Archive for the ‘Gene Therapy Research’ Category

SAN DIEGO and IRVINE, Calif., Aug. 1, 2013 /PRNewswire/ -- Sequenom, Inc. (SQNM), a life sciences company providing innovative diagnostic testing and genetic analysis solutions, and CombiMatrix Corporation (CBMX), a molecular diagnostics laboratory performing DNA-based testing services for developmental disorders and cancer, today jointly announced that the Sequenom Center for Molecular Medicine LLC (Sequenom CMM) and CombiMatrix have entered into a collaboration agreement to market chromosomal microarray analysis (CMA) testing services to broaden and confirm the results of noninvasive, prenatal testing (NIPT) to physicians and their patients.

Under the agreement, the two laboratories will collaboratively use their respective marketing channels and sales forces to promote the use of NIPT and CMA and work together to provide technical training to physicians and counseling, education and support services to physicians and their patients. The two companies plan to provide, when clinically appropriate, a comprehensive test result report for ordering physicians.

Sequenom is a global leader in the NIPT market, and Sequenom CMM, its wholly owned subsidiary laboratory, developed the MaterniT21TM PLUS laboratory-developed test (LDT), a widely-ordered, noninvasive prenatal testing service for fetal chromosomal abnormalities. CombiMatrix is one of the few independent laboratories that specialize in CMA, the primary genetic test to evaluate newborns with birth defects that is now being more widely used for prenatal testing.

"The MaterniT21 PLUS test is the premier, noninvasive prenatal test on the market today, and for an important group of patients a chromosomal microarray test can be a significant adjunct for a comprehensive prenatal analysis," said Bill Welch, President and COO of Sequenom, Inc. "CombiMatrix specializes in CMA and their technology helps further expand our product offering in the growing prenatal genetic testing marketplace."

"The collaboration with Sequenom CMM is a significant validation of the rapidly expanding acceptance of CMA as a standard of prenatal care as well as an endorsement of the services CombiMatrix provides," noted CombiMatrix CEO Mark McDonough.

"We are focused on establishing CombiMatrix as the premier specialty laboratory for chromosomal microarray analysis for prenatal testing," McDonough said. "The fact that Sequenom CMM has joined forces with us and selected us for this collaborative testing relationship demonstrates that we have made great strides in reaching our goal."

The MaterniT21 PLUS test analyzes the relative amount of 21, 18, 13, as well as X and Y chromosomal material in cell-free DNA. The test is intended for use in pregnant women at increased risk for fetal aneuploidy and can be used as early as 10 weeks' gestation.Estimates suggest there are about 750,000 pregnancies at increased risk for fetal aneuploidy each year in the United States. The MaterniT21 PLUS test is available exclusively through the Sequenom CMM as a testing service provided to physicians. To learn more about the test, please visit http://www.Sequenomcmm.com.

About SequenomSequenom, Inc. (SQNM) is a life sciences company committed to improving healthcare through revolutionary genetic analysis solutions. Sequenom develops innovative technology, products and diagnostic tests that target and serve discovery and clinical research, and molecular diagnostics markets. The company was founded in 1994 and is headquartered in San Diego, California. Sequenom maintains a Web site at http://www.sequenom.com to which Sequenom regularly posts copies of its press releases as well as additional information about Sequenom. Interested persons can subscribe on the Sequenom Web site to email alerts or RSS feeds that are sent automatically when Sequenom issues press releases, files its reports with the Securities and Exchange Commission or posts certain other information to the Web site.

About Sequenom Center for Molecular MedicineSequenom Center for Molecular Medicine (Sequenom CMM) is a CAP accredited and CLIA-certified molecular diagnostics reference laboratory currently with three locations dedicated to the development and commercialization of laboratory developed tests for prenatal and eye conditions and diseases. Utilizing innovative proprietary technologies, Sequenom CMM provides test results that can be used by health care professionals in managing patient care. Testing services are available only upon request by physicians. Sequenom CMM works closely with key opinion leaders and experts in obstetrics, retinal care and genetics. Sequenom CMM scientists use a variety of sophisticated and cutting-edge methodologies in the development and validation of tests. Sequenom CMM is changing the landscape in genetic diagnostics. Visit http://www.sequenomcmm.com for more information on laboratory testing services.

SEQUENOM, MaterniT21 and MaterniT21 PLUS are trademarks of Sequenom, Inc. All other trademarks and service marks are the property of their respective owners.

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Sequenom And CombiMatrix Announce Collaboration Agreement To Market Chromosomal Microarray Analysis For Prenatal Testing

Public release date: 31-Jul-2013 [ | E-mail | Share ]

Contact: Mount Sinai Press Office newsmedia@mssm.edu 212-241-9200 The Mount Sinai Hospital / Mount Sinai School of Medicine

The Icahn School of Medicine at Mount Sinai is partnering with the Institute for Family Health to launch the first-ever genetic testing program in the primary care setting to identify genetic risk for kidney disease in patients with hypertension.

The program will be funded through a $3.7 million grant from the National Human Genome Research Institute of the National Institutes of Health. Primary-care providers will use patients' genomic information at the point-of-care to individualize treatment, testing and monitoring with Mount Sinai's Clinical Implementation of Personalized Medicine through Electronic Health Records and Genomic Program, or CLIPMERGE, a novel clinical-decision support engine for delivering guidelines with genetic variants of clinical significance to enhance treatment.

Recent research has shown that one in eight African Americans have two copies of a version of the APOL1 gene, putting them at four to five times greater risk for developing chronic kidney disease or end-stage kidney disease if they have hypertension, or high blood pressure.

"Many patients do not have their blood pressure adequately controlled," said Erwin Bottinger, MD, Director of the Charles Bronfman Institute for Personalized Medicine at the Icahn School of Medicine at Mount Sinai, and one of two principal investigators of the grant. "We believe that with genomic information made available to doctors through a patient's electronic health record, we will be able to achieve better and stricter control of blood pressure and targeted use of medications that inhibit the renin angiotensin system, which are recommended in hypertensive patients at risk for kidney disease. More comprehensive tracking will also help ensure that optimal tests will be performed to stop progression of kidney disease."

A cluster-randomized controlled trial will be conducted at 12 primary care sites in New York, including practices at The Mount Sinai Medical Center and the Institute for Family Health, which operates an independent network of community health centers in Manhattan and the Bronx.

"Genes are another piece of the puzzle that may help explain why people of African descent have poorer health outcomes than people of European descent," said Carol Horowitz, MD, MPH, co-principal investigator and co-director of the Icahn School of Medicine at Mount Sinai's new Center for Health Equity and Community Engaged Research. "We look forward to engaging with and helping educate our multicultural community partners, providers, and patients about the emerging role genetic testing will play in improving health."

Neil Calman, MD, President and Chief Executive Officer of the Institute for Family Health, and Professor and Chair of Family Medicine and Community Health at Mount Sinai said, "Community-based primary care physicians have had little opportunity to incorporate genomics into the care of patients, and this grant offers us a tremendous opportunity. We hope to screen patients, identify those with increased genetic risk and work with them to prevent kidney disease. We will also train community-based primary care providers in how to discuss genetic risk with patients and their families and how to use genetic-based information in the electronic health record."

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Mount Sinai launches first-ever genetic testing program in the primary care setting

Public release date: 30-Jul-2013 [ | E-mail | Share ]

Contact: Shaun Mason smason@mednet.ucla.edu 310-206-2805 University of California - Los Angeles

UCLA scientists, in collaboration with teams in China, have used the powerful technology of single-cell RNA sequencing to track the genetic development of a human and a mouse embryo at an unprecedented level of accuracy.

The technique could lead to earlier and more accurate diagnoses of genetic diseases, even when the embryo consists of only eight cells.

The study was led by Guoping Fan, professor of human genetics and molecular biology and member of both the Jonsson Comprehensive Cancer Center and the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research. The findings were published in the online edition of the journal Nature and will appear later in the print edition.

Single-cell RNA sequencing allows researchers to determine the precise nature of the total gene transcripts, or all of the genes that are actively expressed in a particular cell.

"The advantages of this technique are twofold," Fan said. "It is a much more comprehensive analysis than was achievable before and the technique requires a very minimal amount of sample material just one cell."

Besides its implications for genetic diagnoses such as improving scientists' ability to identify genetic mutations like BRCA1 and BRCA2, which predispose women to breast cancer and ovarian cancer, or genetic diseases that derive from protein dysfunction, such as sickle cell disease the technology may also have important uses in reproductive medicine.

The technique marks a major development in genetic diagnoses, which previously could not be conducted this early in embryonic development and required much larger amounts of biological material.

"Previous to this paper we did not know this much about early human development," said Kevin Huang, the study's co-first author and a postdoctoral scholar in Fan's laboratory. "Now we can define what 'normal' looks like, so in the future we will have a baseline from which to compare possible genetic problems. This is our first comprehensive glance at what is normal."

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UCLA and Chinese scientists analyze genetic makeup of human and mouse embryos in amazing detail

July 31, 2013 Recent technological developments in genomics have revealed a large number of genetic influences on common complex diseases, such as diabetes, asthma, cancer or schizophrenia. However, discovering a genetic variant predisposing to a disease is only a first step. To apply this knowledge towards prevention or cure, including tailoring treatment to the patient's genetic profile -also known as personalized medicine -- we need to know how this genetic variant affects health.

In a study published today in Nature Communications, Dr. Constantin Polychronakos from the Research Institute of the McGill University Health Centre (RI-MUHC), and collaborators from McGill University and The University of Texas, propose a novel approach for scanning the entire genome that will help us understand the effect of genes on human traits.

"This completely new methodology really opens up different ways of understanding how the genome affects the biology of the human body," says Dr. Polychronakos, corresponding author of the study and Director of the Endocrine Genetics Laboratory at the Montreal Children's Hospital and Professor in the Departments of Pediatrics and Human Genetics at McGill University.

DNA is the blueprint according to which our body is constructed and functions. Cells "read" this blueprint by transcribing the information into RNA, which is then used as a template to construct proteins -- the body's building blocks. Genes are scanned based on the association of their RNA with ribosomes -- particles in which protein synthesis takes place.

"Until now, researchers have been focusing on the effects of disease-associated genomic variants on DNA-to-RNA transcription, instead of the challenging question of effects on RNA-to-protein translation," says Dr. Polychronakos. "Thanks to this methodology, we can now better understand the effect of genetic variants on translation of RNA to protein -- a powerful way of developing biomarkers for personalized medicine and new therapies."

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Understanding the effects of genes on human traits

July 31, 2013 UCLA scientists, in collaboration with teams in China, have used the powerful technology of single-cell RNA sequencing to track the genetic development of a human and a mouse embryo at an unprecedented level of accuracy.

The technique could lead to earlier and more accurate diagnoses of genetic diseases, even when the embryo consists of only eight cells.

The study was led by Guoping Fan, professor of human genetics and molecular biology and member of both the Jonsson Comprehensive Cancer Center and the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research. The findings were published in the online edition of the journal Nature and will appear later in the print edition.

Single-cell RNA sequencing allows researchers to determine the precise nature of the total gene transcripts, or all of the genes that are actively expressed in a particular cell.

"The advantages of this technique are twofold," Fan said. "It is a much more comprehensive analysis than was achievable before and the technique requires a very minimal amount of sample material -- just one cell."

Besides its implications for genetic diagnoses -- such as improving scientists' ability to identify genetic mutations like BRCA1 and BRCA2, which predispose women to breast cancer and ovarian cancer, or genetic diseases that derive from protein dysfunction, such as sickle cell disease -- the technology may also have important uses in reproductive medicine.

The technique marks a major development in genetic diagnoses, which previously could not be conducted this early in embryonic development and required much larger amounts of biological material.

"Previous to this paper we did not know this much about early human development," said Kevin Huang, the study's co-first author and a postdoctoral scholar in Fan's laboratory. "Now we can define what 'normal' looks like, so in the future we will have a baseline from which to compare possible genetic problems. This is our first comprehensive glance at what is normal."

With single-cell RNA sequencing, much more gene transcription was detected than before. "The question we asked is, 'How does the gene network drive early development from one cell to two cells, two cells to four cells, and so on?'" Fan said. "Using the genome data analysis methods developed by co-author Steve Horvath at UCLA, we have uncovered crucial gene networks and we can now predict possible future genetic disorders at the eight-cell stage."

The research was supported by the Chinese Ministry of Science and Technology, the International Science and Technology Cooperation Program of China, and the National Natural Science Foundation of China.

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Scientists analyze genetic makeup of human and mouse embryos in amazing detail

Newswise The Icahn School of Medicine at Mount Sinai is partnering with the Institute for Family Health to launch the first-ever genetic testing program in the primary care setting to identify genetic risk for kidney disease in patients with hypertension.

The program will be funded through a $3.7 million grant from the National Human Genome Research Institute of the National Institutes of Health. Primary-care providers will use patients genomic information at the point-of-care to individualize treatment, testing and monitoring with Mount Sinais Clinical Implementation of Personalized Medicine through Electronic Health Records and Genomic Program, or CLIPMERGE, a novel clinical-decision support engine for delivering guidelines with genetic variants of clinical significance to enhance treatment.

Recent research has shown that one in eight African Americans have two copies of a version of the APOL1 gene, putting them at four to five times greater risk for developing chronic kidney disease or end-stage kidney disease if they have hypertension, or high blood pressure.

Many patients do not have their blood pressure adequately controlled, said Erwin Bottinger, MD, Director of the Charles Bronfman Institute for Personalized Medicine at the Icahn School of Medicine at Mount Sinai, and one of two principal investigators of the grant. We believe that with genomic information made available to doctors through a patients electronic health record, we will be able to achieve better and stricter control of blood pressure and targeted use of medications that inhibit the renin angiotensin system, which are recommended in hypertensive patients at risk for kidney disease. More comprehensive tracking will also help ensure that optimal tests will be performed to stop progression of kidney disease.

A cluster-randomized controlled trial will be conducted at 12 primary care sites in New York, including practices at The Mount Sinai Medical Center and the Institute for Family Health, which operates an independent network of community health centers in Manhattan and the Bronx.

Genes are another piece of the puzzle that may help explain why people of African descent have poorer health outcomes than people of European descent, said Carol Horowitz, MD, MPH, co-principal investigator and co-director of the Icahn School of Medicine at Mount Sinai's new Center for Health Equity and Community Engaged Research. We look forward to engaging with and helping educate our multicultural community partners, providers, and patients about the emerging role genetic testing will play in improving health.

Neil Calman, MD, President and Chief Executive Officer of the Institute for Family Health, and Professor and Chair of Family Medicine and Community Health at Mount Sinai said, Community-based primary care physicians have had little opportunity to incorporate genomics into the care of patients, and this grant offers us a tremendous opportunity. We hope to screen patients, identify those with increased genetic risk and work with them to prevent kidney disease. We will also train community-based primary care providers in how to discuss genetic risk with patients and their families and how to use genetic-based information in the electronic health record.

About the Charles Bronfman Institute for Personalized Medicine The Charles Bronfman Institute for Personalized Medicine at the Icahn School of Medicine at Mount Sinai aims to refine disease risk assessment and improve responsiveness to treatments through genomic medicine and data-driven care.

About The Center for Health Equity and Community Engaged Research Founded in 2012, The Center for Health Equity and Community Engaged Research's mission is to improve the health and health care of underserved populations by identifying causes of disparities in health and health care; developing and testing community-partnered, sustainable interventions; and disseminating lessons learned to inform policy and systems change.

About The Institute for Family Health The Institute for Family Health is one of the largest networks of community health centers in the country. The Institute is committed to high-quality, affordable health care for all, regardless of ability to pay, and remains committed to primary care research that informs health policy and practice change.

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Genetic Testing for Kidney Disease Among African-Americans With Hypertension to Become Part of Primary Care Offerings ...

LOS ANGELES, July 31, 2013 (GLOBE NEWSWIRE) -- Response Genetics, Inc. (RGDX), a company focused on the development and sale of molecular diagnostic tests that help determine a patient's response to cancer therapy, will announce its second quarter 2013 financial results and give an operational update in a press release to be issued before the market opens on Thursday, August 8, 2013. The company will host a conference call that same day at 10:00 a.m. EDT to discuss its financial results.

CONFERENCE CALL DETAILS

To access the conference call by phone on August 8 at 10:00 a.m. EDT, dial (800) 537-0745 or (253) 237-1142 for international participants. A telephone replay will be available beginning approximately two hours after the call through August 10, 2013, and may be accessed by dialing (855) 859-2056 or (404) 537-3406. The conference passcode for both the live call and replay is 25563258.

To access the live and archived webcast of the conference call, go to the Investor Relations section of the Company's Web site at http://investor.responsegenetics.com. It is advised that participants connect at least 15 minutes prior to the call to allow for any software downloads that might be necessary.

About Response Genetics, Inc.

Response Genetics, Inc. (the "Company") is a CLIA-certified clinical laboratory focused on the development and sale of molecular diagnostic testing services for cancer. The Company's technologies enable extraction and analysis of genetic information derived from tumor cells stored as formalin-fixed and paraffin-embedded specimens. The Company's principal customers include oncologists and pathologists. In addition to diagnostic testing services, the Company generates revenue from the sale of its proprietary analytical pharmacogenomic testing services of clinical trial specimens to the pharmaceutical industry. The Company's headquarters is located in Los Angeles, California. For more information, please visit http://www.responsegenetics.com.

Forward-Looking Statement Notice

Except for the historical information contained herein, this press release and the statements of representatives of the Company related thereto contain or may contain, among other things, certain forward-looking statements, within the meaning of the Private Securities Litigation Reform Act of 1995.

Such forward-looking statements involve significant risks and uncertainties. Such statements may include, without limitation, statements with respect to the Company's plans, objectives, projections, expectations and intentions, such as the ability of the Company, to provide clinical testing services to the medical community, to continue to strengthen and expand its sales force, to continue to build its digital pathology initiative, to attract and retain qualified management, to continue to strengthen marketing capabilities, to expand the suite of ResponseDX(R) products, to continue to provide clinical trial support to pharmaceutical clients, to enter into new collaborations with pharmaceutical clients, to enter into areas of companion diagnostics, to continue to execute on its business strategy and operations, to continue to analyze cancer samples and the potential for using the results of this research to develop diagnostic tests for cancer, the usefulness of genetic information to tailor treatment to patients, and other statements identified by words such as "project," "may," "could," "would," "should," "believe," "expect," "anticipate," "estimate," "intend," "plan" or similar expressions.

These statements are based upon the current beliefs and expectations of the Company's management and are subject to significant risks and uncertainties, including those detailed in the Company's filings with the Securities Exchange Commission. Actual results, including, without limitation, actual sales results, if any, or the application of funds, may differ from those set forth in the forward-looking statements. These forward-looking statements involve certain risks and uncertainties that are subject to change based on various factors (many of which are beyond the Company's control). The Company undertakes no obligation to publicly update forward-looking statements, whether because of new information, future events or otherwise, except as required by law.

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Response Genetics, Inc. to Release Second Quarter 2013 Financial Results and Host Conference Call on August 8, 2013

Seattle Genetics, Inc.(SGEN) reported second quarter 2013 net loss per share of 6 cents, narrower than the Zacks Consensus Estimate of a loss of 18 cents and the year-ago loss of 15 cents per share.

Second quarter revenues were $73.6 million, compared with $48.8 million in the year-ago quarter. Revenues surpassed the Zacks Consensus Estimate of $57 million.

Net revenues for the second quarter included Adcetris revenues (up 3% to $35.7 million), collaboration and license agreement revenues (up 165.9% to $34.3 million) and royalty revenues (up 185.9% to $3.5 million).

Research and development expenses increased 22.3% year over year to $52.3 million driven by Adcetris development activities and higher investment in other antibody-drug conjugate (ADC) programs. Selling, general and administrative (SG&A) expenses rose by 18.5% year over year to $23.5 million. SG&A expenses increased due to Adcetris-related sales and marketing activities.

Pipeline Update

Seattle Genetics is working on expanding Adcetris' label. In May 2013, Seattle Genetics submitted a supplemental biologics license application (sBLA) for the use of Adcetris in the retreatment of patients and for extended duration of use beyond 16 cycles of therapy. The U.S. Food and Drug Administration (FDA) did not approve Adcetris in this retreatment setting. Seattle Genetics will further interact with the FDA regarding the new data required for approval.The FDA has asked to remove the limitation of 16-cycles timeframe from the label.

Adcetris is approved for the treatment of relapsed or refractory Hodgkin lymphoma (HL) and systemic anaplastic large cell lymphoma (sALCL).

Meanwhile, Seattle Genetics discontinued the development of ASG-5ME (phase I) for prostate, gastric and pancreatic cancer.

Seattle Genetics has collaborations with various companies for the development of ADCs. ADC collaborators are progressing on various programs.

Seattle Genetics and Bayer (BAYRY) entered into an alliance in Jun 2013. This deal will allow Bayer to gain worldwide rights to Seattle Genetics' auristatin-based ADC technology along with antibodies to several oncology targets.

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Narrower-than-Expected Loss at Seattle Genetics - Analyst Blog

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Seattle Genetics reports narrower 2Q loss

ALISO VIEJO, Calif.--(BUSINESS WIRE)--

Ambry Genetics, the worldwide leader in hereditary cancer testing, announced today that it has officially launched its next-generation sequencing (NGS) renal, pancreatic and paraganglioma-pheochromocytoma (PGL/PCC) hereditary cancer panels.

Ambry Genetics was the first commercial laboratory to launch hereditary cancer panels using NGS in 2012 with their comprehensive BreastNext, OvaNext, ColoNext and CancerNext products. With hundreds of clinics and institutions utilizing these panels, Ambry has established itself as the scientific leaders in multi-gene cancer panel testing.

As of today, Ambry Genetics continues to offer the most comprehensive hereditary cancer testing available, said Elizabeth Chao, MD, Chief Medical Officer of Ambry Genetics. As one example, pancreatic cancer is one of the deadliest and most incurable forms of the disease. Our test analyzes 13 genes simultaneously, and identifying a mutation in any one of those genes can lead to treatment options, including prevention. Thats what this type of genetic testing can offer.

Ambrys new offerings include an 18 gene renal cancer panel (RenalNext), 13 gene pancreatic cancer panel (PancNext) and 10 gene PGL/PCC panel (PGLNext). Previously, many of the genes on these panels could be ordered individually using traditional Sanger sequencing. The introduction of Ambrys new NGS panel offerings allows all causative genes to be tested concurrently, saving time and money.

I cant imagine a more exhilarating place to be right now, said AJ Stuenkel, Certified Genetic Counselor at Ambry Genetics. Ambry is buzzing with excitement and a sense of purpose as we embrace recent scientific advances and continue to introduce novel testing options at an unmatched pace.

About Ambry Genetics

Ambry Genetics is a College of American Pathologists (CAP)-accredited and Clinical Laboratory Improvement Amendments (CLIA)-certified commercial clinical laboratory with headquarters in Orange County, California. Since its founding in 1999, it has become a leader in providing genetic services focused on clinical diagnostics and genomic services, particularly in sequencing and array services. Ambry has established a reputation for unparalleled service and for over a decade has been at the forefront of applying new technologies to the clinical molecular diagnostics market and to the advancement of disease research. To learn more about testing and services available through Ambry Genetics, visit http://www.AmbryGen.com.

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Ambry Genetics Reaffirms Status as Worldwide Leader in Hereditary Cancer Testing with New Renal, Pancreatic and PGL ...

Seattle Genetics (NASDAQ: SGEN) announced its results from the second quarter after the market closed on Wednesday. Here are three things you need to know from the the latest update.

1. Adcetris commercialization is advancing nicely.The company's mainstay product, Adcetris, is now approved in 35 countries with Seattle Genetics' partner Takeda/Millenium recently obtaining a regulatory thumbs-up in South Korea. Takeda also submitted for approval in Japan earlier this year.

Total revenue for the second quarter was $73.6 million, with product sales for Adcetris comprising $35.7 million. Royalties from Takeda/Millenium made up an additional $3.5 million. Net sales for the drug -- which is used to treatHodgkin lymphoma andanaplastic large cell lymphoma, or ALCL -- climbed 5% year-over-year.

Seattle Genetics' data shows that Adcetris now has a penetration rate of more than 70%. The company feels comfortable in stating that the drug has now become the standard of care for ALCL and Hodgkin lymphoma. It also has noticed increasing use of Adcetris for off-label uses, although this isn't considered to be a significant part of the business at this point.

Recent discussions with the U.S. Food and Drug Administration led to good news and bad news. The good news is that the company thinks that the FDA will allow it to remove the 16-cycle limitation on duration of use from the Adcetris label. The bad news is that the FDA didn't approve including use of the drug in a retreatment setting. Seattle Genetics intends to pursue the retreatment option further with the agency.

2. The pipeline is percolating.Seattle Genetics counts nine clinical studies under way for Adcetris with the hopes of expanding indications for the drug. Four of these are late-stage studies, four are mid-stage, and one is in phase 1. The company also has four other drugs in phase 1 studies.

The ongoing phase 3 study of Adcetris in treating relapsed diffused large B-cell lymphoma, or DLBCL, shows perhaps the most promise right now. Interim data found a response rate of 44%, with 81% of patients achieving tumor reduction. These positive results have prompted the company to initiate a phase 2 study for newly diagnosed DLBCL patients.

CEO Clay Siegall addressed one concern about Hodgkin lymphoma patients taking Adcetris who developed pancreatitis. Siegall said that the incidence of pancreatitis in all patients treated with Adcetris is 0.16%. He stated that this relatively low risk shouldn't impact plans for Adcetris and noted that several other cancer drugs on the market have been linked to an increased risk of pancreatitis.

3. Collaboration continues.Nearly $34.3 million of Seattle Genetics' total revenue stemmed from payments from collaboration partners. $12 million of this figure was from a new partnership with Bayer Healthcare. Several other large companies have been attracted to Seattle Genetics' antibody-drug conjugate, or ADC, technology in recent years.

AbbVie (NYSE: ABBV) inked a potential $245 million deal with Seattle Genetics last year. The arrangement included an upfront payment of $25 million and potential milestone payments up to $220 million. This expanded an agreement between the two companies made in 2011.

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3 Things You Need to Know From Seattle Genetics' Q2 Update

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

Seattle Genetics, Inc. (SGEN) today reported financial results for the second quarter and six months ended June 30, 2013. The company also highlighted ADCETRIS (brentuximab vedotin) commercialization and clinical development activities, antibody-drug conjugate (ADC) pipeline and collaborator updates and upcoming milestones.

During the second quarter, we continued to make strong progress on our key corporate priorities, including the commercialization of ADCETRIS, the broad clinical development of ADCETRIS in CD30-positive malignancies and the advancement of our product pipeline, said Clay B. Siegall, Ph.D., President and Chief Executive Officer at Seattle Genetics. ADCETRIS is now approved in 35 countries, and our clinical development program comprises more than 20 ongoing clinical trials, including four phase 3 studies. We recently expanded our clinical-stage product pipeline by moving SGN-CD33A, which utilizes our newest ADC technology, into a phase 1 trial and exercising our option to co-develop ASG-15ME with Agensys/Astellas. We also entered into a new ADC collaboration with Bayer.

Recent ADCETRIS Activities

ADCETRIS is currently not approved for use in frontline HL, frontline MTCL, CTCL, first relapse HL patients eligible for a transplant or the B-cell and T-cell non-Hodgkin lymphoma subtypes referenced above.

ADC Pipeline Updates

ADC Collaborator Highlights

Upcoming Milestones

Second Quarter and Six Months Financial Results

Total revenues in the second quarter of 2013 were $73.6 million, compared to $48.8 million in the second quarter of 2012. Revenues for the six month period ended June 30, 2013 were $130.9 million, compared to $97.1 million for the same period in 2012. Revenues in 2013 included ADCETRIS net product sales of $35.7 million in the second quarter and $69.7 million for the year-to-date. Revenues in 2013 also included ADCETRIS royalty revenues of $3.5 million for the second quarter and $5.9 million for the year-to-date from international sales of ADCETRIS by Takeda/Millennium. Revenues also reflect amounts earned under the companys ADCETRIS and ADC collaborations totaling $34.3 million in the second quarter of 2013 and $55.3 for the year-to-date in 2013. Collaboration revenues increased in 2013 driven by the companys ADCETRIS collaboration with Takeda/Millennium as well as ADC collaborations, including the new collaboration with Bayer.

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Seattle Genetics Reports Second Quarter 2013 Financial Results

Part 2 Stem Cell Therapy

By: Tiffany Vinson

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Part 2 Stem Cell Therapy - Video

American scientists said individuals who derive their happiness from a sense of purpose showed favourable gene-expression profiles in their immune cells UCLA research found people with high levels of the type of happiness that comes from consuming goods showed weak antiviral genes Doing good and feeling good have very different effects on the human genome, even though they generate similar levels of positive emotion

By Sarah Griffiths

PUBLISHED: 11:39 EST, 30 July 2013 | UPDATED: 11:41 EST, 30 July 2013

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People who derive their happiness from helping others, like Mother Teresa, have strong antibody genes, UCLA scientists claim

American scientists have found different types of happiness have surprisingly contrary effects on our genes.

UCLA research found that people who derive their happiness from helping others have strong antibody genes, while people who get their kicks from self-gratification can suffer from low antiviral and anitbody gene expression.

The study, which also involved the University of North Carolina, is the first of its kind to examine how positive psychology impacts human gene expression.

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Be happy - your genes will thank you for it: Doing good leads to strong immune cells

Rapidly identify meaningful results with OGT's Genefficiency NGS Report

Oxford, UK Oxford Gene Technology (OGT), provider of innovative genetics research and biomarker solutions to advance molecular medicine, is offering the research community a snapshot of its user-friendly Genefficiency NGS Variant Analysis Report software, with the release of a free-to-download demo version. The interactive report efficiently manages the complete data analysis process for NGS workflows and delivers fully annotated data, allowing the identification of meaningful results in just a few simple mouse clicks.

The demo allows researchers to explore the easy-to-use features of the software with publicly-available sequence data from Tariq et al (2011)1, which identified a novel candidate gene (SHROOM3) for heterotaxy, a severe form of congenital heart disease. It provides researchers with a unique opportunity to experience the interactive report format, discovering what's possible with OGT's Genefficiency NGS Services. The report offers rapid filtering options, built-in quality control metrics and the ability to retrospectively analyse the data with additional or new selection criteria.

On completion of all OGT Genefficiency NGS exome and targeted panel projects, customers receive their results pre-loaded into the Genefficiency NGS Variant Analysis Report, so that they can quickly and easily identify relevant data. OGT's services cover the complete NGS pipeline, from project conception through to the delivery of high-quality, meaningful results via the software, bypassing the bottleneck of complex data analysis.

The Genefficiency NGS Variant Analysis Report is an intuitive solution to NGS data analysis and reporting. Thousands of variations can be filtered within minutes to just a handful requiring further validation with no requirement for additional bioinformatics resources. Bespoke analysis is easily integrated into the reporting software, allowing variants to be filtered based on biological function or other tailored criteria, for the easy identification of the most significant regions of genetic variation relevant to each individual study. Furthermore, the readily accessible in-depth quality metrics provide maximum confidence that the results are of the highest quality.

To download the demo, register here. For more information on OGT's Genefficiency services contact OGT or visit the website. References: 1. Tariq M, et al (2011). SHROOM3 is a novel candidate for heterotaxy identified by whole exome sequencing. Genome Biol. Sep 21;12(9):R91. doi: 10.1186/gb-2011-12-9-r91.

For further information, please contact: Oxford Gene Technology Begbroke Science Park Begbroke Hill Woodstock Road Begbroke, Oxfordshire, OX5 1PF, U.K T: +44 (0) 1865 856826 F: +44 (0) 1865 848684 E: contact@ogt.com W: http://www.ogt.com Twitter: @OxfordGeneTech

About Oxford Gene Technology Founded by Professor Ed Southern, Oxford Gene Technology (OGT) provides innovative genetics research and biomarker solutions to advance molecular medicine. The company has two trading businesses: Genomics comprises of CytoSure cytogenetics array, labelling and interpretation software products and services for the detection of chromosomal abnormalities, and Genefficiency Genomic Services, a unique combination of platforms, expertise and processing capabilities to deliver rapid, relevant genomic data. The Biomarkers business utilises proprietary next generation technologies to build a rich patent-protected portfolio of promising biomarkers for early stage cancer detection including advanced programmes in colorectal and prostate cancer plus the autoimmune disease systemic lupus erythematosus.

CytoSure and Genefficiency NGS browser/report: For Research Use Only; Not for Use in Diagnostic Procedures

CytoSure: This product is provided under an agreement between Agilent Technologies, Inc., and OGT. The manufacture, use, sale or import of this product may be subject to one or more of U.S. patents, pending applications, and corresponding international equivalents, owned by Agilent Technologies, Inc. The purchaser has the non-transferable right to use and consume the product for RESEARCH USE ONLY AND NOT for DIAGNOSTICS PROCEDURES. It is not intended for use, and should not be used, for the diagnosis, prevention, monitoring, treatment or alleviation of any disease or condition, or for the investigation of any physiological process, in any identifiable human, or for any other medical purpose.

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Free NGS Report Demo - Bypass the Data Bottleneck with Genefficiency Services

July 30, 2013

April Flowers for redOrbit.com Your Universe Online

A team of scientists from The Research Institute at Nationwide ChildrensHospital has found a mutation in a gene crucial to normal heart development could play a role in some types of congenital heart defects the most common birth defect in the US. The findings were published in the journal Human Mutation and could help narrow the search for genes that contribute to this genetic defect, which affects as many as 40,000 newborns a year.

So far, several hundred genes have been implicated in the formation of the heart. Mutation in any one of those genes could potentially contribute to a cardiac defect. Vidu Garg, MD, is the principal investigator in the Center for Cardiovascular and Pulmonary Research and director of Translational Research in The Heart Center at Nationwide Childrens. He says identifying which of these genes is responsible for human congenital heart disease has been a challenge for researchers.

We have to ask ourselves, what subset of the more than 20,000 genes that make up the human genome are contributing to congenital heart disease? he says. Right now, we dont know enough about a lot of those genes, so this study provides another piece of the puzzle.

That puzzle piece is the so-called FOXP1 gene, a member of a large gene family that helps regulate tissues throughout the body, including in the heart, lungs and brain. Previous studies of FOXP1 have described its function and role in the development of the heart in animal models, but it took a former colleague calling up Dr. Garg to report the results of particularly interesting case to make him take a closer look at the gene.

Linda Baker, MD, from the University of Texas Southwestern Medical Center, found a rare genetic abnormality while analyzing a DNA sample from an 8-month-old infant who died from complications of complex congenital heart disease. The abnormality was a small chromosomal deletion in the FOXP1 gene.

Dr. Gargs team performed a search of DNA samples from patients with congenital heart disease in a repository at Nationwide Childrens one of the largest in the nation which revealed two additional patients with a similar heart defect who also had a rare mutation in the same gene.

The team analyzed the gene and found this mutation affected the genes ability to express a transcription factor called Nkx2.5, which has been implicated in congenital heart disease.

If you have three unrelated people with an abnormality in the same gene, and they also have an extremely rare type of congenital heart disease, theres a high likelihood that the gene is contributing to the condition, says Dr. Garg, who also is an associate professor of pediatrics at The Ohio State University College of Medicine. Understanding how either deletion or loss of FOXP1 affects normal heart development could help contribute to our understanding of congenital heart disease.

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Rare Genetic Mutation Linked To Congenital Heart Defects

Yes on Proposition 37 to Label Genetically Engineered Food?
In this video Myles discusses an advert promoting proposition 37. Yes on Proposition 37 to Label Genetically Engineered Food? http://mylespower.co.uk/2012/10...

By: Myles Power (powerm1985)

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Yes on Proposition 37 to Label Genetically Engineered Food? - Video

Genetic engineering may save the orange tree from becoming an endangered speciesThere's a virulent disease destroying orange trees around the world, and growers are facing a hard choice genetically engineer the orange to survive this plague, or possibly see the fruit, and their livelihood, disappear.

The effects of Citrus GreeningThe disease, called Citrus Greening (or Huanglongbing or yellow dragon disease), is a bacteria that was first reported in China in the early 20th century, and it's spread by tiny insects called Asian citrus psyllids. Orange trees infected by this bacteria drop their leaves, and their fruit becomes stunted. There's no pesticide that can control the psyllids that carry the disease (they quickly adapted to the ones we have), and there's no treatment for the disease itself. The only way to 'treat' an infection is to quarantine the area and burn the infected trees. However, even those efforts aren't helping much. This disease has spread throughout the southern United States, Mexico and Central America, Brazil, southeast Asia and western Africa, threatening crops from the largest orange producers in the world.

[ Related: GMO companies launch website to fight anti-biotech movement ]

One source of hope, it seems, is genetic engineering.

According to a New York Times article from this past weekend (which I highly recommend reading), growers in Florida have been looking into genetic engineering since 2005, but despite some promising results so far, they are understandably worried about public reaction.

Genetically-modified organisms 'GMOs' have a very bad reputation with the public these days. It's hard to even mention them without someone speaking of the evils of Monsanto, or hearing the word 'frankenfood', or about how genetically-modified foods cause any number of illnesses and diseases. Given that it's our health and our lives that we're talking about, being concerned about what we're being given to eat is understandable.

However, historically, we've been genetically modifying our crops and livestock for centuries, if not millennia. It's been done through cross-breeding and cross-pollination, with mixed results. The point is, though, that it's very unlikely that anything we eat these days has gone without some kind of human manipulation at some point. New techniques of directly changing the DNA of the plant or animal cause more concerns, though, with talk of splicing in animal DNA into plants.

Take it from a self-proclaimed hippy who thinks Monsanto is evil, though, that's not what scientists are doing. They create artificial genes based on what they see in animal genes, rather than splicing animal genes directly into plant DNA. That may not help someone's perception of what's going on, since it still impacts how 'natural' a food is, but in that case, I'll simply point back to the start of the previous paragraph. Also, another point this 'hippy' makes is that the more 'natural' cross-breeding methods are no safer than directly altering DNA (in fact, the more direct method is the safer of the two).

The science speaks on behalf of GMOs as well. The American Association for the Advancement of Science wrote last October that "the World Health Organization, the American Medical Association, the U.S. National Academy of Sciences, the British Royal Society, and 'every other respected organization that has examined the evidence has come to the same conclusion: consuming foods containing ingredients derived from GM crops is no riskier than consuming the same foods containing ingredients from crop plants modified by conventional plant improvement techniques.'"

[ More Geekquinox: U.S. supercomputer now doubles power for hurricane forecasts ]

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Genetic engineering may the only thing that can save the world’s oranges

Unstable genetic "capacitor" can "discharge" to accelerate the pace of evolutionary change.

Scientists historically have argued that evolution proceeds through gradual development of traits. But how can incremental changes apply to the binary switch between two sexes, male or female? Researchers at Case Western Reserve University's School of Medicine have found that a genetic process among the many species of rodents could have significant implications regarding our assumptions about sex determination and the pace of evolution.

"What we addressed is a long-standing puzzle in natural history: why different types of rodents can exhibit profound differences in how male sex is determined in the embryo," said Michael Weiss, MD, PhD, chairman of the Department of Biochemistry, the Cowan-Blum Professor of Cancer Research and a professor of biochemistry and medicine. "Some rodent populations have both XY males and XY females, and in other populations the Y chromosome has disappeared entirely."

In a study published in Proceedings of the National Academy of Sciences, Weiss and his research team analyzed the Sry gene, which is part of the Y chromosome. This mammalian gene, which steers differentiation in the embryonic gonad toward the development of testes, begins the process leading to the birth of males. For most mammals, including primates, Sry is a conserved feature of the Y chromosome, ultimately giving rise to male anatomy; females generally have two X chromosomes and no Y.

But within anomalous families of rodents, common in South America, activation of the Sry gene may have uncertain consequences. Some of these groups have both XY males and XY females as normal components of the population. Other related species have even lost their Y chromosomes altogether. Without the emergence of compensating ways of specifying sex, the species could not produce malesand would become extinct. For such rodents, therefore, evolution meant inventing entirely different methods of sex determination. These mammals have in essence evolved other ways to play nature's mating game.

The CWRU team attributed the rapid evolvability of sex determination in rodents to a novel protein domain added to the SRY protein. Scientists knew that this domain existed, but Weiss and his team wanted to understand more about its function in gene regulation and its role in evolution. The team determined that the new protein domain acts as a "genetic capacitor," providing a protective buffer to the Sry gene. This buffer allowed male development even when a mutation occurs elsewhere in the gene that might otherwise cause sex reversalbut the buffer is unstable over generations. Slippage of DNA during the production of sperm can lead to sudden changes in the length of the buffer and the degree of protection. By analogy to a capacitor in an electric circuit, the team suggested that this domain can "discharge" to accelerate the pace of evolutionary change. The idea of a genetic capacitor was pioneered by MIT Professor Susan Lindquist in studies of heat-shock proteins in fruit flies in (Nature, "Hsp90 as a capacitor for morphological evolution") and the present paper extended this idea to the pace of mammalian evolution.

How did the Sry buffer arise? "We discovered that a genetic accident 20 million years ago in an ancestral rodent holds the key to solving this puzzle. A simple DNA repeat sequence (called a 'micro-satellite') invaded the Y chromosome and was incorporated into the Sry gene. This invasion accelerated the evolvability of Sry and probably the Y chromosome in general, enabling this subgroup of rodents to explore new molecular mechanisms of sex determination," Weiss said.

Weiss and his team will continue this research, but believe these initial results may have additional implications for our understanding of human evolution and genetics. Because rodents have higher mutation rates and shorter life spans, they also evolve more rapidly and so provide a natural laboratory for studies of mammalian evolution.

Research last year at MIT has shown that in humans and other primates the Y chromosome has been stable for at least the past 25 million years (Nature, Strict evolutionary conservation followed rapid gene loss on human and rhesus Y chromosomes), which Weiss suggests may reflect the absence of micro-satellite-related slippage in the Sry gene. Yet the transcriptional strengths of the murine and human Sry factors are similar. The research suggests that human SRY and its specification of male development has evolved to be just above a genetic threshold of activity, which may in turn enable human communities to benefit from a diversity of male characteristics and behaviors.

"A key lesson of this 20 million-year history is that maleness is a 'close call' as the Sry protein functions near the edge of ambiguity," Weiss explained. "We think that the 'genetic decision' in an embryo to create a testis (instead of an ovary) is tenuous in all social mammals, including us. The critical next question is why?"

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Diverse sex-determining mechanisms revealed

Dr. Bogdanka Militescu, Director General Personal Genetics invitata la matinalul Digi 24
Testele genetice, preventia si tratamentele personalizate.

By: Personal Genetics

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Dr. Bogdanka Militescu, Director General Personal Genetics invitata la matinalul Digi 24 - Video

TIGEM (Telethon Institute of Genetics and Medicine), Napoli
L #39;Istituto Telethon di Genetica e Medicina (TIGEM) è stato creato nel 1994 dalla Fondazione Telethon per promuovere l #39;avanzamento della ricerca finalizzata a...

By: abcdsciencenews

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TIGEM (Telethon Institute of Genetics and Medicine), Napoli - Video

SALT LAKE CITY, July 30, 2013 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (MYGN) today announced it will hold its fourth-quarter 2013 sales and earnings conference call with institutional investors and analysts at 4:30 p.m. ET on Tuesday, Aug. 13. During the call, Peter D. Meldrum, president and chief executive officer; James Evans, chief financial officer; and Mark Capone, president, Myriad Genetics will provide an overview of Myriad's financial performance for the quarter and full year.

To listen to the call, interested parties in the United States may dial 800-404-5245 or +1- 303-223-2688 for international callers. All callers will be asked to reference reservation number 21668391. The conference call also will be available through a live webcast at http://www.myriad.com. A replay of the call will be available two hours after the end of the call for seven days and may be accessed by dialing 800-633-8284 within the United States or +1-402-977-9140 for international callers, and entering reservation number 21668391.

About Myriad Genetics

Myriad Genetics is a leading molecular diagnostic company dedicated to making a difference in patients' lives through the discovery and commercialization of transformative tests to assess a person's risk of developing disease, guide treatment decisions and assess risk of disease progression and recurrence. Myriad's portfolio of molecular diagnostic tests are based on an understanding of the role genes play in human disease and were developed with a commitment to improving an individual's decision making process for monitoring and treating disease. Myriad is focused on strategic directives to introduce new products, including companion diagnostics, as well as expanding internationally. For more information on how Myriad is making a difference, please visit the Company's website: http://www.myriad.com.

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Myriad Genetics Announces Date of Full-Year and Fourth-Quarter Fiscal 2013 Financial Results Conference Call

Let #39;s Play Metal Gear Solid - Part 11
During a training mission on Shadow Moses Island in February 2005, at a nuclear weapons facility on a remote island off the coast of Alaska, FOXHOUND and the...

By: Pro Starr

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Let's Play Metal Gear Solid - Part 11 - Video

Public release date: 30-Jul-2013 [ | E-mail | Share ]

Contact: Lauren Woods lauren.woods@mountsinai.org 212-241-2836 The Mount Sinai Hospital / Mount Sinai School of Medicine

The deadly condition known as pulmonary arterial hypertension (PAH), which afflicts up to 150,000 Americans each year, may be reversible by using an inhalable gene therapy, report an international team of researchers led by investigators at the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai.

In their new study, reported in the July 30 issue of the journal Circulation, scientists demonstrated that gene therapy administered through a nebulizer-like inhalation device can completely reverse PAH in rat models of the disease. In the lab, researchers also showed in pulmonary artery PAH patient tissue samples reduced expression of the SERCA2a, an enzyme critical for proper pumping of calcium in calcium compartments within the cells. SERCA2a gene therapy could be sought as a promising therapeutic intervention in PAH.

"The gene therapy could be delivered very easily to patients through simple inhalation -- just like the way nebulizers work to treat asthma," says study co-senior investigator Roger J. Hajjar, MD, Director of the Cardiovascular Research Center and the Arthur & Janet C. Ross Professor of Medicine and Professor of Gene & Cell at Icahn School of Medicine at Mount Sinai. "We are excited about testing this therapy in PAH patients who are in critical need of intervention."

This same SERCA2a dysfunction also occurs in heart failure. This new study utilizes the same gene therapy currently being tested in patients to reverse congestive heart failure in a large phase III clinical trial in the United States and Europe.

"What we have shown is that gene therapy restores function of this crucial enzyme in diseased lungs," says Dr. Hajjar. "We are delighted with these new findings because it suggests that a gene therapy that is already showing great benefit in congestive heart failure patients may be able to help PAH patients who currently have no good treatment options and are in critical need of a life sustaining therapy."

When SERCA2a is down-regulated, calcium stays longer in the cells than it should, and it induces pathways that lead to overgrowth of new and enlarged cells. According to researchers, the delivery of the SERCA2a gene produces SERCA2a enzymes, which helps both heart and lung cells restore their proper use of calcium.

"We are now on a path toward PAH patient clinical trials in the near future," says Dr. Hajjar, who developed the gene therapy approach. Studies in large animal models are now underway. SERCA2a gene therapy has already been approved by the National Institutes of Health for human study.

A Simple Inhalation Corrects Deadly Dysfunction

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Inhalable gene therapy may help pulmonary arterial hypertension patients

AMSTERDAM, THE NETHERLANDS--(Marketwired - Jul 30, 2013) - uniQure B.V., a leader in human gene therapy, today announced it has commenced build-out of a state-of-the-art 55,000 square foot US manufacturing plant in Lexington, Massachusetts for the production of commercial grade Adeno Associated Virus (AAV)-based gene therapy products to complement its current EMA-approved site in Amsterdam, the Netherlands. The Company expects the facility to be operational by early 2015.

The US site will significantly expand the Company's current production capacity. Once completed, it is expected to allow uniQure to capitalize on its modular manufacturing platform and leverage its process leadership in commercial grade AAV manufacturing. With two validated manufacturing sites on stream, the Company will be able to ensure it can supply commercial grade material for its pipeline of products as they advance into pivotal studies and will be positioned as the partner of choice for the manufacturing, development and commercialization of AAV-based gene therapies.

In connection with uniQure's US expansion, the Company has appointed Lance Weed as VP US Operations. Mr. Weed was previously Executive Director, Site Operations at Amgen, and prior to that VP of Operations at BioVex Inc. Mr. Weed has extensive experience with the manufacturing of viral-based pharmaceuticals and oversaw the construction of Amgen's (BioVex's) launch grade viral vector manufacturing facility in Woburn, MA from scratch. He will join the Company on September 1.

In addition, uniQure raised USD 10 million in venture debt from Hercules Technology Growth Capital, Inc. (NYSE: HTGC) ("Hercules") to finance the build-out of the US plant, and for general working capital purposes.

"The construction of this facility represents our commitment to establishing a global, fully integrated gene therapy company as we continue to build on our leadership position in the field of AAV-based gene therapy products," said Jrn Aldag, CEO of uniQure.

About uniQure uniQure is delivering on the promise of gene therapy, single treatments with potentially curative results. We have developed a modular platform to rapidly bring new disease modifying therapies to patients with severe disorders. Our approach is validated by multiple partnerships and the regulatory approval of our lead product Glybera. http://www.uniqure.com.

About Hercules Technology Growth Capital, Inc.Hercules Technology Growth Capital, Inc. (NYSE: HTGC) ("Hercules") is the leading specialty finance company focused on providing senior secured loans to venture capital-backed companies in technology-related markets, including technology, biotechnology, life science and energy technology (aka cleantech) industries at all stages of development. Since inception (December 2003), Hercules has committed more than $3.8 billion to over 240 companies and is the lender of choice for entrepreneurs and venture capital firms seeking growth capital financing.

DisclaimerThis press release contains forward-looking statements based on uniQure's current expectations. These forward-looking statements include statements regarding the completion of the build-out of our new manufacturing facility, receipt of regulatory approval of our manufacturing facility, and the development of additional gene therapies. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties regarding further regulatory requirements, the success of further clinical trials, and competitive pressures. uniQure assumes no responsibility to update such forward-looking statements.

Press release (PDF): http://hugin.info/157414/R/1719581/572296.pdf

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uniQure to Build 55,000 Square Foot State-of-the-Art Gene Therapy Production Plant in US to Leverage AAV-Manufacturing ...