mice with A-T do not experience the more debilitating effects that humans do. In mice with A-T, the cerebellum appears normal and they do not exhibit the obvious degeneration seen in the human brain. Therefore, it was critical to develop a human neural cell model to understand the neurodegenerative process of A-T and create a platform for testing new treatments.

Lee and colleagues used iPSC-derived neural cells developed from skin cells of A-T patients with a specific type of genetic mutation to create a disease-in-a-dish model. The researchers were able to model the characteristics of A-T in the laboratory, such as the cell's lack of ATM protein and inability to repair DNA damage. The model also allowed the researchers to identify potential new therapeutic drugs, called small molecule read-through (SMRT) compounds that increase ATM protein activity and improve the model cells' ability to repair damaged DNA.

"A-T patients with no ATM activity have severe disease but patients with some ATM activity do much better. This makes our discovery promising, because even a small increase in the ATM activity induced by the SMRT drug can potentially translate to positive effects for patients, slowing disease progression and hopefully improving their quality of life." Lee said.

These studies suggest that SMRT compounds may have positive effects on all other cell types in the body, potentially improving A-T patients' immune function and decreasing their cancer susceptibility. Additionally, the patient-specific iPSC-derived neural cells in this study combined with the SMRT compounds can be an invaluable tool for understanding the development and progression of A-T. This iPSC-neural cell A-T disease model also can be a platform to identify more potent SMRT drugs. The SMRT drugs identified using this model can potentially be applied to most other genetic diseases with the same types of mutation. This research was supported by training and research grants from the California Institute of Regenerative Medicine (CIRM), the National Institutes of Health, APRAT, A-T Ease and Scott Richards Foundation.

Disclaimer: This article is not intended to provide medical advice, diagnosis or treatment. Views expressed here do not necessarily reflect those of ScienceDaily or its staff.

Continued here:
Stem cell researchers move toward treatments for rare genetic nerve disease

Recommendation and review posted by Bethany Smith

Newswise UCLA researchers led by Drs. Peiyee Lee and Richard Gatti at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research have used induced pluripotent stem cells (iPSC) to advance disease-in-a-dish modeling of a rare genetic disorder, Ataxia Telangiectasia (A-T). Their discovery shows the positive effects of drugs that may lead to effective new treatments for the neurodegenerative disease. iPSC are made from patient skin cells rather than from embryos and can become any type of cells, including brain cells, in the laboratory. The study appears online ahead of print today in the journal Nature Communications.

Patients with A-T begin life with neurological deficits that become devastating through progressive loss of function in a part of the brain called the cerebellum, which leads to severe difficulty with movement and coordination. A-T patients also suffer frequent infections due to their weakened immune systems and have increased cancer risk. A-T is caused by lost function in a gene, ATM, which normally repairs damaged DNA in the cells and preserves normal function.

Laboratory mouse models are commonly used to study A-T; however, mice with A-T do not experience the more debilitating effects that humans do. In mice with A-T, the cerebellum appears normal and they do not exhibit the obvious degeneration seen in the human brain. Therefore, it was critical to develop a human neural cell model to understand the neurodegenerative process of A-T and create a platform for testing new treatments.

Lee and colleagues used iPSC-derived neural cells developed from skin cells of A-T patients with a specific type of genetic mutation to create a disease-in-a-dish model. The researchers were able to model the characteristics of A-T in the laboratory, such as the cells lack of ATM protein and inability to repair DNA damage. The model also allowed the researchers to identify potential new therapeutic drugs, called small molecule read-through (SMRT) compounds that increase ATM protein activity and improve the model cells ability to repair damaged DNA.

A-T patients with no ATM activity have severe disease but patients with some ATM activity do much better. This makes our discovery promising, because even a small increase in the ATM activity induced by the SMRT drug can potentially translate to positive effects for patients, slowing disease progression and hopefully improving their quality of life. Lee said.

These studies suggest that SMRT compounds may have positive effects on all other cell types in the body, potentially improving A-T patients immune function and decreasing their cancer susceptibility. Additionally, the patient-specific iPSC-derived neural cells in this study combined with the SMRT compounds can be an invaluable tool for understanding the development and progression of A-T. This iPSC-neural cell A-T disease model also can be a platform to identify more potent SMRT drugs. The SMRT drugs identified using this model can potentially be applied to most other genetic diseases with the same types of mutation. This research was supported by training and research grants from the California Institute of Regenerative Medicine (CIRM), the National Institutes of Health, APRAT, A-T Ease and Scott Richards Foundation.

The stem cell center was launched in 2005 with a UCLA commitment of $20 million over five years. A $20 million gift from the Eli and Edythe Broad Foundation in 2007 resulted in the renaming of the center. With more than 200 members, the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research is committed to a multi-disciplinary, integrated collaboration of scientific, academic and medical disciplines for the purpose of understanding adult and human embryonic stem cells. The center supports innovation, excellence and the highest ethical standards focused on stem cell research with the intent of facilitating basic scientific inquiry directed towards future clinical applications to treat disease. The center is a collaboration of the David Geffen School of Medicine, UCLAs Jonsson Cancer Center, the Henry Samueli School of Engineering and Applied Science and the UCLA College of Letters and Science. To learn more about the center, visit our web site at http://www.stemcell.ucla.edu.

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UCLA Stem Cell Researchers Move Toward Treatments for Rare Genetic Nerve Disease

Recommendation and review posted by Bethany Smith

The University of Arizona Genetics Core (UAGC), located in the Thomas W. Keating Bioresearch Building on the UA campus, is home to rows of rectangular machines that hum busily at all hours of the day and night. These machines, with names like Illumina HISEQ 2000 and Ion Torrent, are the inanimate workhorses of the UAGC, producing hundreds of thousands of pieces of data in a single run. They sequence DNA and RNA, the genetic code and the genetic messages that program the building blocks forming a plant, animal or human. This is Ryan Sprissler's workplace and his intellectual playground.Sprissler, staff scientist and manager at the UAGC, and a Ph.D. candidate in the UA genetics program, enjoys spreading the word about genetics and the mission of the UAGC. He and his colleagues use genetic tools to explore topics including the story of human migration, and the mysteries of human disease. Sprissler describes a particularly interesting project to which the UAGC contributed--the Genographic Project.The purpose of the Genographic Project, a collaboration involving the UAGC, National Geographic, IBM and researchers from around the world, was to study patterns of migration based on the genetics of human populations. If a population stays in one place long enough and experiences little or no exchange of genes with other groups, gene sequences common to the group will become fixed. Once fixed, they serve as genetic identifiers or markers for that group.Geneticists can test for these markers, associate them with locations on the globe and track human migration patterns--sometimes over tens of thousands of years.During the course of the project, more than 500,000 genetic samples were analyzed in laboratories like the UAGC. The UAGC provided kits for the collection of cheek cells for people who wanted to make personal DNA contributions. The UA completed its obligation to the Genographic Project and Sprissler has a new mission."My favorite projects are those that are the most applied," says Sprissler. "They are [the projects] using genetic research to directly affect individuals diagnostically." He seems most fond of an undertaking that may help discover the genetic causes of human neurological disorders.He tells the story of a young girl with a devastating disease of the nervous system. Doctors could not find a cause, and for years the disorder was misdiagnosed. The girl's physicians thought she might have Rett syndrome or a seizure disorder. Relying on trial and error, they prescribed medication that made little improvement in her condition.Eventually the girl and her family were connected with UAGC scientists. The UAGC sequenced the girl's individual exome--her genome minus uninformative repetitive sequences. They found the mutation that caused her disorder. Unfortunately there was still no cure for the disease, and the girl eventually died. Understanding the genetic underpinnings of the girl's disorder, however, brought some comfort to her family.Helping to solve genetic mysteries relating to human disease pushes Sprissler to work in overdrive. His goal is making personalized medicine a reality. Someday, he hopes, people will be able to sequence their genomes quickly and inexpensively in order to find disease susceptibility genes.Sprissler is an outgoing, but modest man who doesn't like to be the center of attention. His colleagues notice that quality and speak up on his behalf. "Ryan is a good leader, not just because of his skills in molecular biology, but also because he always has a positive attitude, making it fun to work with him," says Taylor Edwards, Assistant Staff Scientist at the UAGC and recent winner of the10th Jarchow Conservation award for his work on desert tortoise conservation genetics."This is like watching my mom talk about me," says Sprissler in response to Edwards' comments.Susan Miller, manager of scientific data analysis with the UA Biocomputing Service, also works with Sprissler. She says, "I love working with Ryan because, for whatever reason, we always end up laughing." She points to doodles Sprissler left on her whiteboard. "There's a part of him that's still a kid."It's probably that very quality that makes Sprissler a favorite of students who visit the UAGC. On a tour last fall, Sprissler bounded down the hall with a dozen molecular biology students in tow. He explained his favorite projects, and ended the tour by passing out his business cards. "Call me if you're interested in working here," said Sprissler to the students, as they got ready to leave. "Awesome," said a couple of them as they took his card. Follow Scientific American on Twitter @SciAm and @SciamBlogs.Visit ScientificAmerican.com for the latest in science, health and technology news. 2013 ScientificAmerican.com. All rights reserved.

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Solving the Mysteries of the Human Condition Using Genetic Tools

Recommendation and review posted by Bethany Smith

Public release date: 7-May-2013 [ | E-mail | Share ]

Contact: Markus M. Lerch lerch@uni-greifswald.de The JAMA Network Journals

Two genome-wide association studies and a subsequent meta-analysis have found that certain genetic variations are associated with susceptibility to Helicobacter pylori, a bacteria that is a major cause of gastritis and stomach ulcers and is linked to stomach cancer, findings that may help explain some of the observed variation in individual risk for H pylori infection, according to a study in the May 8 issue of JAMA.

"[H pylori] is the major cause of gastritis (80 percent) and gastroduodenal ulcer disease (15 percent-20 percent) and the only bacterial pathogen believed to cause cancer," according to background information in the article. "H pylori prevalence is as high as 90 percent in some developing countries but 10 percent of a given population is never colonized, regardless of exposure. Genetic factors are hypothesized to confer H pylori susceptibility."

Julia Mayerle, M.D., of University Medicine Greifswald, Greifswald, Germany, and colleagues conducted a study to identify genetic loci associated with H pylori seroprevalence. Two independent genome-wide association studies (GWASs) and a subsequent meta-analysis were conducted for anti-H pylori immunoglobulin G (IgG) serology in the Study of Health in Pomerania (SHIP) (recruitment, 1997-2001 [n =3,830]) as well as the Rotterdam Study (RS-I) (recruitment, 1990-1993) and RS-II (recruitment, 2000-2001 [n=7,108]) populations. Whole-blood RNA gene expression profiles were analyzed in RS-III (recruitment, 2006-2008 [n = 762]) and SHIP-TREND (recruitment, 2008-2012 [n=991]), and fecal H pylori antigen in SHIP-TREND (n=961).

Of 10,938 participants, 6,160 (56.3 percent) were seropositive for H pylori. GWAS meta-analysis identified an association between the gene TLR1 and H pylori seroprevalence, "a finding that requires replication in non-white populations," the authors write.

"At this time, the clinical implications of the current findings are unknown. Based on these data, genetic testing to evaluate H pylori susceptibility outside of research projects would be premature."

"If confirmed, genetic variations in TLR1 may help explain some of the observed variation in individual risk for H pylori infection," the researchers conclude.

(JAMA. 2013;309(18):1912-1920; Available pre-embargo to the media at http://media.JAMAnetwork.com)

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

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Genetic variations associated with susceptibility to bacteria linked to stomach disorders

Recommendation and review posted by Bethany Smith

Let #39;s Play The Sims 3 - Perfect Genetics Challenge - Episode 1
This is a POSSIBLE LP that I am starting - I have recorded 3 episodes and will look at the responses (comments and likes) to determine if I will add this cha...

By: Llandros09

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Let's Play The Sims 3 - Perfect Genetics Challenge - Episode 1 - Video

Recommendation and review posted by Bethany Smith

Myriad Genetics Inc. (MYGN: Quote) Tuesday reported third-quarter profit of $37.9 million or $0.46 per share, up from $29.6 million or $0.34 per share in same period last year.

On average, 17 analysts polled by Thomson Reuters expected the company to earn $0.40 per share for the quarter. Analysts' estimates typically exclude special items.

Revenues improved to $156.47 million from $129.78 million last year. Analysts expected revenues of $148.25 million.

Looking ahead, the company has raised its full-year 2013 earnings outlook to a range of $1.65 to $1.67 per share from the previous guidance of $1.55 to $1.58 per share.

Revenues are currently projected in a range of $595 million to $600 million, up from $575 million to $585 million, issued previously. Analysts currently expect the company to report full-year earnings of $1.58 per share on revenues of $583.50 million.

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Myriad Genetics Q3 Profit Up 28%, Lifts FY13 Outlook - Quick Facts

Recommendation and review posted by Bethany Smith

OTTAWA, ONTARIO--(Marketwired - May 7, 2013) - Beginning today, Canadian producers will benefit from more export opportunities in the Middle East. Agriculture Minister Gerry Ritz announced today that Turkey has approved imports of sheep and goat genetics from Canada.

"We are pleased that Turkey recognizes the safety and high quality of Canadian agricultural products," said Minister Ritz. "This agreement is an important achievement in our continued efforts to expand market access so Canadian producers can continue to grow our economy."

Access to the Turkish market flows from the Government's trade expansion goals and is the result of its focused efforts to create new opportunities and science-based trade for Canadian producers. Successful expansion of the Turkish market will also result in better awareness of Canadian products and services in the surrounding countries, leading to potential new market opportunities. Advancing trade with other countries in this region has been at the forefront for Canadian producers, as Turkey is viewed as a priority and emerging market.

"The Canadian Livestock Genetics Association (CLGA) thanks the Government of Canada for finalizing these protocols," said Rick McRonald, President of the CLGA. "The demand in Turkey for Canadian sheep and goat genetics is growing, so the resolution of the interruption in technical market access came at a crucial time. Canadian exporters will now be able to engage with their Turkish clients and partners in the confidence that technical barriers to trade in semen and embryos have been removed."

Total Canadian exports of animal genetics (semen and embryos) reached $103.6 million in 2012, representing an important export commodity. Producers will now have access to another market open to our high-quality agricultural products. The CLGA estimates the potential value of this market to be $250 000 over five years.

End-of-year trade statistics indicate that 2012 was Canada's best export year on record for the agriculture and food industry. In 2012, exports of Canadian agricultural and seafood products reached $47.7 billion, up 7.4 per cent from $44.4 billion in 2011.

Today's announcement is another example of what is being accomplished to enhance competitiveness and long-term growth in Canadian agriculture-priorities under the Growing Forward 2 policy framework. In addition to multi-year funding for risk management programs, Growing Forward 2 includes $3 billion in strategic initiatives for innovation, competitiveness, and market development.

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Canada Expands Export Opportunities for Sheep and Goat Genetics to Turkey

Recommendation and review posted by Bethany Smith

WASHINGTON--(BUSINESS WIRE)--

Today, the Genetics Policy Institute (GPI) announced the opening of a new, GPI National Affairs Office in Washington, D.C.

Recent years have seen GPIs involvement with policy and regulatory issues impacting the translation of stem cells and other advanced technologies into medicine steadily increase, necessitating the establishment of a new, permanent office location in the nations capital.

This new location will also give GPI the opportunity to begin hosting an array of high-level workshops, seminars, and events aimed at removing roadblocks to innovative bio-medical solutions found within the fields of stem cell research and regenerative medicine.

Said GPIs Executive Director, Bernard Siegel of the need for advocacy driven workshops and related events, While many other countries are establishing funded, national programs or strategies to advance cell therapies and regenerative medicine, the US lags.

He added that time is of the essence as patients are facing the crushing burden of chronic disease and immense human suffering. To fulfill the curative promise in years, rather than decades, Washington must declare regenerative medicine a national priority and immediately establish coordination between federal agencies. We need a consensus strategy that includes all necessary funding.

Being at the intersection of research, medicine, law and patient advocacy, GPI boasts a track record of bringing stakeholders together with the aim of delivering on the promise of stem cells and regenerative medicine.

About the Genetics Policy Institute (GPI): GPI is a 501(c)(3) nonprofit corporation with the mission to promote stem cell research and its application in medicine to develop therapeutics and cures for many otherwise intractable diseases and disorders. GPI pursues this mission through production of its flagship annual World Stem Cell Summit, publication of the World Stem Cell Report, the online newsletter 360 Weekly, special projects, speaking engagements, educational initiatives, and strategic collaborations.

GPI maintains offices in Palm Beach, Florida; Palo Alto, California and Washington, D.C. For more information, visit http://www.genpol.org.

Please direct all media inquires to GPI Associate Director, Alan Fernandez at Genetics Policy Institute, 701 8th Street NW, Suite 400, Washington, DC, telephone 888-238-1427 or email alan@genpol.org.

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Genetics Policy Institute Opens Washington, D.C. Office

Recommendation and review posted by Bethany Smith

SALT LAKE CITY, May 7, 2013 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (MYGN) today announced results for its third fiscal quarter and nine months ended March 31, 2013. Revenue for the third fiscal quarter increased 21 percent over the same period in the prior year to $156.5 million. Third fiscal quarter earnings per diluted share were $0.46, an increase of 34 percent over the same period of the prior year.

"I am pleased with not only the solid revenue growth across all of our business segments during the third quarter but also the Company's ability to drive financial leverage as we continue to invest in our extensive product pipeline," said Peter D. Meldrum, President and Chief Executive Officer of Myriad Genetics, Inc. "We are very optimistic regarding the future outlook as we seek to expand our core markets, diversify our business through new product introductions, and expand internationally."

Third Fiscal Quarter 2013 Results

Year-to-Date Performance

Business Highlights during the Third Quarter of Fiscal 2013

Fiscal Year 2013 Outlook

The Company is raising its revenue expectations for fiscal year 2013 financial performance. Total revenue is now expected to be in a range of $595 million to $600 million, an increase to the previous guidance range of $575 million to $585 million. This represents 20 percent to 21 percent growth over our prior fiscal year. The Company is also increasing its guidance for fiscal year 2013 diluted earnings per share to $1.65 to $1.67, up from the previous guidance of $1.55 to $1.58 per share. The new range represents 27 percent to 28 percent growth over fiscal year 2012 diluted EPS. These projections are forward looking statements and are subject to the risks summarized in the safe harbor statement at the end of this press release. The Company will provide further detail on its business outlook during the conference call it is holding today to discuss its fiscal 2013 third quarter financial results.

Share Repurchase Program

Between May 2010 and March 2013, the Company repurchased $525 million of its outstanding common stock. In February 2013, our Board of Directors authorized the Company to repurchase an additional $200 million of our outstanding common stock. As of March 31, 2013, there is approximately $175 million available to repurchase shares under this authorization. The Company is authorized to repurchase shares from time to time through open market transactions or privately negotiated transactions as determined by management. The amount and timing of stock repurchases under the program will depend on business and market conditions, stock price, trading restrictions, acquisition activity and other factors.

Conference Call and Webcast

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Myriad Genetics Reports Third Quarter Fiscal Year 2013 Results

Recommendation and review posted by Bethany Smith

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

Seattle Genetics, Inc. (SGEN) today reported financial results for the first quarter ended March 31, 2013. The company also highlighted ADCETRIS (brentuximab vedotin) commercialization and clinical development activities, progress with its antibody-drug conjugate (ADC) pipeline and technology and upcoming milestones.

In 2013 we have continued to deliver on the ambitious goals we have set for ADCETRIS, our pipeline and our ADC technology, said Clay B. Siegall, Ph.D., President and Chief Executive Officer at Seattle Genetics. We and our collaborator, Millennium: The Takeda Oncology Company, are bringing ADCETRIS to patients in need through approvals to date in the United States, Canada, European Union and Switzerland. In addition, across both corporate and investigator-sponsored studies, there are currently more than 20 ongoing ADCETRIS clinical trials, including four phase 3 studies. Seattle Genetics is also advancing several additional ADCs, including two programs planned for phase 1 trial initiations during 2013, while our collaborators continue to advance more than a dozen ADCs in clinical development.

Recent ADCETRIS Highlights

Other Recent Highlights

Upcoming Milestones

First Quarter Financial Results

Total revenues in the first quarter of 2013 were $57.3 million, compared to $48.2 million in the first quarter of 2012. First quarter 2013 revenues include ADCETRIS net product sales of $33.9 million and ADCETRIS royalty revenues of $2.4 million from international sales of ADCETRIS by Takeda/Millennium. Revenues also reflect amounts earned under the companys ADCETRIS and ADC collaborations totaling $21.0 million in the first quarter of 2013.

Research and development expenses for the first quarter of 2013 were $47.7 million, compared to $38.5 million for the first quarter of 2012. This planned increase in 2013 was primarily driven by ADCETRIS expenses, including clinical trials to explore potential additional uses of ADCETRIS and drug supply to Takeda/Millennium under the collaboration. Research and development expenses also include increased investment in the companys ADC pipeline programs. Selling, general and administrative expenses for the first quarter of 2013 were $21.9 million, compared to $22.2 million for the first quarter of 2012.

Under the ADCETRIS collaboration with Takeda/Millennium, development costs incurred by Seattle Genetics are included in research and development expense. Joint development costs are co-funded by Takeda/Millennium on a 50:50 basis. Net reimbursement funding received from Takeda/Millennium is recognized as revenue over the development period of the collaboration along with other development payments received, including the upfront payment and development milestone payments. Seattle Genetics co-funds development activities performed by Takeda/Millennium under the collaboration, which reduces the amount of reimbursement funding received from Takeda/Millennium.

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Seattle Genetics Reports First Quarter 2013 Financial Results

Recommendation and review posted by Bethany Smith

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/lvclfm/gene_therapy) has announced the addition of Jain PharmaBiotech's new report "Gene Therapy - Technologies, Markets and Companies" to their offering.

Gene therapy can be broadly defined as the transfer of defined genetic material to specific target cells of a patient for the ultimate purpose of preventing or altering a particular disease state. Genes and DNA are now being introduced without the use of vectors and various techniques are being used to modify the function of genes in vivo without gene transfer. If one adds to this the cell therapy particularly with use of genetically modified cells, the scope of gene therapy becomes much broader. Gene therapy can now combined with antisense techniques such as RNA interference (RNAi), further increasing the therapeutic applications. This report takes broad overview of gene therapy and is the most up-to-date presentation from the author on this topic built-up from a series of gene therapy report written by him during the past decade including a textbook of gene therapy and a book on gene therapy companies. This report describes the setbacks of gene therapy and renewed interest in the topic

Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.

Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included.

Research and development is in progress in both the academic and the industrial sectors. The National Institutes of Health (NIH) of the US is playing an important part. As of 2012, over 2030 clinical trials have been completed, are ongoing or have been approved worldwide.A breakdown of these trials is shown according to the areas of application.

The voluminous literature on gene therapy was reviewed and selected 750 references are appended in the bibliography.The references are constantly updated. The text is supplemented with 73 tables and 15 figures.

Profiles of 181 companies involved in developing gene therapy are presented along with 204 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade. These companies have been followed up since they were the topic of a book on gene therapy companies by the author of this report. John Wiley & Sons published the book in 2000 and from 2001 to 2003, updated versions of these companies (approximately 160 at mid-2003) were available on Wiley's web site. Since that free service was discontinued and the rights reverted to the author, this report remains the only authorized continuously updated version on gene therapy companies.

Benefits of this report

- Up-to-date on-stop information on gene therapy with 73 tables and 15 figures

Link:
Research and Markets: Gene Therapy - Technologies, Markets and Companies - Updated 2013 Report

Recommendation and review posted by Bethany Smith

Stem Cell Patient Treated With Adult Stem Cell Therapy

By: Shaun Scott

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Stem Cell Patient Treated With Adult Stem Cell Therapy - Video

Recommendation and review posted by Bethany Smith