Gene mutations cause massive brain asymmetry

ScienceDaily (June 24, 2012) Hemimegalencephaly is a rare but dramatic condition in which the brain grows asymmetrically, with one hemisphere becoming massively enlarged. Though frequently diagnosed in children with severe epilepsy, the cause of hemimegalencephaly is unknown and current treatment is radical: surgical removal of some or all of the diseased half of the brain.

In a paper published in the June 24, 2012 online issue of Nature Genetics, a team of doctors and scientists, led by researchers at the University of California, San Diego School of Medicine and the Howard Hughes Medical Institute, say de novo somatic mutations in a trio of genes that help regulate cell size and proliferation are likely culprits for causing hemimegalencephaly, though perhaps not the only ones.

De novo somatic mutations are genetic changes in non-sex cells that are neither possessed nor transmitted by either parent. The scientists' findings -- a collaboration between Joseph G. Gleeson, MD, professor of neurosciences and pediatrics at UC San Diego School of Medicine and Rady Children's Hospital-San Diego; Gary W. Mathern, MD, a neurosurgeon at UC Los Angeles' Mattel Children's Hospital; and colleagues -- suggest it may be possible to design drugs that inhibit or turn down signals from these mutated genes, reducing or even preventing the need for surgery.

Gleeson's lab studied a group of 20 patients with hemimegalencephaly upon whom Mathern had operated, analyzing and comparing DNA sequences from removed brain tissue with DNA from the patients' blood and saliva.

"Mathern had reported a family with identical twins, in which one had hemimegalencephaly and one did not. Since such twins share all inherited DNA, we got to thinking that there may be a new mutation that arose in the diseased brain that causes the condition," said Gleeson. Realizing they shared the same ideas about potential causes, the physicians set out to tackle this question using new exome sequencing technology, which allows sequencing of all of the protein-coding exons of the genome at the same time.

The researchers ultimately identified three gene mutations found only in the diseased brain samples. All three mutated genes had previously been linked to cancers.

"We found mutations in a high percentage of the cells in genes regulating the cellular growth pathways in hemimegalencephaly," said Gleeson. "These same mutations have been found in various solid malignancies, including breast and pancreatic cancer. For reasons we do not yet understand, our patients do not develop cancer, but rather this unusual brain condition. Either there are other mutations required for cancer propagation that are missing in these patients, or neurons are not capable of forming these types of cancers."

The mutations were found in 30 percent of the patients studied, indicating other factors are involved. Nonetheless, the researchers have begun investigating potential treatments that address the known gene mutations, with the clear goal of finding a way to avoid the need for surgery.

"Although counterintuitive, hemimegalencephaly patients are far better off following the functional removal or disconnection of the enlarged hemisphere," said Mathern. "Prior to the surgery, most patients have devastating epilepsy, with hundreds of seizures per day, completely resistant to even our most powerful anti-seizure medications. The surgery disconnects the affected hemisphere from the rest of the brain, causing the seizures to stop. If performed at a young age and with appropriate rehabilitation, most children suffer less language or cognitive delay due to neural plasticity of the remaining hemisphere."

But a less-invasive drug therapy would still be more appealing.

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Gene mutations cause massive brain asymmetry

uniQure Collaborates with UCSF on GDNF Gene Therapy in Parkinson's Disease

AMSTERDAM, June 21, 2012 /PRNewswire/ --

uniQure, a leader in the field of human gene therapy, announced today the signing of a collaborative agreement with two leading neurology experts to develop further a gene therapy incorporating uniQure's GDNF (glial cell derived neurotrophic factor) gene for the treatment of Parkinson's disease.

Professor Krystof Bankiewicz at the University of California, San Francisco (UCSF), a world expert in GDNF gene therapy, and Professor Howard Federoff of Georgetown University, a preeminent physician-neuroscientist, have developed a product approved to start clinical trials in the U.S. using uniQure's GDNF gene incorporated into an adeno-associated virus-2 (AAV-2) delivery vector. The GDNF gene contains the information to produce a protein necessary for the development and survival of nerve cells. The positive effect of GDNF on nerve cells has already been demonstrated in early research by uniQure in collaboration with the University of Lund, Sweden.

UCSF entered into a collaboration with Dr. Russell Lonser, neurosurgeon and Chief of the Neurosurgical Branch of the NINDS, a division of the National Institutes of Health, to commence a Phase I study of the gene therapy in patients with Parkinson's disease. Patient enrollment is expected to begin mid-2012. Collaborating on the study will be Drs. Krystof Bankiewicz of UCSF, Howard Federoff of Georgetown University and NINDS co-investigator neurologists Drs. Mark Hallett and Walter Koroshetz.

"This agreement provides uniQure with access to the data from a Parkinson's disease GDNF clinical study conducted by two of the world's leading medical researchers in the field. If successful, we intend to manufacture the vector construct ourselves and with a partner progress the product into advanced clinical studies," said Jrn Aldag, CEO of uniQure. "GDNF has been shown to be involved in several other CNS disorders so if we reach the proof of concept stage in Parkinson's, we can potentially expand product development quickly and efficiently into clinical trials for other indications, such as Huntington's and Multiple System Atrophy (MSA)."

"The development of AAV2-GDNF, sponsored by both NIH and by Parkinson's foundations, has taken us 10 years to complete. We are very pleased that a path for clinical development of AAV2-GDNF as a possible treatment for PD is now in place," said Dr. Krystof Bankiewicz, UCSF Principal Investigator.

Under the terms of uniQure's agreement with UCSF, uniQure holds the exclusive commercial rights to all UCSF preclinical data and to IND enabling Phase I clinical data provided to UCSF by NINDS. In the event that the Phase 1 study shows proof of concept, uniQure will use its proprietary manufacturing system for future production of the AAV construct and take responsibility for future development of the gene therapy product. uniQure holds the exclusive license to the GDNF gene from Amgen.

About uniQure

uniQure is a world leader in the development of human gene based therapies. uniQure has a product pipeline of gene therapy products in development for hemophilia B, acute intermittent porphyria, Parkinson's disease and SanfilippoB. Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate probably the world's first stable and scalable AAV manufacturing platform. This proprietary platform can be applied to a large number of rare (orphan) diseases caused by one faulty gene and allows uniQure to pursue its strategy of focusing on this sector of the industry. Further information can be found at http://www.uniqure.com.

Certain statements in this press release are "forward-looking statements" including those that refer to management's plans and expectations for future operations, prospects and financial condition. Words such as "strategy," "expects," "plans," "anticipates," "believes," "will," "continues," "estimates," "intends," "projects," "goals," "targets" and other words of similar meaning are intended to identify such forward-looking statements. Such statements are based on the current expectations of the management of uniQure only. Undue reliance should not be placed on these statements because, by their nature, they are subject to known and unknown risks and can be affected by factors that are beyond the control of uniQure. Actual results could differ materially from current expectations due to a number of factors and uncertainties affecting uniQure's business. uniQure expressly disclaims any intent or obligation to update any forward-looking statements herein except as required by law.

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uniQure Collaborates with UCSF on GDNF Gene Therapy in Parkinson's Disease

Gold nanoparticles capable of 'unzipping' DNA

ScienceDaily (June 20, 2012) New research from North Carolina State University finds that gold nanoparticles with a slight positive charge work collectively to unravel DNA's double helix. This finding has ramifications for gene therapy research and the emerging field of DNA-based electronics.

"We began this work with the goal of improving methods of packaging genetic material for use in gene therapy," says Dr. Anatoli Melechko, an associate professor of materials science and engineering at NC State and co-author of a paper describing the research. Gene therapy is an approach for addressing certain medical conditions by modifying the DNA in relevant cells.

The research team introduced gold nanoparticles, approximately 1.5 nanometers in diameter, into a solution containing double-stranded DNA. The nanoparticles were coated with organic molecules called ligands. Some of the ligands held a positive charge, while others were hydrophobic -- meaning they were repelled by water.

Because the gold nanoparticles had a slight positive charge from the ligands, and DNA is always negatively charged, the DNA and nanoparticles were pulled together into complex packages.

"However, we found that the DNA was actually being unzipped by the gold nanoparticles," Melechko says. The positively-charged ligands on the nanoparticles attached to the DNA as predicted, but the hydrophobic ligands of the nanoparticles became tangled with each other. As this tangling pulled the nanoparticles into clusters, the nanoparticles pulled the DNA apart.

"We think gold nanoparticles still hold promise for gene therapy," says Dr. Yaroslava Yingling, an assistant professor of materials science and engineering at NC State and co-author of the paper. "But it's clear that we need to tailor the ligands, charge and chemistry of these materials to ensure the DNA's structural integrity is not compromised."

The finding is also relevant to research on DNA-based electronics, which hopes to use DNA as a template for creating nanoelectronic circuits. Because some work in that field involves placing metal nanoparticles on DNA, this finding indicates that researchers will have to pay close attention to the characteristics of those nanoparticles -- or risk undermining the structural integrity of the DNA.

The research was supported by the National Science Foundation.

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Gold nanoparticles capable of 'unzipping' DNA

Close to a cure: Greater Hartford takes on rare Jewish genetic disease

By Cindy Mindell ~

WEST HARTFORD Its not often that philanthropic donors to a medical cause can see their money pushing researchers over the finish line. But those helping to fund efforts to cure Glycogen Storage Disease (GSD) Type 1a may soon become the rare exception. Last month, A Cure for Sure raised $470,000 toward the $2 million needed to get FDA approval on new gene therapy that has already cured GSD-stricken dogs. The fundraiser was organized and chaired by Gayle and Steve Temkin, and Regan and Barry Stein, two West Hartford couples whose children have the disease. GSD is an inherited disorder caused by the buildup of a complex sugar, glycogen, in the bodys cells. The accumulation of glycogen in certain organs and tissues, especially the liver, kidneys, and small intestines, impairs their ability to function normally. The disease was almost universally fatal until 1971, when the first effective therapy edible cornstarch was discovered. When treated properly, patients are healthy, but inadequate treatment may result in permanent neurologic damage, seizures, or even death.

Dr. David Weinstein

Dr. David Weinstein treats 400 of the estimated 1,100 people afflicted with this type of GSD throughout the world, mostly Jewish. In 2005, he left Harvard Medical School and Boston Childrens Hospital to create the University of Florida Glycogen Storage Disease Program in Gainsville, where he could perform gene therapy on dogs born with GSD. Since then, Weinstein and his research team have cured 10 dogs of the disease at what is the worlds largest clinical and research program for glycogen storage diseases. When the Temkins first brought their daughter, Alyssa, to Weinstein six years ago for lifesaving treatment, she was 15 months old and the doctor had cured one dog. The Temkins went on to establish the Alyssas Angel Fund, which funds travel and treatment for other families with children who have GSD. Every Labor Day weekend for the last three years, the Mandell JCC of Greater Hartford Family Room Parenting Center has sponsored the Big Wheel Derby fundraiser to benefit the fund. Last year, before the event, Weinstein came to West Hartford and met with the Temkins and Steins. We would do anything in our power to support David, says Gayle Temkin. He told us that he needed $2 million to take the gene therapy to the FDA and start human trials. Why couldnt we at least start the fundraising for him? I felt that if I didnt work hard to find a cure and make the effort public, nobody would ever know about it. The Steins approached Alan and Marcia Lazowski, whose son is best friends with their son. According to Temkin, Alan told them, Ive been waiting for an opportunity to help you, and agreed to host a fundraising event. In the wake of A Cure for Sure, the $250,000 goal was nearly doubled by donations from Greater Hartford and Chicago, where the Temkins and Steins hope to organize another fundraiser. People are really feeling that this is the first time ever that theyre part of a cure, Gayle says. For me as a parent, its amazing to be able to say that. Human trials of Weinsteins gene therapy are slated to begin in 2013. For the Temkins, that goal is critical, as Alyssa is the only identified person with GSD who cannot tolerate the cornstarch therapy. But as plans for the fourth annual Big Wheel Derby start to take shape, the fundraiser is infused with new hope and energy, Gayle says. Whats so special now about Alyssas Angel Fund is that, once the cure happens, we can help families without insurance to get the gene therapy. Most of the dogs in Weinsteins program receive two intravenous treatments, at $15,000 each; human subjects will undergo both IV and surgical procedures. So right now, pushing for Alyssas Angel Fund is as important as pushing for A Cure for Sure, she says.

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Close to a cure: Greater Hartford takes on rare Jewish genetic disease

bluebird bio Receives U.S. and European Orphan Drug Designation for Novel Gene Therapy to Treat Adrenoleukodystrophy

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

bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, announced today that both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted an orphan drug designation to its investigational gene therapy product for the treatment of adrenoleukodystrophy (ALD). The product consists of the patients own CD34+ hematopoietic stem cells transduced with bluebird bios lentiviral vector, Lenti-D, encoding the human ABCD1 cDNA. Based on promising early clinical proof of concept results, bluebird bio plans to initiate a Phase 2/3 clinical study in childhood cerebral ALD in both the United States and Europe in 2013.

Receiving orphan drug designation is a positive step forward in our efforts to bring hope to ALD patients and their families, said David Davidson, M.D., chief medical officer of bluebird bio. We believe our lentiviral technology has the potential to be a one-time transformative therapy for patients suffering from rare genetic disorders like ALD for whom there are limited treatment options. bluebird is committed to advancing the clinical and commercial development of our gene therapy platform because of the dramatic benefit it may have on the lives of patients.

Orphan drug designation, which is intended to facilitate drug development for rare diseases, provides substantial benefits to the sponsor, including the potential for funding for certain clinical studies, study-design assistance, and several years of market exclusivity for the product upon regulatory approval.

About ALD

Adrenoleukodystrophy (ALD) is a rare X-linked, inherited neurological disorder that, in its most severe form, causes damage to the myelin sheath (an insulating layer of membranes that surrounds nerve cells in the brain) and progressive dysfunction of the adrenal glands. Also known as Lorenzo's Oil disease, ALD is estimated to affect one in every 21,000 boys worldwide. In the childhood cerebral form (CCALD), symptoms usually occur between the ages of 4 and 10. Boys afflicted with this form of ALD develop normally until the onset of symptoms. The symptoms of this disorder often progress rapidly and, in a matter of years, can lead to a vegetative state and, ultimately, death. Current treatment options are limited to allogeneic stem cell transplantation when there is an appropriate donor. Allogeneic transplants carry a significant risk of serious morbidity and death.

About bluebird bio's CCALD Product Development

bluebird bios CCALD product program has the potential to halt the progression of CCALD by providing a functional ABCD1 gene to the patients own stem cells. These stem cells proliferate, and some of the progeny cells travel to the brain where they become microglial cells incorporating the corrective gene. Data from the first clinical study treating X-linked CCALD patients with the companys lentiviral gene therapy product demonstrated continued stable expression of the transgene and the corresponding ABCD-1 protein for over four years in two CCALD patients, resulting in prolonged disease stabilization. bluebird bio plans to initiate a Phase 2/3 clinical study in CCALD in both the United States and Europe in 2013.

About bluebird bio

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

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bluebird bio Receives U.S. and European Orphan Drug Designation for Novel Gene Therapy to Treat Adrenoleukodystrophy

Anti-cocaine vaccine described in Human Gene Therapy Journal

Public release date: 18-Jun-2012 [ | E-mail | Share ]

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, June 18, 2012A single-dose vaccine capable of providing immunity against the effects of cocaine offers a novel and groundbreaking strategy for treating cocaine addiction is described in an article published Instant Online in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc. (http://www.liebertpub.com) The article is available free online at the Human Gene Therapy website (http://www.liebertpub.com/hum).

"This is a very novel approach for addressing the huge medical problem of cocaine addiction," says James M. Wilson, MD, PhD, Editor-in-Chief, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.

In the article "AAVrh.10-Mediated Expression of an Anti-Cocaine Antibody Mediates Persistent Passive Immunization That Suppresses Cocaine-Induced Behavior," (http://online.liebertpub.com/doi/pdfplus/10.1089/hum.2011.178) a team of researchers from Weill Cornell Medical College (New York, NY), The Scripps Research Institute (La Jolla, CA), and Cornell University (Ithaca, NY) used a virus-based delivery vehicle in mice to transfer a gene that produces a protein capable of binding to cocaine present in the blood, preventing the cocaine from crossing into the brain. The protein is a monoclonal antibody that sequesters cocaine, making the vaccinated mice resistant to the drug's effects. Whereas unvaccinated mice exhibited hyperactivity when exposed to intravenous cocaine, the immunized mice showed no effects, according to authors Jonathan Rosenberg, et al.

###

About the Journal

Human Gene Therapy (http://www.liebertpub.com/hum), the Official Journal of the European Society of Gene and Cell Therapy, British Society for Gene Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies is an authoritative peer-reviewed journal published monthly in print and online that presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Tables of contents and a free sample issue may be viewed online at the Human Gene Therapy website (http://www.liebertpub.com/hum).

About the Publisher

Mary Ann Liebert, Inc. (http://www.liebertpub.com) is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Tissue Engineering, Stem Cells and Development, and Cellular Reprogramming. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 70 journals, books, and newsmagazines is available at the Mary Ann Liebert, Inc. website (http://www.liebertpub.com).

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Anti-cocaine vaccine described in Human Gene Therapy Journal

uniQure Extends Collaboration with Protein Sciences Corporation on Use of its expresSF+® Cell Line for Gene Therapy

AMSTERDAM, The Netherlands, June 18, 2012 /PRNewswire/ --

uniQure, a leader in the field of human gene therapy, announced today the extension of its collaboration with Protein Sciences Corporation ("Protein Sciences") for the exclusive use of Protein Sciences' expresSF+ (SF+) insect cell line in uniQure's AAV gene therapy programs for three specific disease indications. uniQure has the option to extend this exclusivity further into additional indications in the future.

"This agreement strengthens uniQure's gene therapy platform and further demonstrates the quality of the cell line developed by Protein Sciences." said Joern Aldag, Chief Executive of uniQure. "Protein Sciences' cell line license agreement with Merck made earlier this year and the anticipated approval of its influenza vaccine FluBlok implies SF+ technology will hold a prominent place in the manufacture of biologics."

The SF+ cell line developed by Protein Sciences is an integral component of uniQure's validated, world leading manufacturing platform. uniQure believes that this platform is the only commercially-scalable platform available for manufacturing AAV gene therapy products.

Financial terms were not disclosed.

About uniQure

uniQure is a world leader in the development ofhuman gene based therapies.uniQure has a product pipeline of gene therapy products in development for hemophilia B, acute intermittent porphyria, Parkinson's disease and SanfilippoB. Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate probably the world's first stable and scalable AAV manufacturing platform.This proprietary platform can be applied to a large number of rare(orphan) diseases caused by one faulty gene and allows uniQure to pursue its strategy of focusing on this sector of the industry. Further information can be found at http://www.uniqure.com.

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uniQure Extends Collaboration with Protein Sciences Corporation on Use of its expresSF+® Cell Line for Gene Therapy

New York Law Firm’s MesotheliomaHelp.net Site Publishes Interview with Gene Therapy Author

New York mesothelioma lawyer Joseph W. Belluck says indications that research may lead to a gene therapy approach to mesothelioma treatment provides a source of hope to asbestos-disease victims.New York, NY (PRWEB) June 16, 2012 Gene therapy may one day be a viable part of mesothelioma treatment, New York-based geneticist and author Ricki Lewis says in an exclusive interview on the ...

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New York Law Firm’s MesotheliomaHelp.net Site Publishes Interview with Gene Therapy Author

Research and Markets: Gene Therapy – Global Strategic Business Report – 2012

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/g2sg6b/gene_therapy_glo) has announced the addition of the "Gene Therapy - Global Strategic Business Report" report to their offering.

This report analyzes the Global market for Gene Therapy in US$ Million. Annual estimates and forecasts are provided for the period 2009 through 2017. The report profiles 98 companies including many key and niche players such as:

- AnGes MG, Inc.

- BioSante Pharmaceuticals

- GenVec

- Oxford BioMedica

- Shenzhen SiBiono GeneTech Co., Ltd.

- Transgene

- Vical Inc

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Research and Markets: Gene Therapy - Global Strategic Business Report - 2012

SalutarisMD Announces Positive Case Report of a New Investigational Wet AMD Therapy at ARVO

DENVER--(BUSINESS WIRE)--

Positive results from a trial of episcleral brachytherapy to treat Wet Age-related Macular Degeneration (Wet AMD) were presented today at the ARVO Drug and Gene Delivery to the Back of the Eye Conference. The case reported was drawn from a Phase 1 study to assess the safety of this new investigational therapy for the leading cause of vision loss and blindness. Salutaris Medical Devices, Inc. (SalutarisMD) developed the device and sponsored the study.

Presented was the clinical course of a 78 year-old man newly diagnosed with Wet AMD who experienced substantial improvement in visual acuity and required no additional anti-VEGF injections throughout one-year follow-up; visual acuity in the study eye demonstrated a gain in Best Corrected Visual Acuity (BCVA) of +13 ETDRS letters, with no sign of subretinal hemorrhage, fluid or macular edema, and resolution of the Pigmented Epithelial Detachment (PED) present at study enrollment.

Co-author, Dr. Laurence Marsteller, Chief Operating Officer, SalutarisMD, cautioned, "While the results presented are not intended to be extrapolated for statistical significance, this promising case report from the Phase 1 trial supports the need for additional research to confirm our preliminary observations."

The poster presentation included details of the device and the sub-Tenon episcleral approach to delivering brachytherapy that avoids adverse effects of more invasive approaches. The SalutarisMD device is designed to enable retina specialists to administer a practical procedural therapy that can be performed in the same clinical environment as current anti-VEGF injections: a physician's office or other outpatient setting under local anesthesia, in approximately 15 minutes. The intraocular space is never violated. Episcleral placement allows for consistent, stable and repeatable control of the distance to the target tissue. Utilizing this minimally invasive technology, the retina specialist delivers precise, lesion-specific, localized tissue treatment.

"What is most intriguing about the study is that the application of radiation is done through the posterior sclera, thus avoiding the need for vitreous surgery," said Dr. Reid Schindler, principal investigator of the study, a clinical ophthalmologist and retina specialist with Retina Specialists of Southern Arizona, and clinical associate professor, University of Arizona Department of Ophthalmology.

Michael Voevodsky, President and CEO of SalutarisMD, said, "It was a privilege to have the SalutarisMD technology presented at this prestigious gathering. We believe our investigational therapy for treating Wet AMD has the potential to improve the quality of life for persons suffering from this debilitating disease. We are excited about the prospect of conducting additional clinical trials to further test our approach and its ability to positively effect clinical outcomes."

The purpose of the ARVO Conference,Drug and Gene Delivery to the Back of the Eye: from Bench to Bedside, "is to share cutting edge science, based on drug product development principles among a diverse group of participants" and it focuses "on topics related to current and emerging technologies for drug/gene delivery for the treatment of diseases of the back of the eye," according to the event description.

Caution: Investigational Device. Limited by Federal Law to Investigational Use in the United States.

About SalutarisMD

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SalutarisMD Announces Positive Case Report of a New Investigational Wet AMD Therapy at ARVO

PIK3CA Gene Patent for Predicting Response to Targeted Therapy Issued – Exclusively Licensed to Transgenomic

OMAHA, Neb.--(BUSINESS WIRE)--

Transgenomic, Inc. (TBIO) announced that the US Patent and Trademark Office has issued patent number US 8,137,919 entitled Method of Determining the Sensitivity of Cancer Cells to EGFR Inhibitors including Cetuximab, Panitumumab and Erlotinib. The patent was exclusively licensed to Transgenomic by the Montefiore Medical Center (Bronx, NY, US) and includes all tumor types and targeted therapies that may be influenced by PIK3CA mutation status.

Montefiore inventors Drs. Sanjay Goel and John Mariadason have demonstrated that key mutations in the gene PIK3CA are powerful predictors for the efficacy of EGFR-targeted therapies such as cetuximab (Erbitux), panitumumab (Vectibix) and erlotinib (Tarceva). These findings were published in the June 2012 issue of Clinical Colorectal Cancer by the same researchers and have been reproduced in other independent studies.

Assays using Transgenomics proprietary SURVEYOR Scan, REVEAL ICE COLD-PCR and BLOCker-Sequencing for complete detection of PIK3CA mutations have been developed. The extremely high sensitivity of Transgenomics REVEAL ICE COLD-PCR technology enables the use of virtually any sample type including blood and circulating tumor cells. Non-invasive testing allows for more frequent and accurate profiling of a cancer as it responds to treatment and gains additional mutations.

The recent issuing of this important patent is a significant milestone in the continued development of our genetic biomarker intellectual property portfolio, said Craig Tuttle, CEO of Transgenomic. Since exclusively licensing this patent we have been able to effectively apply our high sensitivity mutation detection technologies, such as SURVEYOR Scan, REVEAL ICE COLD-PCR and BLOCker-sequencing, to PIK3CA assays in order to be able to detect genetic variations in very low mutant load samples, such as plasma, serum and circulating tumor cells.

Tuttle added that, The number of genes associated with the effectiveness of targeted cancer treatments is increasing; our strategy is to provide a complete portfolio of best-in-class kits for clinically relevant mutations using our proprietary and extremely sensitive technologies. These assays will also be available through our CLIA and Pharmacogenomics laboratories to support clinicians and pharmaceutical research and trials.

About Transgenomic

Transgenomic, Inc. (www.transgenomic.com) is a global biotechnology company advancing personalized medicine in cancer and inherited diseases through its proprietary molecular technologies and world-class clinical and research services. The Company has three complementary business divisions: Transgenomic Pharmacogenomic Services is a contract research laboratory that specializes in supporting all phases of pre-clinical and clinical trials for oncology drugs in development. Transgenomic Clinical Laboratories specializes in molecular diagnostics for cardiology, neurology, mitochondrial disorders, and oncology. Transgenomic Diagnostic Tools produces equipment, reagents, and other consumables that empower clinical and research applications in molecular testing and cytogenetics. Transgenomic believes there is significant opportunity for continued growth across all three businesses by leveraging their synergistic capabilities, technologies, and expertise. The Company actively develops and acquires new technology and other intellectual property that strengthen its leadership in personalized medicine.

Forward-Looking Statements

Certain statements in this press release constitute forward-looking statements of Transgenomic within the meaning of the Private Securities Litigation Reform Act of 1995, which involve known and unknown risks, uncertainties and other factors that may cause actual results to be materially different from any future results, performance or achievements expressed or implied by such statements. Forward-looking statements include, but are not limited to, those with respect to management's current views and estimates of future economic circumstances, industry conditions, company performance and financial results, including the ability of the Company to grow its involvement in the diagnostic products and services markets. The known risks, uncertainties and other factors affecting these forward-looking statements are described from time to time in Transgenomic's filings with the Securities and Exchange Commission. Any change in such factors, risks and uncertainties may cause the actual results, events and performance to differ materially from those referred to in such statements. Accordingly, the Company claims the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995 with respect to all statements contained in this press release. All information in this press release is as of the date of the release and Transgenomic does not undertake any duty to update this information, including any forward-looking statements, unless required by law.

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PIK3CA Gene Patent for Predicting Response to Targeted Therapy Issued – Exclusively Licensed to Transgenomic

Baxter Inks Deal with Chatham

Global medical products and services company, Baxter International, Inc. (BAX), recently inked a global agreement with Chatham Therapeutics, LLC. Per the deal, Baxter will use Chathams gene therapy know-how to develop and market new products for curing hemophilia B.

Baxter believes that investment in research and development (R&D) will further expand its BioScience segments hemophilia product portfolio and be accretive to future sales. Bioscience sales were $1,462 million, up 4% (up 5% in constant currency) year over year, in the most recent quarter. The performance was attributable to higher demand for products utilized in the treatment of hemophilia and immune disorders, such as Advate and Gammagard Liquid (Immune Globulin Intravenous Human), several specialty plasma-based therapeutics and vaccines.

Baxter wishes to run clinical trials on Chathams unique Biological Nano Particles (BNP), a gene therapy technology, which has generated successful results during initial trials. The company paid $25 million for the program during its initial trials (which it will record as a special pre-tax in-process R&D expense in the second quarter of 2012) and plans to invest further, subject to certain terms and conditions.

The company has already been conducting several researches in hemophilia, including the BAX326 clinical trial for treating hemophilia B. It plans to file for an U.S. approval of BAX326 by late 2012.

Privately-owned Chatham Therapeutics, LLC is an associate of Asklepios BioPharmaceutical, Inc. (AskBio), which focuses on developing gene therapy based treatments for both hemophilia A and B.

The news regarding Baxter still remains mixed. On the positive side, Baxters focus on life-sustaining products, which are not commoditized, partly insulates it from an economic downturn. The company is able to generate recurring revenues, and consistent cash flow, owing to its focus on chronic diseases. Among other positive factors, Baxter retains a strong product pipeline with several products in late-stage clinical development.

Baxter struck a deal, in December 2011, to buy Synovis Life Technologies, a well-known provider of mechanical and biological products for the repair of soft tissue utilized in a large number of surgical operations. The acquisition will further expand Baxters offerings in the area of biosurgery and regenerative treatment.

Earlier, in November 2011, Baxter completed its acquisition of Baxa Corporation. The takeover highlights the companys continued commitment toward patient safety and nutrition. It also permits Baxter to provide a wider set of solutions for the safe preparation and delivery of IV medication. Baxas know-how will benefit patients across the globe.

On the flip side, despite resilience in Plasma Proteins and Antibody Therapy sub-segments, we are concerned about stagnation in sales, a slightly somber outlook for hospital spending and tightening of reimbursement. We also account for the unfavorable impact of foreign exchange translation and possible dilution associated with the companys acquisitions of Baxa and Synovis.

Improved execution has lifted sentiment somewhat toward Baxter. It is a good bet for value investors willing to wait as fundamentals improve further. Among others, the company competes with Becton, Dickinson and Company (BDX) in certain niches. We currently have a Neutral long-term rating on Baxter. The stock currently retains a Zacks #3 Rank, which translates into a short-term Hold recommendation.

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Baxter Inks Deal with Chatham

Transgenomic Launches Breakthrough Blood-Based Cancer Gene Testing Technology at 2012 ASCO Annual Meeting

OMAHA, Neb.--(BUSINESS WIRE)--

Transgenomic, Inc. (TBIO) today announced the commercial launch of its ICE COLD-PCR mutation detection technology, a breakthrough technology enabling unmatched sensitivity and complete DNA mutation detection using the standard sequencing equipment already installed in laboratories around the world. The launch coincides with the 2012 annual meeting of the American Society of Clinical Oncology.

ICE COLD-PCR is capable of identifying mutation frequencies as low or lower than 0.01% which surpasses the limits of currently available mutation detection tests. This extremely high sensitivity enables detection of mutations from virtually any sample type including tissue biopsies, blood, and circulating tumor cells (CTCs). Mutation profiling from blood and CTCs may benefit cancer patients because it avoids the risks of additional surgical procedures while providing an up-to-date picture of any additional mutations the cancer may have acquired throughout treatment.

An ICE COLD-PCR kit for enrichment of KRAS mutations is now available worldwide to molecular diagnostic laboratories for use with standard DNA sequencing equipment. Transgenomic plans to expand the ICE COLD-PCR testing platform to include other therapeutically relevant mutations including BRAF, EGFR, and PIK3CA.

The broad use of ICE COLD-PCR has the potential to revolutionize cancer screening, diagnosis, monitoring, and therapy selection said Craig Tuttle, Chief Executive Officer of Transgenomic. It offers us the ability to accurately perform safer, less invasive, and more frequent assessments of a cancer and its mutations, all through a simple blood draw. Ultimately, the goal is to provide real-time monitoring of cancer progression, resistance mutations and response to therapy. With the proliferation of targeted anti-cancer drugs now available or in clinical trials, ICE COLD-PCR will help determine the right path for each patient at every step of his or her treatment, making precision medicine even more precise.

Mr. Tuttle added: ICE COLD-PCR provides extreme sensitivity and coverage to ensure that mutations are not missed, both are needed for reliable mutation profiling from blood, CTCs, and small sample sizes. Because it is used with the sequencing equipment already installed in labs around the world, we expect broad and sustained adoption of this technology, with kit sales beginning this year. Each of the markets addressed by ICE COLD-PCR diagnosis, monitoring, and disease recurrence is substantial, providing a significant value-creation opportunity for Transgenomic.

ICE COLD-PCR technology was developed in collaboration with the Dana-Farber Cancer Institute and is supported by multiple validation studies confirming reproducible mutation detection at very high sensitivity up to 1,000 times more sensitive than traditional PCR techniques. The technology is also being evaluated in an ongoing study with The University of Texas MD Anderson Cancer Center to analyze DNA isolated from CTCs.

About Transgenomic, Inc.

Transgenomic, Inc. (www.transgenomic.com) is a global biotechnology company advancing personalized medicine in cancer and inherited diseases through its proprietary molecular technologies and world-class clinical and research services. The Company has three complementary business divisions: Transgenomic Pharmacogenomic Services is a contract research laboratory that specializes in supporting all phases of pre-clinical and clinical trials for oncology drugs in development, Transgenomic Clinical Laboratories, which specializes in molecular diagnostics for cardiology, neurology, mitochondrial disorders, and oncology, and Transgenomic Diagnostic Tools which produces equipment, reagents, and other consumables that empower clinical and research applications in molecular testing and cytogenetics. Transgenomic believes there is significant opportunity for continued growth across all three businesses by leveraging their synergistic capabilities, technologies, and expertise. The Company actively develops and acquires new technology and other intellectual property that strengthen its leadership in personalized medicine.

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Transgenomic Launches Breakthrough Blood-Based Cancer Gene Testing Technology at 2012 ASCO Annual Meeting

Baxter Announces Collaboration with Chatham Therapeutics for Factor IX Hemophilia B Gene Therapy Treatment

DEERFIELD, Ill.--(BUSINESS WIRE)--

Baxter International Inc. (BAX) today announced that it has entered into an exclusive global agreement with Chatham Therapeutics, LLC, an affiliate of Asklepios BioPharmaceutical, Inc. (AskBio), for the development and commercialization of potential treatments for hemophilia B utilizing Chathams gene therapy technology.

The collaboration will allow Baxter to investigate Chathams Biological Nano ParticlesTM (BNP), an advanced recombinant adeno-associated virus-(rAAV) based gene therapy technology that has shown potential therapeutic benefit in early clinical studies. A small independent study involving six patients using Chatham technology components was the topic of a 2011 article in The New England Journal of Medicine.i This agreement will involve the next generation of this gene therapy technology, which Baxter and Chatham will investigate through U.S.-based hemophilia B clinical trials. Baxter has obtained global rights for the marketing and commercialization of the new treatment.

''This collaboration demonstrates Baxters ongoing commitment to scientific innovation in advancing treatment options for patients living with hemophilia. This initiative complements Baxters extensive hemophilia portfolio and helps to address unmet needs of hemophilia patients,'' said Ludwig Hantson, Ph.D., president of Baxters BioScience business.

Baxter made a $25 million upfront cash payment for the development and advancement of the program through early clinical trials, and will record this amount as a special pre-tax in-process research and development charge in the second quarter of 2012. Baxter may make additional payments over the next several years based on certain development and commercial milestones.

''This agreement initiates a clinical development collaboration dedicated to advancing a potential long-term treatment paradigm for hemophilia patients. We look forward to working with Baxter and view this transaction as the optimal path toward providing a sustainable therapeutic to a worldwide patient population,'' said Jade Samulski, Vice President at AskBio and Co-Founder of Chatham Therapeutics.

Hemophilia B is the second most common typeof hemophilia (also known as Christmas disease) and is the result of insufficient amounts of clotting factor IX, a naturally occurring protein in blood that controls bleeding.ii Hemophilia B occurs in about one in 25,000 males, with approximately 4,000 people in the United States currently diagnosed with the disease.iii Hemophilia B is often a debilitating, chronic disease with complications that include bleeding episodes, hemophilic arthropathy (bleeding into a joint) and hospitalization.iv

Baxter is pursuing a number of research opportunities in hemophilia. The company is conducting a Phase I/III clinical trial of BAX326, a recombinant Factor IX being evaluated for the treatment of patients with hemophilia B, and expects to file for U.S. approval by the end of 2012.

About Baxter International Inc.

Baxter International Inc., through its subsidiaries, develops, manufactures and markets products that save and sustain the lives of people with hemophilia, immune disorders, cancer, infectious diseases, kidney disease, trauma and other chronic and acute medical conditions. As a global, diversified healthcare company, Baxter applies a unique combination of expertise in medical devices, pharmaceuticals and biotechnology to create products that advance patient care worldwide.

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Baxter Announces Collaboration with Chatham Therapeutics for Factor IX Hemophilia B Gene Therapy Treatment

Alliance for Cancer Gene Therapy celebrates 10 years

Written by Greenwich Post Staff Tuesday, 29 May 2012 08:00

The Alliance for Cancer Gene Therapy (ACGT) celebrated its 10-year anniversary and New Frontiers in the field of cancer gene therapy research with a gala benefit in April at the Hyatt Regency Greenwich. More than 450 supporters of ACGT and over 30 of the nations top cancer research scientists gathered to celebrate progress that has been made in the field. The event raised more than $1.4 million, of which 100% will go directly toward funding new grants and clinical trials using cancer gene therapy.

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

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Alliance for Cancer Gene Therapy celebrates 10 years

Gene therapy can correct forms of severe combined immunodeficiency, study suggests

ScienceDaily (May 24, 2012) Severe combined immunodeficiency is defect in the immune system that results in a loss of the adaptive immune cells known as B cells and T cells. Mutations in several different genes can lead to the development of severe combined immunodeficiency, including mutation of the adenosine deaminase (ADA) gene. Traditional treatment options, such as enzyme replacement therapy, are of limited efficacy, but bone marrow transplant from a compatible donor leads to a better response.

A recent clinical trial indicated that gene therapy to insert the correct ADA gene in the patient's own bone marrow cells can also lead to a good response.

However, patients were noted to have defects in B cell tolerance, meaning that some B cells that react to antigens from the body fail to be eliminated, leading to an autoimmune response. Dr. Eric Meffre and colleages at Yale University in New Haven, Connecticut and Alessandro Aiuti in Milan, Italy joined together to better understand why patients developed B cell tolerance problems. They found that loss of the ADA gene directly contributes to B cell tolerance problems and that these defects are mostly corrected after gene therapy.

Their results point to a previously unknown role for ADA in B cell response and support the use of gene therapy as an effective treatment option for ADA-deficient severe combined immunodeficiency patients.

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Gene therapy can correct forms of severe combined immunodeficiency, study suggests