Inform Genomics Announces Preliminary Results of OnPART™ Personalized Medicine Product: SNP Network Identifies …
BOSTON--(BUSINESS WIRE)--
Inform Genomics, Inc., a private company focused on developing novel platforms of genomic based personalized medicine products for cancer supportive care and inflammatory diseases, today announced preliminary results from its initial study for its lead product, OnPART, which is designed to determine an individuals risk of side-effects associated with chemotherapy regimens based on his or her individual genomic profile. The study utilized advanced Bayesian network technology to identify (single-nucleotide polymorphism) SNP networks associated with common side-effects of chemotherapy regimens. In the plenary session Molecular Predictors in Supportive Care, Dr. Stephen T. Sonis, D.M.D., D.M.Sc. who is a co-founder of Inform Genomics and also a Clinical Professor of Oral Medicine at the Harvard School of Dental Medicine, reported that for patients receiving dose-dense doxorubicin, cyclophosphamide, plus paclitaxel (AC+T) a preliminary SNP network correctly identified patients at risk for chemotherapy-induced diarrhea with an accuracy of 96.7% and an area under the receiver operating characteristic (ROC) curve of 0.994.
We are very encouraged by these early results and they are in line with what we previously demonstrated with a high-degree of precision using our Bayesian network technology to predict oral mucositis with our transplant product, said Ed Rubenstein, President & CEO of Inform Genomics. We are excited to see further data demonstrating OnPARTs ability to predict serious toxicities from chemotherapy regimens based on his or her individual genomic profile which may lead to actions to reduce the burden of potentially curable interventions for patients with cancer.
About OnPART
OnPART, Oncology Preferences And Risk of Toxicity, is Inform Genomics lead platform personalized medicine product for treatment decisions in patients who will receive chemotherapy for colorectal, breast, lung, or ovarian cancer. Based upon response rates and survival, more than one chemotherapy regimen may be considered appropriate care for patients with these common solid tumors, yet the regimens vary widely in their side-effect profiles. OnPART is being developed to assess genomic risk for common and often debilitating therapy-related side-effects, including fatigue, nausea and vomiting, diarrhea, oral mucositis, cognitive dysfunction and peripheral neuropathy. The product includes a differentiating factor in personalized medicine, quantifying patient concerns for side-effects, using a copyrighted patient questionnaire (Preference Assessment Inventory). OnPART is expected to provide valuable information for patients and medical oncologists to help clarify critical clinical choices and be commercially available in 2014.
About Cancer Supportive Care
Most patients with cancer receive supportive care as part of their multimodal anti-cancer therapy, regardless of cancer diagnosis, stage of disease, or treatment modality. Common symptoms associated with cancer or its treatments include fatigue, nausea and vomiting, diarrhea, oral mucositis, cognitive dysfunction, and peripheral neuropathy. Some of these conditions are manageable with commercially available medications, while others are the focus of drug development programs. The development of these side-effects may interfere with ongoing anti-cancer treatment, impair patient functioning, negatively impact the patients quality of life, and may increase the risk of mortality. Treatment of these side-effects also results in significant costs for payers and providers.
About Inform Genomics
Inform Genomics, Inc. is a private company focused on developing novel platforms of genomic based personalized medicine products for cancer supportive care and inflammatory diseases, including its lead platform product, OnPART, designed to determine an individuals risk of side-effects associated with chemotherapy regimens based on his or her individual genomic profile. The companys business model leverages existing technology in conjunction with proprietary analytic methods for conducting genome-wide association studies. Product development programs will lead to commercial, single source laboratory tests consisting of single-nucleotide polymorphism (SNP) networks that determine the likelihood of individual patient clinical outcomes to drug therapies. The U.S. market opportunity for these differentiated products exceeds $2 billion annually. Inform Genomics is headquartered in Boston, Massachusetts. For more information, please visit http://www.informgenomics.com.
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ACT Announces Second Patient with Stargardt’s Disease Treated in EU Clinical Trial
MARLBOROUGH, Mass.--(BUSINESS WIRE)--
Advanced Cell Technology, Inc. (ACT; OTCBB: ACTC), a leader in the field of regenerative medicine, today announced treatment of the second patient in its Phase 1/2 clinical trial for Stargardts macular dystrophy (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The surgery was performed on Friday, June 29 at Moorfields Eye Hospital in London, the same site as the first patient treatment in January, by a team of surgeons led by Professor James Bainbridge, consultant surgeon at Moorfields and Chair of Retinal Studies at University College London. The procedure was successfully performed without any complications. ACT and Moorfields Eye Hospital recently received clearance from the Data and Safety Monitoring Board (DSMB) to treat the final two patients in the first cohort of this clinical trial.
We are very pleased to continue our forward momentum with both our U.S. trials and our European trial, commented Gary Rabin, chairman and CEO. It was less than a month ago that we received DSMB approval to treat the second and third patients in our E.U. trial, and it is very gratifying to have already completed dosing of the second. It is a pleasure to be working with Professor Bainbridge and the rest of his team at Moorfields Eye Hospital, and we continue to be encouraged by the steady progress of the trial thus far.
The Phase 1/2 trial is designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation in patients with SMD at 12 months, the studys primary endpoint. It will involve a total of 12 patients, with cohorts of three patients each in an ascending dosage format. It is similar in design to the U.S. trial for SMD that was initiated in July 2011.
The European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) has officially designated ACT's human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cells as an orphan medicinal product for the treatment of Stargardt's Macular Dystrophy (SMD).
More information on the status of the companys clinical trials will be posted today on Mr. Rabins Chairmans blog.
About Stargardts Disease Stargardts disease or Stargardts Macular Dystrophy is a genetic disease that causes progressive vision loss, usually starting in children between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, called the retinal pigment epithelium, which is the site of damage that the company believes the hESC-derived RPE may be able to target for repair after administration.
About Advanced Cell Technology, Inc. Advanced Cell Technology, Inc. is a biotechnology company applying cellular technology in the field of regenerative medicine. For more information, visit http://www.advancedcell.com.
Forward-Looking Statements Statements in this news release regarding future financial and operating results, future growth in research and development programs, potential applications of our technology, opportunities for the company and any other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words will, believes, plans, anticipates, expects, estimates, and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements, including: limited operating history, need for future capital, risks inherent in the development and commercialization of potential products, protection of our intellectual property, and economic conditions generally. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in the companys periodic reports, including the report on Form 10-K for the year ended December 31, 2011. Forward-looking statements are based on the beliefs, opinions, and expectations of the companys management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. Forward-looking statements are based on the beliefs, opinions, and expectations of the companys management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. There can be no assurance that the Companys clinical trials will be successful.
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ACT Announces Second Patient with Stargardt’s Disease Treated in EU Clinical Trial
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DPR Construction Awarded Multi-Million Dollar Contract to Manage Build Out of Shire Advance BioHealing Campus in …
SAN DIEGO, CA--(Marketwire -07/02/12)- DPR Construction (DPR) has been awarded a multi-million dollar contract to manage the construction of a regenerative medicine campus on behalf of Advanced BioHealing and Shire plc, the global specialty biopharmaceutical company, with real estate partner BioMed Realty Trust, Inc. and designers Ferguson Pape Baldwin Architects.
The new campus in Sorrento Mesa will expand the operational footprint of Shire's regenerative medicine business, Advanced BioHealing, Inc., and create the increased capacity it needs to meet future demand for its lead product, DERMAGRAFT, while offering additional space and infrastructure to manufacture new regenerative medicine products.
"This project will generate hundreds of jobs, including much needed construction jobs, during the build out over the new two years, and we are honored to be a part of it," said Jay Leopold, Regional Manager of DPR's San Diego office. "We look forward to working with the project partners to implement our High Performance Team strategies and create a model facility for attracting more biomedical business here in San Diego."
Phase I of the site development will be in excess of 150,000 square feet and will house the company's manufacturing and associated support operations, commercial operations, corporate, and administrative functions. Preconstruction and design is underway with construction scheduled to begin in 2013. Initial occupancy is targeted for 2014.
About DPR ConstructionDPR Construction, named by FORTUNE as #13 of the "100 Best Companies To Work For," is a forward-thinking national general contractor and construction manager specializing in life science, healthcare, corporate office, advanced technology and higher education projects. A privately held, employee-owned company, DPR has grown to more than $1 billion in annual revenue, making it one of the largest general contractors in the nation. Locally, DPR is a perennial Top 5 general contractor with annual revenue averaging well over $200 million in projects ranging from $5k to $600M. DPR's San Diego office is located at 5010 Shoreham Place, San Diego, CA 92122. For more information about DPR, visit: http://www.dpr.com.
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A Court Allows Payment for Bone Marrow. Should People Be Able to Sell Their Parts?
Ed Reschke / Getty Images
A color-enhanced photograph of spongy (Cancellous) bone red bone marrow fills the space.
How much would it take for you to consider selling your bone marrow? A U.S. appeals court puts the price at about $3,000 in a ruling that now makes it legal to pay donors for their bone marrow tissue.
The courts decision may well help thousands of sick patients who need bone marrow transplants to survive, but it also begs the question, what other body parts might next be up for sale?
The ruling came about at the end of 2011, in a decision to an October 2009 lawsuit brought by a group of cancer patients, parents and bone marrow donation advocates against the government over the federal law banning the buying and selling of bodily organs. The plaintiffs were led by Doreen Flynn, who has three daughters who suffer from Fanconi anemia, a blood disorder that requires bone marrow transplants to treat. Flynn and the other plaintiffs said that too many such patients die waiting for transplants and argued that we should be allowed to pay people to donate their marrow as a way of ensuring a more reliable supply. The U.S. Court of Appeals for the Ninth Circuit agreed.
(MORE: Facebook Now Lets Organ Donors Tell Their Friends)
At the core of the plaintiffs argument was the National Organ Transplantation Act (NOTA), which since 1984 has forbid the buying and selling of human organs, including bone marrow. But new developments in bone marrow extraction have made marrow donation not much different from donating blood: traditionally, bone marrow donation required anesthesia and long needles to extract the marrow from the hipbones of donors. Now, a technique called peripheral apheresis allows doctors to extract blood stem cells directly from the blood, instead of the bone patients first take a drug that pulls stem cells from the bone and into the blood meaning that the marrow cells should be considered a fluid like blood, rather than an organ, the plaintiffs argued. NOTA doesnt prohibit payments for blood or other fluids, such as plasma or semen.
U.S. Attorney General Eric Holder decided not to ask the U.S. Supreme Court to review the appellate courts decision, which would have been the next step in overturning it. That means the ruling stands and that people can now be paid up to $3,000 for their marrow, as long as it is collected by apheresis. In a concession to the spirit of NOTA, however, the compensation cant be in cash; it needs to be in the form of a voucher that can be applied to things such as scholarships, education, housing or a donation to a charity.
While the decision applies only to the nine states covered by the Ninth Circuit court, and only to bone marrow obtained through apheresis, it does raise bigger questions about how we will look at organ donation in the future. With about 114,000 people waiting for organs in the U.S. alone on any given day, and only 3,300 donors, the urgent medical need runs up against moral standards of the value human life. Once we start paying for the parts we need, though, how far do we go? We dont allow people to buy and sell human beings, thats slavery, says Dr. Robert Klitzman, director of the bioethics program at Columbia University. Should we allow people to buy and sell human body parts?
(MORE: Where Do (Some) Babies Come From? In Washington, a New Law Bans Anonymous Sperm and Egg Donors)
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A Court Allows Payment for Bone Marrow. Should People Be Able to Sell Their Parts?
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Paying for Bone Marrow: Should We Be Able to Sell Our Body Parts?
Ed Reschke / Getty Images
A color-enhanced photograph of spongy (Cancellous) bone red bone marrow fills the space.
How much would it take for you to consider selling your bone marrow? A U.S. appeals court puts the price at about $3,000 in a ruling that now makes it legal to pay donors for their bone marrow tissue.
The courts decision may well help thousands of sick patients who need bone marrow transplants to survive, but it also begs the question, what other body parts might next be up for sale?
The ruling came about at the end of 2011, in a decision to an October 2009 lawsuit brought by a group of cancer patients, parents and bone marrow donation advocates against the government over the federal law banning the buying and selling of bodily organs. The plaintiffs were led by Doreen Flynn, who has three daughters who suffer from Fanconi anemia, a blood disorder that requires bone marrow transplants to treat. Flynn and the other plaintiffs said that too many such patients die waiting for transplants and argued that we should be allowed to pay people to donate their marrow as a way of ensuring a more reliable supply. The U.S. Court of Appeals for the Ninth Circuit agreed.
(MORE: Facebook Now Lets Organ Donors Tell Their Friends)
At the core of the plaintiffs argument was the National Organ Transplantation Act (NOTA), which since 1984 has forbid the buying and selling of human organs, including bone marrow. But new developments in bone marrow extraction have made marrow donation not much different from donating blood: traditionally, bone marrow donation required anesthesia and long needles to extract the marrow from the hipbones of donors. Now, a technique called peripheral apheresis allows doctors to extract blood stem cells directly from the blood, instead of the bone patients first take a drug that pulls stem cells from the bone and into the blood meaning that the marrow cells should be considered a fluid like blood, rather than an organ, the plaintiffs argued. NOTA doesnt prohibit payments for blood or other fluids, such as plasma or semen.
U.S. Attorney General Eric Holder decided not to ask the U.S. Supreme Court to review the appellate courts decision, which would have been the next step in overturning it. That means the ruling stands and that people can now be paid up to $3,000 for their marrow, as long as it is collected by apheresis. In a concession to the spirit of NOTA, however, the compensation cant be in cash; it needs to be in the form of a voucher that can be applied to things such as scholarships, education, housing or a donation to a charity.
While the decision applies only to the nine states covered by the Ninth Circuit court, and only to bone marrow obtained through apheresis, it does raise bigger questions about how we will look at organ donation in the future. With about 114,000 people waiting for organs in the U.S. alone on any given day, and only 3,300 donors, the urgent medical need runs up against moral standards of the value human life. Once we start paying for the parts we need, though, how far do we go? We dont allow people to buy and sell human beings, thats slavery, says Dr. Robert Klitzman, director of the bioethics program at Columbia University. Should we allow people to buy and sell human body parts?
(MORE: Where Do (Some) Babies Come From? In Washington, a New Law Bans Anonymous Sperm and Egg Donors)
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Paying for Bone Marrow: Should We Be Able to Sell Our Body Parts?
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SONA: Stem cell therapy, kaya raw makapagpabata ng pangangatawan – Video
02-07-2012 09:43 State of the Nation is a nightly newscast anchored by award-winning broadcast journalist, Jessica Soho. It airs Mondays to Fridays at 9:00 PM (PHL Time) on GMA News TV Channel 11. For more videos from State of the Nation, visit fthenation.
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SONA: Stem cell therapy, kaya raw makapagpabata ng pangangatawan - Video
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Generating dopamine via cell therapy for Parkinson's disease
Public release date: 2-Jul-2012 [ | E-mail | Share ]
Contact: Sarah Jackson press_releases@the-jci.org Journal of Clinical Investigation
In Parkinson's disease, the loss of dopamine-producing cells in the midbrain causes well-characterized motor symptoms. Though embryonic stem cells could potentially be used to replace dopaminergic (DA) neurons in Parkinson's disease patients, such cell therapy options must still overcome technical obstacles before the approach is ready for the clinic. Embryonic stem cell-based transplantation regimens carry a risk of introducing inappropriate cells or even cancer-prone cells. To develop cell purification strategies to minimize these risks, Dr. Lorenza Studer and colleagues at Memorial Sloan Kettering Cancer Center in New York developed three different mouse lines to fluorescently label dopaminergic neurons at early, mid, and late stages of differentiation. Their data suggest that mouse embryonic stem cells induced to the mid stage of neuronal differentiation are best suited for transplantation to replace dopaminergic neurons. Further, their work identified new genes associated with each stage of neuronal differentiation. Their results in the mouse model system help define the differentiation stage and specific attributes of embryonic stem cell-derived, dopamine-generating cells that hold promise for cell therapy applications.
###
TITLE:
Identification of embryonic stem cellderived midbrain dopaminergic neurons for engraftment
AUTHOR CONTACT:
Lorenz Studer
Memorial Sloan Kettering Cancer Center, New York, NY, USA
Phone: 212.639.6126; E-mail: studerl@mskcc.org
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Generating dopamine via cell therapy for Parkinson's disease
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Identifying athletic genes matters to Kathryn North – Video
01-07-2012 19:52 Does identifying athletic genes matter to you? Professor Kathryn North's research led to the discovery of the 'gene for speed'. Learn more and have your say at
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Identifying athletic genes matters to Kathryn North - Video
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Breaking the skin barrier
Public release date: 2-Jul-2012 [ | E-mail | Share ]
Contact: Marla Paul marla-paul@northwestern.edu 312-503-8928 Northwestern University
EVANSTON, Ill. --- Getting under your skin takes on a brave new meaning thanks to Northwestern University research that could transform gene regulation.
A team led by a physician-scientist and a chemist -- from the fields of dermatology and nanotechnology -- is the first to demonstrate the use of commercial moisturizers to deliver gene regulation technology that has great potential for life-saving therapies for skin cancers.
The topical delivery of gene regulation technology to cells deep in the skin is extremely difficult because of the formidable defenses skin provides for the body. The Northwestern approach takes advantage of drugs consisting of novel spherical arrangements of nucleic acids. These structures, each about 1,000 times smaller than the diameter of a human hair, have the unique ability to recruit and bind to natural proteins that allow them to traverse the skin and enter cells.
Applied directly to the skin, the drug penetrates all of the skins layers and can selectively target disease-causing genes while sparing normal genes. Once in cells, the drug simply flips the switch of the troublesome genes to off.
A detailed study of a method that could dramatically redefine the field of gene regulation will be published online during the week of July 2 by the Proceedings of the National Academy of Sciences (PNAS).
Early targets of the novel treatment are melanoma and squamous cell carcinoma (two of the most common types of skin cancer), the common inflammatory skin disorder psoriasis, diabetic wound healing and a rare genetic skin disorder that has no effective treatment (epidermolytic ichthyosis). Other targets could even include wrinkles that come with aging skin.
The technology developed by my collaborator Chad Mirkin and his lab is incredibly exciting because it can break through the skin barrier, said co-senior author Amy S. Paller, M.D., the Walter J. Hamlin Professor, chair of dermatology and professor of pediatrics at Northwestern University Feinberg School of Medicine. She also is director of Northwesterns Skin Disease Research Center.
This allows us to treat a skin problem precisely where it is manifesting -- on the skin, she said. We can target our therapy to the drivers of disease, at a level so minute that it can distinguish mutant genes from normal genes. Risks are minimized, and side effects have not been seen to date in our human skin and mouse models.
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Breaking the skin barrier
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Gene healing in a lotion? Researchers are close
By Brian Alexander
Most people who buy cosmetic lotions and potions know that while the people working behind the department store makeup counters may wear white lab coats, the stuff they sell is more about packaging than science.
But a Northwestern University team is bucking that image, reporting today that theyve created a way to regulate genes affecting the skin -- merely by applying moisturizer.
Not only could their technology pave the way for cosmetics that actually work, but it also might also prove to be a valuable weapon in fighting melanoma, the deadliest form of skin cancer, or diseases like psoriasis, and wounds like the intractable sores that often plague diabetics.
This is a blockbuster in the ways we will treat diseases of the skin, saidChad Mirkin, director of the International Institute for Nanotechnology and the George B. Rathmann Professor of Chemistry at Northwestern said. Were talking about ailments, scarring, wound healing, ways of regulating them or retarding them.
In a research paper published today in the Proceedings of the National Academy of Sciences, Mirkin and his colleagues describe not a drug, exactly, but a way of delivering small sections of nucleic acids (DNA and RNA are nucleic acids) called short interfering RNA, or siRNA, to cells. The cells take up the siRNA, which then alters the way a gene inside each cell can be read by the protein-making system.
The team used gold particles with a diameter of 13 nanometers. (One nanometer is 1-billionth of a meter. A typical strand of human hair is roughly 60,000 nanometers wide.) They coated the particles with siRNA to create what they call spherical nucleic acid nanoparticleconjugates, or SNAs. Millions of SNAs were then added to a commercially available petroleum-based skin moisturizer and the mixture was applied to mice and to lab-grown human skin.
In their key experiment in mice, they used their new system to tamp down the activity of a gene called epidermal growth factor receptor, or EGFR, thats involved in the growth of melanoma. As its name implies, EGFR receives messages from the epidermal growth factor protein. So toning down EGFR will interrupt the message; growth will be reduced or stop.
After mice were treated with the mixture three times per week for three weeks, the expression of the EGFR gene was reduced by 65 percent.
'Impressive' resultsSteve Dowdy, professor of cellular and molecular medicine at the University of California San Diego, and a Howard Hughes Medical Institute investigator specializing in RNA inhibition and ways to deliver siRNAs, called that result impressive.
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Gene healing in a lotion? Researchers are close
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James A. Shapiro: Experimental Evolution II: More Ways to Watch Natural Genetic Engineering in Real Time
In February last year, The New York Times published an interview with my University of Chicago colleague Janet Rowley. Janet is deservedly famous for finding a repeated chromosome rearrangement in certain types of leukemia. This was one of the earliest indications that genome changes in cancer cells do not occur randomly.
In the interview, Janet explained how she discovered this particular chromosome change, now called the "Philadelphia Chromosome." She was just looking through the microscope, motivated by her curiosity to know more about these tumor cells.
Janet pointed out that she might well not be able to repeat her discovery in today's scientific environment. She was practicing what she called "observationally driven research." Today, she said, granting agencies don't support that kind of work. "That's the kiss of death if you're looking for funding today. We're so fixated now on hypothesis-driven research that if you do what I did, it would be called a 'fishing expedition,' a bad thing."
In other words, you have to know what kind of result to expect before the funding agencies will give you money to look for it. Surprises are not fundable. But "surprise" is just another word for "discovery." As Janet put it, "I keep saying that fishing is good. You're fishing because you want to know what's there."
Let's look at how we would "fish" for complex genomic novelty through natural genetic engineering. I can think of two approaches. There will definitely turn out to be more.
One approach was included in my book. The idea was to do interspecific hybridization with a well-characterized organism, like the mustard weed Arabidopsis, and follow what happens with the genetically unstable hybrid progeny.
We know that interspecific hybridization and genome duplication lead to high levels of genomic and phenotypic variation. DNA sequencing has found evidence of genome duplication at many critical points of evolutionary divergence, especially in plants. There is a fine Scientific American article by the famous 20th-century evolutionist G. Ledyard Stebbins entitled "Cataclysmic Evolution," which describes how hybridization between two wild grasses can recreate the origin of flour wheat.
The hybrid progeny can be followed, and those plants that develop significant new traits, such as flower patterns, can then be analyzed. Sequencing the whole Arabidopsis genome in a short time is now feasible. The sequence data will let the Arabidopsis genome speak for itself in telling us how the new traits evolved.
We can then look for multiple changes that show signs of coordination in the underlying natural genetic engineering events. Such coordinated events might be insertions of the same or related mobile elements at distinct locations in the genome or the addition of the same domains to more than one protein in the network responsible for development of the novel trait.
The second "fishing" approach to asking how a novel feature can evolve would use a microbe, as suggested in the previous blog on experimental evolution. In this case, however, the changes would not be pre-targeted to a number of different sites in the genome.
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James A. Shapiro: Experimental Evolution II: More Ways to Watch Natural Genetic Engineering in Real Time
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Gene Mutations Associated With Enlarged Brain Size, Disorders
July 2, 2012
Lawrence LeBlond for redOrbit.com Your Universe Online
At least three genetic mutations found in the human brain have been linked to enlarged brain size (megalencephaly) and a number of disorders, including cancer, epilepsy and autism, according to new research led by Seattle Childrens Research Institute.
The mutations were found in the genes AKT3, PIK3R2 and PIK3CA. The mutations were also linked to vascular disorders and skin growth disorders, said the researchers. The study, published in the online edition of the journal Nature Genetics on June 24, offers important implications for the future of medicine through the research findings.
Study leaders, geneticist William Dobyns, MD, and Jean-Baptiste Rivire, PhD, discovered through their research additional proof that the genetic makeup of a person is not completely determined at the moment of conception. The new evidence ties in with previous research that recognized that genetic changes can occur after conception, although considered quite rare.
The researchers also discovered the genetic causes of these human diseases, including developmental disorders, may also directly lead to new possibilities for treatment.
AKT3, PIK3R2 and PIK3CA are found in all humans, but only when they are mutated do they lead to the diseases and disorders. PIK3CA is known as a cancer-related gene, and appears to make cancer more aggressive. Boston Childrens Hospital researchers recently found a common link between the PIK3CA gene and a rare condition known as CLOVES syndrome.
James Olson, MD, PhD, a pediatric cancer expert at Seattle Childrens and Fred Hutchinson Cancer Research Center acknowledged the two decades-worth of work that led to the findings.
This study represents ideal integration of clinical medicine and cutting-edge genomics, said Olson, who was not involved in the latest research. I hope and believe that the research will establish a foundation for successfully using drugs that were originally developed to treat cancer in a way that helps normalize intellectual and physical development of affected children.
He noted that the team did an excellent job by deep sequencing exceptionally rare familial cases and unrelated cases to identify the culprit pathway. He further noted that the three genes all encode core components of the phosphatidylinositol-3-kinase/AKT pathway, the culprit pathway, as referenced by his work.
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Gene Mutations Associated With Enlarged Brain Size, Disorders
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Moffitt Cancer Center Study Validates Activity of Rare Genetic Variant in Glioma
Newswise Researchers at Moffitt Cancer Center working with colleagues at three other institutions have validated a link between a rare genetic variant and the risk of glioma, the most common and lethal type of brain tumor. The validation study also uncovered an association between the same rare genetic variant and improved rates of survival for patients with glioma.
The study, the first to confirm a rare susceptibility variant in glioma, appeared in a recent issue of the Journal of Medical Genetics, a journal published by the British Medical Association.
"Glioma is a poorly understood cancer with high morbidity and devastating outcomes," said study lead author Kathleen M. Egan, Sc.D., interim program leader of Cancer Epidemiology and vice chair of the Department of Cancer Epidemiology. "However, the discovery of the association of the TP53 genetic variant rs78378222 with glioma provides new insights into these tumors and offers better prospects for identifying people at risk."
According to the authors, their study "genotyped' the single nucleotide polymorphism (SNP, or "snip") rs78378222 in TP53, an important tumor suppressor gene. The researchers said the SNP disrupts the TP53 signal and, because of its activity, has been linked to a variety of cancers. This study linked the presence of the rare form of rs78378222 to deadly glioma.
The researchers conducted a large, clinic-based, case-control study of individuals age 18 and older with a recent glioma diagnosis. A total of 566 glioma cases and 603 controls were genotyped for the rs78378222 variant.
Study results reveal that the odds of developing glioma were increased 3.5 times among the rare variant allele carriers. However, when researchers examined the impact of rs78378222 on survival, they found an approximately 50 percent reduction in death rates for those who were variant allele carriers.
"That the variant increased survival chances was an unexpected finding," Egan said. "It is tempting to speculate that the presence of the risk allele could direct tumor development into a less aggressive path."
The researchers concluded that their study results "may shed light on the etiology and progression of these tumors."
In addition to researchers from Moffitt, researchers from The University of Alabama at Birmingham, Emory School of Medicine and Vanderbilt University participated in the study and co-authored the paper.
The study was supported by funding from Public Health Service Grants R01CA11674 from the National Cancer Institute and the U.S. Department of Health and Human Services, as well as institutional funding from Moffitt and the Vanderbilt-Ingram Cancer Center.
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Moffitt Cancer Center Study Validates Activity of Rare Genetic Variant in Glioma
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Activity of rare genetic variant in glioma validated
ScienceDaily (July 2, 2012) Researchers at Moffitt Cancer Center working with colleagues at three other institutions have validated a link between a rare genetic variant and the risk of glioma, the most common and lethal type of brain tumor. The validation study also uncovered an association between the same rare genetic variant and improved rates of survival for patients with glioma.
The study, the first to confirm a rare susceptibility variant in glioma, appeared in a recent issue of the Journal of Medical Genetics, a journal published by the British Medical Association.
"Glioma is a poorly understood cancer with high morbidity and devastating outcomes," said study lead author Kathleen M. Egan, Sc.D., interim program leader of Cancer Epidemiology and vice chair of the Department of Cancer Epidemiology. "However, the discovery of the association of the TP53 genetic variant rs78378222 with glioma provides new insights into these tumors and offers better prospects for identifying people at risk."
According to the authors, their study "genotyped' the single nucleotide polymorphism (SNP, or "snip") rs78378222 in TP53, an important tumor suppressor gene. The researchers said the SNP disrupts the TP53 signal and, because of its activity, has been linked to a variety of cancers. This study linked the presence of the rare form of rs78378222 to deadly glioma.
The researchers conducted a large, clinic-based, case-control study of individuals age 18 and older with a recent glioma diagnosis. A total of 566 glioma cases and 603 controls were genotyped for the rs78378222 variant.
Study results reveal that the odds of developing glioma were increased 3.5 times among the rare variant allele carriers. However, when researchers examined the impact of rs78378222 on survival, they found an approximately 50 percent reduction in death rates for those who were variant allele carriers.
"That the variant increased survival chances was an unexpected finding," Egan said. "It is tempting to speculate that the presence of the risk allele could direct tumor development into a less aggressive path."
The researchers concluded that their study results "may shed light on the etiology and progression of these tumors."
In addition to researchers from Moffitt, researchers from The University of Alabama at Birmingham, Emory School of Medicine and Vanderbilt University participated in the study and co-authored the paper.
The study was supported by funding from Public Health Service Grants R01CA11674 from the National Cancer Institute and the U.S. Department of Health and Human Services, as well as institutional funding from Moffitt and the Vanderbilt-Ingram Cancer Center.
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Activity of rare genetic variant in glioma validated
Recommendation and review posted by Bethany Smith
Stem-cell research leaders to meet in NUIG
The Irish Times - Monday, July 2, 2012
LORNA SIGGINS
WORLD leaders in stem-cell technology are due to exchange knowledge of potential treatments at a conference opening in NUI Galway today.
Researchers from NUIG, University College Cork and NUI Maynooth will participate in the event, which has been billed as the first major conference on stem-cell therapy in Ireland.
Prof Anthony Hollander of the University of Bristol, England who was one of a team which successful created and then transplanted the first tissue-engineered trachea or windpipe is among a number of international speakers presenting findings.
The gathering will focus on the realities of stem-cell treatment, Prof Frank Barry, director of NUIGs National Centre for Biomedical Engineering Science has said.
The therapy is complex and controversial, and sometimes exaggerated claims are made, he said.
The researchers are specialists in Mesenchymal, or adult, stem cells, and will be concentrating on what is likely in the future, he added.
The list of conditions which could be treated successfully by stem cells is small, but growing, Prof Barry said.
Leukaemia and other diseases of the blood appear to respond best.
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Stem-cell research leaders to meet in NUIG
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ThermoGenesis Announces Sale Of CryoSeal® Product Line To Asahi
RANCHO CORDOVA, Calif., July 2, 2012 /PRNewswire/ -- ThermoGenesis Corp. (KOOL), a leading supplier of enabling technologies for the processing, storage and administration of cell therapies, said today that Asahi Kasei Medical Co., Ltd., has exercised its option to purchase the Company's CryoSeal Fibrin Sealant System wound care product line for $2 million in cash, effective June 30, 2012. Payment is due during the Company's first fiscal quarter of 2013.
This transaction is the culmination of the Company's previously announced strategy and option agreement to divest the CryoSeal product line, according to Matthew Plavan, Chief Executive Officer of ThermoGenesis. "We are very pleased to consummate the sale of the CryoSeal product line to our long-time strategic partner, Asahi. We look forward to their successful commercialization of CryoSeal in the wound care market," Plavan said.
"This divestiture is an important milestone in our long-term strategy to focus on the development of enabling technologies for the stem cell regenerative medicine market. It significantly strengthens our balance sheet and the proceeds from this transaction will be used to fund our market expansion efforts for our cord blood and bone marrow stem cell processing and storage offerings, particularly in markets outside North America. In addition, it frees up management and corporate resources to address these more strategic market opportunities, and furthers our initiative to reduce operating costs," Plavan added.
About ThermoGenesis Corp.ThermoGenesis Corp. (www.thermogenesis.com)is a leader in developing and manufacturing automated blood processing systems and disposable products that enable the manufacture, preservation and delivery of cell and tissue therapy products. These include:
This press release contains forward-looking statements. These statements involve risks and uncertainties that could cause actual outcomes to differ materially from those contemplated by the forward-looking statements. Several factors including timing of FDA and foreign regulatory approvals, changes in customer forecasts, our failure to meet customers' purchase order and quality requirements, supply shortages, production delays, changes in the markets for customers' products, introduction timing and acceptance of our new products scheduled for fiscal years 2012 and 2013, and introduction of competitive products and other factors beyond our control could result in a materially different revenue outcome and/or in our failure to achieve the revenue levels we expect for fiscal 2012 and 2013. A more complete description of these and other risks that could cause actual events to differ from the outcomes predicted by our forward-looking statements is set forth under the caption "Risk Factors" in our annual report on Form 10-K and other reports we file with the Securities and Exchange Commission from time to time, and you should consider each of those factors when evaluating the forward-looking statements.
ThermoGenesis Corp.Web site: http://www.thermogenesis.comContact: Investor Relations+1-916-858-5107, orir@thermogenesis.com
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ThermoGenesis Announces Sale Of CryoSeal® Product Line To Asahi
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ACT Announces Second Patient with Stargardt’s Disease Treated in EU Clinical Trial
MARLBOROUGH, Mass.--(BUSINESS WIRE)--
Advanced Cell Technology, Inc. (ACT; OTCBB: ACTC), a leader in the field of regenerative medicine, today announced treatment of the second patient in its Phase 1/2 clinical trial for Stargardts macular dystrophy (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The surgery was performed on Friday, June 29 at Moorfields Eye Hospital in London, the same site as the first patient treatment in January, by a team of surgeons led by Professor James Bainbridge, consultant surgeon at Moorfields and Chair of Retinal Studies at University College London. The procedure was successfully performed without any complications. ACT and Moorfields Eye Hospital recently received clearance from the Data and Safety Monitoring Board (DSMB) to treat the final two patients in the first cohort of this clinical trial.
We are very pleased to continue our forward momentum with both our U.S. trials and our European trial, commented Gary Rabin, chairman and CEO. It was less than a month ago that we received DSMB approval to treat the second and third patients in our E.U. trial, and it is very gratifying to have already completed dosing of the second. It is a pleasure to be working with Professor Bainbridge and the rest of his team at Moorfields Eye Hospital, and we continue to be encouraged by the steady progress of the trial thus far.
The Phase 1/2 trial is designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation in patients with SMD at 12 months, the studys primary endpoint. It will involve a total of 12 patients, with cohorts of three patients each in an ascending dosage format. It is similar in design to the U.S. trial for SMD that was initiated in July 2011.
The European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) has officially designated ACT's human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cells as an orphan medicinal product for the treatment of Stargardt's Macular Dystrophy (SMD).
More information on the status of the companys clinical trials will be posted today on Mr. Rabins Chairmans blog.
About Stargardts Disease Stargardts disease or Stargardts Macular Dystrophy is a genetic disease that causes progressive vision loss, usually starting in children between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, called the retinal pigment epithelium, which is the site of damage that the company believes the hESC-derived RPE may be able to target for repair after administration.
About Advanced Cell Technology, Inc. Advanced Cell Technology, Inc. is a biotechnology company applying cellular technology in the field of regenerative medicine. For more information, visit http://www.advancedcell.com.
Forward-Looking Statements Statements in this news release regarding future financial and operating results, future growth in research and development programs, potential applications of our technology, opportunities for the company and any other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words will, believes, plans, anticipates, expects, estimates, and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements, including: limited operating history, need for future capital, risks inherent in the development and commercialization of potential products, protection of our intellectual property, and economic conditions generally. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in the companys periodic reports, including the report on Form 10-K for the year ended December 31, 2011. Forward-looking statements are based on the beliefs, opinions, and expectations of the companys management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. Forward-looking statements are based on the beliefs, opinions, and expectations of the companys management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. There can be no assurance that the Companys clinical trials will be successful.
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ACT Announces Second Patient with Stargardt’s Disease Treated in EU Clinical Trial
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Prochymal Significantly Reduces Hypertrophy, Arrhythmia and Progression to Heart Failure in Patients Suffering a Heart …
COLUMBIA, Md.--(BUSINESS WIRE)--
Osiris Therapeutics, Inc. (OSIR), announced today interim one-year results from its groundbreaking clinical trial evaluating Prochymal (remestemcel-L) for the treatment of patients experiencing first-time acute myocardial infarction. The trial is the largest study of allogeneic or "off-the-shelf" stem cells ever conducted in heart attack patients. A total of 220 patients were given a single infusion of either Prochymal or placebo through a standard intravenous line within seven days of an acute heart attack.
Cardiac MRI assessments were conducted for six months following infarct to evaluate cardiac remodeling. Patients receiving Prochymal had significantly less cardiac hypertrophy, as measured by cardiac MRI, compared to patients receiving placebo (p<0.05). Patients treated with Prochymal also experienced significantly less stress-induced ventricular arrhythmia (p<0.05). Cardiac hypertrophy and ventricular arrhythmia are indicators of pathological remodeling following heart injury and provide insight into the mechanism by which mesenchymal stem cells attenuate heart injury following a myocardial infarction.
The mechanistic data is complemented by clinical data showing treatment with Prochymal resulted in a statistically significant reduction in heart failure. In the study, seven patients who were treated with placebo have progressed to heart failure requiring treatment with intravenous diuretics, compared to none of the Prochymal patients (p=0.01). Furthermore, patients receiving placebo tended to require re-hospitalization for cardiac issues sooner than the patients receiving Prochymal (median 27.5 days vs. 85.5 days).
This study is the largest of its kind and provides key insights into the mechanism of action of mesenchymal stem cells in the setting of acute myocardial infarction, said Lode Debrabandere, Ph.D., Senior Vice President of Therapeutics at Osiris. These important mechanistic observations are consistent with data obtained from our preclinical models and from the first placebo-controlled human trial with Prochymal published in the Journal of the American College of Cardiology. Given the quality of the data and highly encouraging results observed thus far, we are extending the trial's duration to capture a better understanding of the long-term clinical benefits of MSCs."
The trial also demonstrated that treatment with Prochymal was safe. There were no infusional toxicities observed in patients receiving Prochymal. Serious adverse events occurred with equal frequency in both treatment groups (31.8%). To date, there have been 5 deaths in the trial, 2 in the Prochymal group and 3 in the placebo group.
For interventional cardiologists, keeping our myocardial infarction patients from progressing to heart failure is central to our mission, said Mark Vesely, M.D., Principal Investigator on the Study and Assistant Professor of Medicine (Interventional Cardiology) at the University of Maryland School of Medicine. It is remarkable and very encouraging to see significant changes in clinically meaningful parameters this early in the study. We look forward to the additional data that will be gathered as the study progresses, which will help us to better understand both the magnitude and durability of the benefit to treatment.
Prochymal, the worlds first and only stem cell drug approved by an internationally recognized regulatory authority, is used for the treatment of graft vs. host disease (GvHD). GvHD is a devastating complication of bone marrow transplantation that kills up to 80 percent of children affected. Prochymal is now approved in Canada and New Zealand, and is currently available in seven other countries including the United States under an Expanded Access Program (EAP).
About the Trial
This Phase 2, multi-center, randomized, double-blind, placebo-controlled study is evaluating the safety and efficacy of Prochymal (ex-vivo cultured adult human mesenchymal stem cells) intravenous infusion following acute myocardial infarction. A total of 220 patients were randomized (1:1) at 33 centers in the United States and Canada and received a single intravenous infusion of Prochymal or placebo within 7 days following first acute myocardial infarction. In addition to screening and baseline visits prior to the infusion, initially follow-up evaluations were scheduled to be conducted through 2 years. Given the encouraging results observed at the one year time-point, the trial is being extended to include 5 years of follow-up. Both male and female subjects between 21 and 85 years of age were enrolled. Patients had to have a left ventricular ejection fraction (LVEF) between 20% and 45% as determined by quantitative echocardiography or cardiac MRI at least 24 hours after successful reperfusion of the culprit vessel. In addition, troponin levels must have been greater than 4 times the upper limit of normal during the first 72 hours of hospitalization for the MI.
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Prochymal Significantly Reduces Hypertrophy, Arrhythmia and Progression to Heart Failure in Patients Suffering a Heart ...
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Myelodysplastic syndromes (MDS) linked to abnormal stem cells
Public release date: 2-Jul-2012 [ | E-mail | Share ]
Contact: Kim Newman sciencenews@einstein.yu.edu 718-430-3101 Albert Einstein College of Medicine
July 2, 2012 -- (Bronx, NY) -- Researchers at Albert Einstein College of Medicine of Yeshiva University have found that abnormal bone marrow stem cells drive the development of myelodysplastic syndromes (MDS), serious blood diseases that are common among the elderly and that can progress to acute leukemia. The findings could lead to targeted therapies against MDS and prevent MDS-related cancers. The study is published today in the online edition of the journal Blood.
"Researchers have suspected that MDS is a 'stem cell disease,' and now we finally have proof," said co-senior author Amit Verma, M.B.B.S., associate professor of medicine and of developmental and molecular biology at Einstein and attending physician in oncology at Montefiore Einstein Center for Cancer Care. "Equally important, we found that even after MDS standard treatment, abnormal stem cells persist in the bone marrow. So, although the patient may be in remission, those stem cells don't die and the disease will inevitably return. Based on our findings, it's clear that we need to wipe out the abnormal stem cells in order to improve cure rates."
MDS are a diverse group of incurable diseases that affect the bone marrow and lead to low numbers of blood cells. While some forms of MDS are mild and easily managed, some 25 to 30 percent of cases develop into an aggressive disease called acute myeloid leukemia. Each year, about 10,000 to 15,000 people in the U.S. are diagnosed with MDS, according to the National Marrow Donor Program.
Most cases of MDS occur in people over age 60, but the disease can affect people of any age and is more common in men than women. Symptoms vary widely, ranging from anemia to infections, fever and bleeding. Treatment usually involves chemotherapy to destroy abnormal blood cells plus supportive care such as blood transfusions.
In the current study, lead author Britta Will, Ph.D., research associate in the department of cell biology, and her colleagues analyzed bone marrow stem cells and progenitor cells (i.e., cells formed by stem cells) from 16 patients with various types of MDS and 17 healthy controls. The stem and progenitor cells were isolated from bone marrow using novel cell-sorting methods developed in the laboratory of co-senior author Ulrich Steidl, M.D., Ph.D., assistant professor of cell biology and of medicine and the Diane and Arthur B. Belfer Faculty Scholar in Cancer Research at Einstein.
Genome-wide analysis revealed widespread genetic and epigenetic alterations in stem and progenitor cells taken from MDS patients, in comparison to cells taken from healthy controls. The abnormalities were more pronounced in patients with types of MDS likely to prove fatal than in patients with lower-risk types.
"Our study offers new hope that MDS can be more effectively treated, with therapies that specifically target genes that are deregulated in early stem and progenitor cells," said Dr. Steidl. "In addition, our findings could help to detect minimal residual disease in patients in remission, allowing for more individualized treatment strategies that permanently eradicate the disease."
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Myelodysplastic syndromes (MDS) linked to abnormal stem cells
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Bioinformatics 'Cacophony', Genomics of Infectious Disease Research, Gene Patenting Wars Continue, More
Douglas Kell has been reappointed as chief executive and deputy chair of the UK Biotechnology and Biological Sciences Research Council, the Minister for Universities and Science David Willetts said this week.
Kell has held the top post at BBSRC since 2008, and before that he was director of the Manchester Centre for Integrative Systems Biology. He also has served as director of research at the Institute of Biological Sciences at the University of Aberystwyth, and he was a founding director of Aber Instruments. His research has included a range of topics including systems biology, analytical chemistry, and biochemical and data modeling.
The Institute for Systems Biology has appointed Robert Lipshutz to be chief business officer and senior VP for strategic partnerships. Lipshutz spent nearly two decades at Affymetrix in various roles focusing on business development, licensing, diagnostics, and emerging markets, and most recently as senior VP for corporate development.
Verinata Health CEO Caren Mason has resigned but will continue to provide the company with consultative services. Mason joined Verinata in November 2010. She was previously the president and CEO of Quidel, president and CEO of MiraMedica, CEO of eMed Technologies, and general manager of GE Healthcare. The firm plans to recruit a new CEO.
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Bioinformatics 'Cacophony', Genomics of Infectious Disease Research, Gene Patenting Wars Continue, More
Recommendation and review posted by Bethany Smith
Tegal Corporation to Acquire CollabRx, Inc.
PETALUMA, Calif.--(BUSINESS WIRE)--
Tegal Corporation (TGAL) today announced that it has signed a definitive agreement to acquire CollabRx, Inc., a privately held technology company in the rapidly growing market of interpretive content and data analytics for genomics-based medicine.
CollabRx offers cloud-based expert systems that provide clinically relevant interpretive knowledge to institutions, physicians, researchers and patients for genomics-based medicine in cancer and other diseases to inform health care decision making. With access to approximately 50 clinical and scientific advisors at leading academic institutions and a suite of tools and processes that combine artificial intelligence-based analytics with proprietary interpretive content, the company is well positioned to participate in the $300 billion value-added big data opportunity in the US health care market (as reported by McKinsey Global Institute), over half of which specifically targets areas in cancer and cancer genomics.1
The Chief Executive Officers of the two constituent companies, Thomas Mika of Tegal and James Karis of CollabRx, plan to serve as co-CEOs of the combined, publicly traded company, with headquarters in San Francisco, CA. Tegal entered into an employment agreement with Mr. Karis that will become effective at the closing, and Mr. Karis will also be appointed to Tegals Board of Directors. Tegal will continue to operate under its current name and ticker symbol for the time being, but plans to seek stockholder approval at its upcoming annual meeting in September 2012 for an amendment to its Certificate of Incorporation, changing its corporate name to CollabRx, Inc.
Originally founded in 2008 by Silicon Valley Internet pioneer Jay (Marty) Tenenbaum, CollabRx has developed clinical advisory networks, expert systems, proprietary tools and processes, and a pipeline of commercial data products and applications (apps) for cancer. CollabRx Therapy Finders, its first commercial product, provides sophisticated, credible, personalized, and actionable information to physicians and patients for rapidly determining which medical tests, therapies, and clinical trials may be considered in cancer treatment planning with a specific emphasis on the tumor genetic profile.
CollabRx Therapy Finders are web-based apps that serve as one type of user interface to access proprietary CollabRx content. CollabRx content is dynamically updated and organized in a knowledgebase that includes information on molecular diagnostics, medical tests, clinical trials, drugs, biologics and other information relevant for cancer treatment planning. Capturing how highly respected practicing physicians use this information in the clinical setting further refines the knowledgebase.
Upon the acquisitions closing, Tegal will issue an aggregate of 236,433 shares of common stock, representing 14% of Tegals total shares outstanding prior to the closing, to former CollabRx stockholders in exchange for 100% of the capital stock of CollabRx, Inc. Tegal and certain former CollabRx stockholders will enter into a Stockholders Agreement providing for, among other things, registration rights, transfer restrictions and voting and standstill agreements. Tegal also will assume $500,000 of existing CollabRx indebtedness through the issuance of 5-year promissory notes in substitution for outstanding notes previously issued by CollabRx. In addition, Tegal will grant a total of 368,417 RSUs and options as inducement grants to newly hired management and employees, all subject to four-year vesting and other restrictions.
Medicine is entering a new era of low cost genome sequencing and the proliferation of personalized treatments based on specific genetic mutations, said James Karis, CEO of CollabRx. With the technology platform and expert system leadership position that CollabRx has developed over the past few years, we believe that the new company is in a position to lead the market for accurate, credible and current genomic information in the cancer space. We are excited to be joining the Tegal management team in a well-resourced, publicly-traded entity.
This acquisition marks both the successful conclusion of a transition process and the beginning of a new chapter for Tegal Corporation, said Thomas Mika, Tegals Chairman, President and CEO. We are excited to help drive the rapid growth of this market while we meet a critical and consequential human need. This is a mission Tegals board has embraced wholeheartedly. I am very pleased to be working with James Karis as Co-CEO and fellow director, and look forward, along with the entire team at CollabRx and Tegal, to building a dynamic company in a new era of genomic medicine.
About Tegal
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Tegal Corporation to Acquire CollabRx, Inc.
Recommendation and review posted by Bethany Smith
Nature Biotechnology Publications Showcase Value of PacBio RS in De Novo Genome Assembly
MENLO PARK, Calif.--(BUSINESS WIRE)--
Two papers in Nature Biotechnology, both published online on July 1, 2012 highlight the unique value for de novo genome assembly provided by the PacBioRS High Resolution Genetic Analyzer from Pacific Biosciences of California, Inc. (PACB).
Due to the inherent limitations of commonly used short-read sequencing technologies, the genomes of very few species have been completely sequenced, or finished. PacBios single molecule, real-time (SMRT) technology offers very long reads that reduce the number of contiguous sequences, or contigs, to simplify and improve genome assembly. These multi-kilobase reads allow scientists to sequence through long repeat regions and to identify structural variation, which are common in genomes but not possible to resolve completely with short-read platforms. As a result, PacBio long reads can lead to final assemblies that matchand in some cases even exceedthe quality that previously counted as finished, approaching the gold standard of a perfect genome.
In the publication from Koren et al., titled Hybrid error correction and de novo assembly of single-molecule sequencing reads, the authors demonstrate a new pipeline for assembly of the parrot genome. Using PacBio long reads in combination with high-accuracy short reads and an updated version of Celera Assembler, they assembled for the first time regulatory regions of genes involved in vocal learning circuits. The hybrid reads represent the most complete assembled bird genome now available.
"Repetitive regions are the biggest impediment to all assembly algorithms and sequencing technologies as they introduce ambiguity in the reconstruction of the genome, said Sergey Koren, Ph.D., Scientist of Bioinformatics at the National Biodefense Analysis and Countermeasures Center. Using the long reads we have access to longer sequences, which increases the probability of spanning a repeat and leads to better assemblies at lower depths than short reads.
A separate publication from Bashir et al., titled A hybrid approach for the automated finishing of bacterial genomes, describes combining contigs from second-generation sequencing technologies with PacBio sequence data for the cholera strain responsible for the 2010 Haitian outbreak. The authors show that their hybrid assembly resolved complex regions with several repeats and suggest that the approach offers a solution for rapid identification and assembly of full microbial genomes.
The publication of these two studies is evidence of how our long-read technology is emerging as the gold standard for finishing genome assemblies and identifying, annotating and deciphering genomic structure, said Mike Hunkapiller, President and CEO at Pacific Biosciences. Moreover, investigators are finding creative ways to take advantage of the unique benefits provided by our SMRT sequencing and enabling scientific applications that are simply not possible with short-read sequencing platforms.
The Nature Biotechnology publication details are as follows:
For more information on de novo genome assembly with the PacBioRS, please visit our website at http://www.pacb.com/denovo.
About Pacific Biosciences
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Nature Biotechnology Publications Showcase Value of PacBio RS in De Novo Genome Assembly
Recommendation and review posted by Bethany Smith
The prevention of hereditary breast and ovarian cancer by PGD is 'feasible'
Public release date: 2-Jul-2012 [ | E-mail | Share ]
Contact: Christine Bauquis christine@eshre.eu 32-499-258-046 European Society of Human Reproduction and Embryology
Istanbul, 2 July 2012: Preimplantation genetic diagnosis (PGD) for the breast cancer genes BRCA1/2 is now feasible and established, with good success rates for those treated, according to investigators from the reproduction, oncology and genetics centres of the university hospitals of Maastricht and Brussels. The results follow a review of the largest number of PGD treatments for BRCA1/2 in Europe and were presented today at the annual meeting of ESHRE (European Society of Human Reproduction and Embryology) by Professor Willem Verpoest from the Centre for Reproductive Medicine at Vrije Universiteit Brussel, Belgium.
Behind his vote of confidence lie 145 PGD cycles for BRCA1/2 mutations performed in 70 couples at the two centres (a mean of 2.1 cycles per woman). Almost 60% of the mutation carriers were female, two-thirds with a BRCA1 mutation. Just over one quarter (26.2%) of female carriers had undergone a prophylactic bilateral mastectomy.
Following IVF, 717 embryos were found suitable for genetic analysis, and of these 43.1% were diagnosed as affected by the mutation, with 40.7% unaffected and thus suitable for transfer (the remainder had an abnormal genotype or the analysis was inconclusive). Hence, 62.1% of the PGD cycles led to fresh embryo transfer - with 3.6% transferred from one or two frozen-thawed unaffected embryos - resulting in 42 pregnancies in 40 women. Pregnancy rates were 41.4% per fresh embryo transfer and 23.1% per frozen. The overall pregnancy rate was 29%.
The series also included three cases of PGD on embryos previously cryopreserved for fertility preservation prior to chemotherapy, and these too resulted in two ongoing pregnancies.
Two female BRCA1 carriers were diagnosed with breast cancer within three months of the PGD treatment, despite breast screening shortly before treatment. One had a history of breast cancer, the other patient hadn't. The former patient went on to have healthy twins three years after the second breast surgery and chemotherapy, and following frozen/thawed embryo transfer.
So far, PGD for BRCA1 and BRCA2 gene mutations has been considered controversial. While most PGD procedures are indicated to remove completely the risk of inherited sex-linked and single-gene diseases (such as cystic fibrosis) in the children of affected couples, PGD for the breast cancer mutations cannot remove the risk completely - because the 10% background risk of breast cancer remains, even after PGD. Moreover, breast and ovarian cancers are usually of late onset, with prevention and therapeutic options constantly improving - so the chances of successful treatment, and many years of healthy life, are high.
Nor is breast cancer inevitable for a woman (or man) carrying a BRCA1/2 mutation. The controversy thus rests on the fact that a mutation in the BRCA genes increases susceptibility to breast or ovarian cancer, but does not reflect an inevitability for developing the diseases. However, with female carriers of a mutation in either gene having a lifetime risk of 60-80% for breast cancer, and a risk of 30-60% (BRCA1) or 5-20% (BRCA2) for ovarian cancer, many authorities have recognised the gravity of the risk and accepted a BRCA gene mutation as an indication for PGD.
So far, only five pregnancies after PGD for BRCA1/2 have been reported since the first was described in 2008.(1) The slow uptake reflects not just the controversial nature of the procedure, but also concerns over patient selection and the safety of hormonal stimulation for IVF in women at risk themselves of breast and ovarian cancers.
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The prevention of hereditary breast and ovarian cancer by PGD is 'feasible'
Recommendation and review posted by Bethany Smith
Interleukin Genetics Completes $3,000,000 Private Placement with Its Strategic Partner, Delta Dental Plan of Michigan
WALTHAM, Mass.--(BUSINESS WIRE)--
Interleukin Genetics,Inc. (ILIU) which is pioneering the use of genetic testing for the prevention and improved management of certain chronic conditions, today announced that it has completed a financing for $3,000,000 with Delta Dental Plan of Michigan, Inc. The investment consists of the purchase of 500,000 shares of the Companys Series B convertible preferred stock. The Company expects to receive net proceeds of approximately $2.7 million after deducting fees and expenses. Each share of Series B preferred stock is convertible into approximately 21.86 shares of common stock reflecting a conversion price of $0.2745 per share. Delta Dental will obtain one board seat effective immediately replacing one of the Series A directors. BTIG LLC acted as the exclusive placement agent for the transaction.
"We are extremely pleased by the vote of confidence this transaction demonstrates by our current strategic partner Delta Dental Plan of Michigan," said Lewis H. Bender, Chief Executive Officer of Interleukin Genetics, Inc. Delta Dental of Michigan has supported the companys efforts to advance our unique periodontal disease susceptibility risk test, PST. We believe this financing makes a strong statement about our companys value to our partners and to our future potential. As new members to our Board of Directors, we are looking forward to our new investors input on our strategic objectives and business planning.
About Interleukin Genetics
Interleukin Genetics, Inc. (ILIU) develops and markets a line of genetic tests under the Inherent Health and PST brands. The products empower individuals to prevent certain chronic conditions and manage their existing health and wellness through genetic-based insights with actionable guidance. Interleukin Genetics leverages its research, intellectual property and genetic panel development expertise in metabolism and inflammation to facilitate the emerging personalized healthcare market. The Company markets its tests through partnerships with health and wellness companies, healthcare professionals and other distribution channels. Interleukin Genetics flagship products include its proprietary PST genetic risk panel for periodontal disease and tooth loss susceptibility sold through dentists, and the Inherent Health Weight Management Genetic Test that identifies the most effective diet and exercise program for an individual based on genetics. Interleukin Genetics is headquartered in Waltham, Mass. and operates an on-site, state-of-the-art DNA testing laboratory certified under the Clinical Laboratories Improvements Act (CLIA). For more information please visit http://www.ilgenetics.com.
Dental Plan of Michigan
Delta Dental of Michigan is a leader in the dental insurance industry. The Company is part of a group of affiliated companies that collectively is among the largest and oldest group dental benefits providers in the United States, covering 8.6 million people and paying out $2.2 billion in dental benefits as of the end of 2011.
Certain statements contained herein are forward-looking statements.Because such statements include risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements.Factors that could cause actual results to differ materially from those expressed or implied by such forward-looking statements include, but are not limited to, those risks and uncertainties described in the Companys annual report on Form 10-K for the year ended December 31, 2011 and other filings with the Securities and Exchange Commission. The Company disclaims any obligation or intention to update these forward-looking statements.
Recommendation and review posted by Bethany Smith
Gene Therapy Market to 2018 – Product Development Slowed by Clinical Failures, Close Regulatory Surveillance and High …
NEW YORK, July 2, 2012 /PRNewswire/ --Reportlinker.com announces that a new market research report is available in its catalogue:
Gene Therapy Market to 2018 - Product Development Slowed by Clinical Failures, Close Regulatory Surveillance and High Compliance Standards
This report is built using data and information sourced from proprietary databases, primary and secondary research, and in-house analysis by GBI Research's team of industry experts.
Gene therapies are biological compounds, which modify or replace disease-causing genes. These therapies are the new therapeutic class aimed at treating diseases associated with genetic mutations. Gene therapy promises to provide new treatments for a large number of inherited and acquired diseases. The basic concept of gene therapy is to introduce a piece of genetic material into target cells, which will result in either a cure for the disease or a slowdown in the progression of the disease. It involves the transfer of a functional gene copy into specific cells of an individual in order to repair a faulty gene copy. It may be used to replace a defective gene, or to introduce a new gene to cure a condition.
For example, mutations in genes on the X chromosome lead to X chromosome-linked genetic diseases such as Duchenne muscular dystrophy and hemophilia. Since males have only one copy of the genes from this chromosome, there is no other normal copy available to fulfill a defective gene's function which is present on the X chromosome. If the normal copy of the mutated gene is delivered in the nucleus externally through a delivery agent, the cells can produce the normal gene products and the disease would be treated.
From a commercial perspective, there is a huge unmet need in oncology and autoimmune diseases, amongst others, that could further drive growth of the pharmaceutical and biotech industry. The unmet need is largely driven by the lack of efficacious and safe therapeutic products based on conventional pharmaceutical and biotech research. Gene therapy is a new therapeutic category that has the potential to satisfy this unmet need, especially considering how efficacious and safe this therapeutic category is expected to be.
GBI Research's analysis suggests that therapies developed using gene therapy technology can address the majority of the unmet needs prevailing in the current pharmaceutical market. The inherent structure of gene therapies and their potential to replace the functions of defective genes make them highly effective to knockdown any gene that was previously unapproachable by conventional therapies. Gene therapies are poised to become the next most promising class of drugs in the pharmaceutical industry. Currently there are only three approved products, namely Gendicine, Oncorine and Rexin-G, with a collective market little above $2.8m. Since first movers always have the competitive edge, many large pharmaceutical and biotechnology companies have already commenced their R&D activities on gene therapies.
This report provides insights into the major unmet needs prevailing in the current pharmaceutical industry, and points to gene therapies as the solution to these unmet needs. The report also elucidates the promising late-stage gene therapy pipeline, and provides insights into the gene therapeutics R&D pipeline and funding opportunities.
- Analysis of the leading therapeutic segments for which clinical development in gene therapy is being conducted.
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Gene Therapy Market to 2018 - Product Development Slowed by Clinical Failures, Close Regulatory Surveillance and High ...
Recommendation and review posted by Bethany Smith