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July 2010

 
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CURE's Annual Meeting and BioHaven Series Showcase Connecticut Biopharma
Yale Cell Biology Researcher Wins $1 Million Prize
Healthcare Reform Focus of CURE Annual Meeting
Ion Torrent & 4 Biotechs Draw Venture Capital
JS Genetics Presents at CURE/Yale BioHaven Series
Amarin Corporation Focus of CURE/Yale BioHaven Presentation
Jonathan Rothberg Presents at CURE Yale BioHaven
CT Stem Cell Research Roundup
CURE Member News Digest
CURE Member News Digest
 

Salute to new member
Health Sciences International, Inc.
is a clinical research organization established in 1997 that comes to Connecticut with expertise in the conduct of Phase I-IV clinical trials. HSI says it has a unique business model that assures that Phase I-IV clinical trials, in multiple therapeutic areas, meet milestone timelines while adhering to enforceable SOPs, GCP, Standards of the Industry, and CFR/ ICH Guidelines. More at http://www.hsiresearch.org/

454 Life Sciences (Branford) reports that in a study of the Mimivirus, the world's largest known virus, a research team from the Structural and Genomic Information Laboratory at the Mediterranean Institute of Microbiology in Marseille, France, used the Genome Sequencer FLX System from 454 Life Sciences to perform the first ever viral transcriptome analysis by deep sequencing RNAs expressed by infected amoebas throughout the Mimivirus replication cycle. Unexpectedly, the analysis revealed 75 new genes, including 26 producing non-coding RNAs. The existence, and so far unknown function, of these additional genes in the already plethoric Mimivirus genome could radically change the current understanding of the way large viruses operate.

Roche (parent company of 454 Life Sciences) and IBM announced an agreement to develop a nanopore-based sequencer that will directly read and decode human DNA quickly and efficiently. The novel technology, developed by IBM Research, offers true single molecule sequencing by decoding molecules of DNA as they are threaded through a nanometer-sized pore in a silicon chip. The approach holds the promise of significant advantages in cost, throughput, scalability, and speed compared to sequencing technologies currently available or in development, Roche says.

Achillion Pharmaceuticals, Inc. (New Haven) has received a Phase 1 Small Business Innovation Research Grant from the National Institute of Allergy and Infectious Disease branch of the National Institutes of Health for the further study and characterization of a back-up series of compounds related to its novel antibacterial, ACH-702. The new compounds in the series to be studied demonstrate excellent inhibition of the essential bacterial enzyme DNA gyrase, and have improved metabolic stability. In particular, preliminary data show potent target inhibition of the Mycobacterium tuberculosis gyrase enzyme which translated in vitro to good antibacterial activity against drug-resistant tuberculosis.

Alexion Pharmaceuticals (Cheshire) said it is accelerating by three months to the third quarter the introduction of its Soliris® blood disorder treatment in Japan, a move that enhances the Cheshire drug maker's 2010 financial outlook. With the Japan sales stream coming on line sooner than expected, Alexion raised its revenue guidance for 2010 to $515 million to $530 million — $10 million more than the previous upper and lower limits.

Earlier the company announced that it has completed enrolling patients in all four previously announced prospective, open-label clinical studies being conducted in North America and multiple European countries investigating Soliris© (eculizumab) as a potential treatment for patients with atypical Hemolytic Uremic Syndrome (aHUS). "Given the potential for rapid and life-threatening deterioration observed in patients with aHUS, and the inability to predict sudden worsening in an individual, we are increasingly focused on diligently advancing our development efforts. We look forward to presenting preliminary results from these studies later in 2010," said Leonard Bell, M.D., Chief Executive Officer of Alexion. "In addition, because a significant number of patients with aHUS are children, we are committed to expanding our aHUS program to include studies of Soliris in pediatric patients with aHUS."

Amarin Corp (Mystic) said its two clinical trials for a heart-disease treatment are moving ahead of schedule, adding that its $44 million in cash on hand as of March 31 is more than enough to support the trials. The trials will test Amarin's AMR101 treatment for reducing high levels of triglycerides, the bloodstream's fatty chemical linked to clogged and hardened arteries.

Meanwhile, the company, originally based in Ireland, has moved the majority of its operations to Mystic. The consolidation in Mystic comes after the company secured $70 million in new financing last year.

Applied Spine Technologies, Inc. (Rocky Hill) has been awarded a new patent titled "Spinal Stabilization Device with Weld Cap." U.S. patent No. 7,699,875, issued by the U.S. Patent and Trademark Office on April 20, 2010, claims geometry for a spring based spinal stabilization device including members to mechanically connect the spring to other structural components. "One of the documented clinical benefits of our Stabilimax device is its allowance of significant interpedicular travel,” said Craig Corrance, president and CEO. "The issuance of this patent recognizes the uniqueness associated with our device and adds yet another layer to our patent portfolio."

Bayer HealthCare (Leverkusen, Germany/West Haven) reports that an orphan drug designation has been granted by the U.S. Food and Drug Administration for ciprofloxacin dry powder inhaler (DPI) for management of pulmonary infection due to Pseudomonas aeruginosa in cystic fibrosis (CF) patients. A similar designation has already been granted by the European Medicines Agency. Ciprofloxacin DPI is an investigational drug–device combination that combines ciprofloxacin dry powder formulated using Novartis' Proprietary PulmoSphere® technology with a delivery inhaler. Ciprofloxacin DPI is in Phase 2 development and is being studied for its safety and potential to improve lung function, as measured by the forced expiratory volume in 1 second (FEV1), in patients with CF.

BioRelix, Inc. (New Haven) and Dalton Medicinal Chemistry have jointly announced an expansion and renewal of their exclusive worldwide collaboration and agreement to discover and develop new therapeutics. Dalton will apply their proprietary insights to the design and synthesis of new classes of compounds against selected Riboswitch targets. BioRelix will advance these compounds with their array of biological assays and knowledge of Riboswitch drug discovery and development, the companies said.

Boehringer Ingelheim (Ingelheim, Germany/Ridgefield) continued to grow in 2009, increasing its net sales by 9.7% to EUR 12.7 billion. Even without taking into account positive currency effects, growth was once again above the average growth for the worldwide pharmaceutical market, at 6.7%. This means, the company said, that for the tenth consecutive time Boehringer Ingelheim grew faster than the pharmaceutical market and in 2009 was the fastest growing company of the world’s 15 largest pharmaceutical companies.

Boehringer Ingelheim received the CURE Award for Education at the CURE Annual Meeting April 27 in New Haven. (See story is this issue).

Bristol-Myers Squibb Company (New York/Wallingford) has authorized the repurchase of up to $3 billion of its common stock. The decision reflects the company’s strong financial position, the company said, including cash and marketable securities of $9.8 billion at the end of the first quarter, its positive outlook on its future cash position, and its focus on increasing shareholder value.

CMD Bioscience (Orange) announced that the US Patent Office has issued a notice of allowability for the provisional applications 60/785,232 and 60/878,217. "The key to successful structure-based peptide drug discovery is a rapid and accurate method for estimating and scoring protein-peptide binding affinities. The just granted Affinity Score 1.0 patent clearly affirms the innovativeness of the CMDBioscience methodology and confirms our leadership in computational peptide drug design and discovery," said CMDBioscience cofounder and CEO Dr. Joseph Audie.

Connecticut Innovations (Rocky Hill) announced that it has accepted NovaTract Surgical LLC of Guilford into its Pre-Seed Support Services Program. NovaTract is developing a medical device that will be used in single-incision laparoscopic surgery and natural orifice transluminal endoscopic surgery. Separately, CyVek of Wallingford said it had accepted $802,500 from Connecticut Innovations. The company is developing a platform to measure substances under chemical analysis in clinical diagnostic settings. Earlier, Connecticut Innovations announced it had invested $500,000 in Soft Tissue Regeneration of Stamford.

GlaxoSmithKline plc (Research Triangle Park, NC) and Human Genome Sciences, Inc. announced topline secondary endpoints from BLISS-76, the second of two pivotal Phase 3 trials of Benlysta™ (belimumab) in seropositive patients with systemic lupus erythematosus. Carlo Russo, M.D., Senior Vice President, Biopharm Development, GSK, said, “Based on the totality of data in BLISS-52 and BLISS-76, we believe that belimumab could deliver a significant therapeutic option for patients with lupus, a chronic condition which has a devastating effect on the lives of patients living with the disease.”

Hartford Hospital (Hartford) said that Anthem Blue Cross and Blue Shield of Connecticut has designated Hartford Hospital as a Blue Distinction Center for Spine SurgerySM and a Blue Distinction Center for Knee and Hip ReplacementSM. These designations are awarded to medical facilities that have demonstrated expertise in delivering quality health care.

HistoRx (New Haven) received a $1.5 million loan from Connecticut Innovations. The funding will be used to help HistoRx build out laboratory space and purchase laboratory equipment.

Ipsogen (Marseille, France/New Haven) said that Corinne Danan has joined the company as Vice President Commercial Operations. Prior to joining Ipsogen, she was European Director for Monogram Biosciences, a California company specializing in individualized medicine.

Johnson & Johnson (New Brunswick, NJ) said that the SEDASYS® System, the first computer-assisted personalized sedation system, was granted a CE Mark for the European Union for routine colonoscopy and screening of the upper gastrointestinal tract; and Health Canada granted regulatory approval for the SEDASYS® System for use by healthcare professional teams during routine colonoscopy.

MannKind Corporation (Valencia, CA/Danbury) completed Study 117, an efficacy study in patients with type 1 diabetes, and reports that the company's AFREZZA™ product met the primary endpoint of non-inferiority in HbA1C levels compared to standard insulin therapy.

MannKind received the CURE New Operations Award at the CURE Annual Meeting April 27 in New Haven. (See story is this issue).

MiraDx Inc. (New Haven) received $1 million from the Connecticut Innovations Eli Whitney Fund as part of a $4 million Series A round that also involved individual investors. MiraDx is applying microRNA (miRNA) discoveries licensed from Yale University to develop gene-based laboratory tests that will provide individualized information on the likelihood of disease occurrence and response to certain types of therapy. MiRNAs are small genetic regulators that control cell development, cell cycle, cell differentiation and cell death; they have also been linked to certain cancers and other disease states.

NanoVircides (West Haven) announced that its anti-Dengue drug candidates demonstrated significant efficacy in recently completed preliminary cell culture studies. The studies were performed in the laboratory of Dr. Eva Harris, Professor of Infectious Diseases at the University of California, Berkeley (UC Berkeley).

Separately, the company said its anti-HIV drug candidates demonstrated efficacy in recently completed cell culture studies using two distinctly different HIV-1 isolates. The studies were performed in the laboratory of Carol Lackman-Smith at the Southern Research Institute, Frederick, Maryland.

Earlier the company said it has received $5 million from Seaside 88, a Florida limited partnership that has financed several bio-pharma companies. "This financing positions us for meeting the listing standards of major exchanges," said Eugene Seymour, MD, MPH, who is CEO of the Company. "We now have sufficient cash in hand to meet our operating needs for the next eighteen months at the current rate of expenditure."

Pfizer Inc. (New York, NY/Groton/New London) and the World Bank announced they will collaborate to improve the healthcare infrastructure, specifically the supply chain, in developing countries, starting with Africa.

Pfizer received the CURE Award for Excellence at the CURE Annual Meeting April 27 in New Haven. (See story is this issue).

Vascular Insights LLC (Madison) said it has won approval for use of the CE Mark on its ClariVein®-OC catheter, paving the way for marketing of the device in Europe. ClariVein®-OC is an infusion catheter system intended for endovascular occlusion of incompetent veins in patients with superficial venous reflux. Infusion is through an opening at the distal end of the catheter and fluid delivery is enhanced by the use of a rotating dispersion wire to mix and disperse the infused fluid in the blood stream and on the vessel wall.

Following is recent news from The University of Connecticut (Storrs) and the University of Connecticut Health Center (Farmington).

Plans to renovate John Dempsey Hospital and build a new patient care tower at the UConn Health Center have won approval from the Connecticut legislature. "This is a significant legislative victory for the Health Center and the entire University," said Dr. Cato T. Laurencin, the Health Center’s vice president for health affairs and dean of the UConn School of Medicine. more

Dr. Justin Radolf, a professor in the Departments of Medicine and Genetics and Developmental Biology at UConn, has published a paper that evaluates the major protective antioxidant systems of the bacterium that causes syphilis. more

Amy Anderson and Dennis Wright, both associate professors of medicinal chemistry in the School of Pharmacy, are fighting a small, but potent enemy – methicillin-resistant Staphylococcus aureus, better known as MRSA. The research of this husband and wife research team focuses on the development of new drugs to treat MRSA infections as well as other infectious diseases. more

Improving care for patients at risk for osteoporosis; minimizing pain for patients with sickle cell disease; preventing central line infections for critically ill patients: these are the quality-improvement goals that fueled the work of three Health Center nurse practitioners who recently completed a rigorous new doctorate program in nursing practice. more

Dr. William B. White, professor of medicine and division chief of hypertension and pharmacology at the UConn Health Center’s Calhoun Cardiology Center, has published an expert commentary about the impact of the increase in blood pressure that occurs when we awake. more

When Kelci Stringer was looking for a home for a research institute honoring her late husband – All-Pro NFL lineman Korey Stringer – the University of Connecticut and its renowned kinesiology department were her first choice. Korey Stringer died from complications due to an exertional heat stroke he suffered during a Minnesota Vikings pre-season training camp in 2001. more

The University of Connecticut Neag School of Education, with the support of the National Football League and Gatorade, is opening a new institute on the Storrs campus to further research, education, and advocacy for the prevention of heat stroke and sudden death in sport. more

Following is recent news from Yale University and the Yale School of Medicine (New Haven).

Prions, not plaques, appear to be the cause of memory loss in mice with Alzheimer's, says Stephen Strittmatter, the Vincent Coates Professor of Neurology and professor of neurobiology at Yale. He is senior author of a new study; other Yale authors of the study are David Gimbel, Haakon Nygaard, Erin Coffey, Zachary Gimbel, and Erik Gunther.

A Yale-led team of scientists, using advanced DNA sequencing technology, has for the first time pinpointed individual variations of where, along the chain of 3 billion letters that make up the human genome, important gene regulatory elements do their jobs. Maya Kasowski, an M.D./Ph.D. candidate in Yale’s Department of Molecular, Cellular and Developmental Biology is co-lead author of the paper with Fabian Grubert, formerly a postdoctoral researcher at Yale now a post-doc at Stanford.

A team of Yale University scientists has discovered a previously unknown type of "molecular scissors" that can tailor micro-RNAs, tiny snippets of genetic material that play a key role in regulating many of life’s functions. "We are still just beginning to scratch the surface in our understanding of small RNAs,” said Antonio J. Giraldez, the Lois and Franklin H. Top, Jr. Yale Scholar in the genetics department of the Yale School of Medicine and senior author of the study."

A single, very unusual family with Tourette syndrome (TS) has led Yale School of Medicine researchers to identify a rare mutation in a gene that is required to produce histamine. The finding provides a new framework to understand many years of data on the role of histamine function in the brain and points to a potentially novel approach to treatment of tics and Tourette. The team was led by Matthew State, M.D., the Donald J. Cohen Associate Professor in the Yale Child Study Center and in the Departments of Psychiatry and Genetics, and co-director of the  Yale Program on Neurogenetics.

A Yale-led study reveals, for the first time, the process by which a particular genetic mutation causes seizures and ataxia, the neurodegenerative disorder believed to have afflicted Abraham Lincoln. Senior author is Jon S. Morrow, M.D., Ph.D., professor of pathology and molecular, cellular and developmental biology.

Telomeres — the repeat sequences of DNA at the end of chromosomes — are much more likely to be damaged by UV radiation than are other common cellular structures, Yale researchers report. Douglas Brash, professor of therapeutic radiology, genetics and dermatology, and a researcher for the Yale Cancer Center, is senior author of the study.

Yale University engineers have found that the defects in carbon nanotubes cause T cell antigens to cluster in the blood and stimulate the body’s natural immune response. Their findings could improve current adoptive immunotherapy, a treatment used to boost the body’s ability to fight cancer. Tarek Fahmy, associate professor of chemical engineering and biomedical engineering, is senior author of the paper.

Researchers at Yale School of Medicine have discovered that exposure during pregnancy to Bisphenol A (BPA), a common component of plastics, causes permanent abnormalities in the uterus of offspring, including alteration in their DNA. Led by Hugh S. Taylor, M.D., professor in the Department of Obstetrics, Gynecology & Reproductive Sciences at Yale, the study is the first to show that BPA exposure permanently affects sensitivity to estrogen.

A Yale team compared the evolution of organisms and computer operating systems by analyzing the control networks in both a bacterium Escherichia coli and the Linux operating system. Mark Gerstein, the Albert L. Williams Professor of Biomedical Informatics is senior author of the paper.

Depression among low-income mothers could last well beyond the postpartum period and become a chronic condition, suggests a new study by researchers at Yale School of Medicine. Lead author is Carol C. Weitzman, M.D., associate professor of pediatrics.

Children with asthma are more likely to develop a range of health and social problems as they enter into adulthood, new research by the Yale School of Public Health has found. Lead author is Jason M. Fletcher, an assistant professor in the division of Health Policy and Administration.

Jon Soderstrom of Yale's Office of Cooperative Research received the CURE Atlas Award for Venture Capital Achievementat the CURE Annual Meeting April 27 in New Haven. (See story is this issue).

For more member news, see the April 2010 issue of CURE News

 
 
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