Archive for the ‘Gene Therapy Research’ Category

CSA SmartGroup - Part 2 of Success Masterclass - Five Success Secrets in CSA.
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CSA SmartGroup - Part 2 of Success Masterclass - Five Success Secrets in CSA. - Video

Pokemon Y 3DS - Update - New Mewtwo Form or Mewthree Confirmed
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Pokemon Y 3DS - Update - New Mewtwo Form or Mewthree Confirmed - Video

AMES, Iowa, April 8, 2013 /PRNewswire/ -- NewLink Genetics Corporation (NLNK), a biopharmaceutical company primarily focused on discovering, developing and commercializing immunotherapeutic products in oncology, announced today that the company presented preclinical data for NLG919, a potent IDO (indoleamine-(2,3)-dioxygenase) pathway inhibitor, at the American Association for Cancer Research (AACR) 2013 annual meeting. These data demonstrate that NLG919 potently inhibits the IDO pathway in vitro and in cell based assays, is orally bioavailable and has a favorable pharmacologic and toxicity profile. NLG919 is the second pipeline candidate from NewLink's IDO Pathway Inhibitor technology platform to be announced. This platform has also produced indoximod (NLG8189 or D1-MT) which is currently in Phase 2 clinical trials for the treatment of prostate cancer and metastatic breast cancer. Furthermore, NLG919 and indoximod show remarkable synergistic T-cell activation and antitumor activity.

"These data further support our view that IDO is a complex pathway with critical downstream immunomodulatory effects, and strategies that aim at multiple targets within this pathway may hold the most promise," commented Dr. Charles Link, Chairman and Chief Executive Officer of NewLink. "We are encouraged by NLG919's anti-tumor and pharmacological properties and will continue to evaluate it, along with other IDO pathway inhibitors in our preclinical and clinical pipeline, as an innovative approach to harnessing the immune system to treat cancer."

In a poster presentation entitled "NLG919, a novel indoleamine-2,3-dioxygenase (IDO)-pathway inhibitor drug candidate for cancer therapy," NewLink scientists presented preclinical data to demonstrate NLG919's on target anti-tumor effects as well as favorable oral bioavailability, pharmacokinetic and pharmacodynamic profiles.

The principal findings reported in the NLG919 study include:

About inhibition of the IDO pathway

IDO pathway inhibitors are another class of immune check point inhibitors akin to the recently developed antibodies targeting CTLA-4 and PD-1 which represent a potential breakthrough approach to cancer therapy. The IDO pathway regulates immune response by suppressing T-cell function and enabling local tumor immune escape. Recent studies have demonstrated that the IDO pathway is active in many cancers, both within tumor cells as a direct defense against T-cell attack, and also within antigen presenting cells in tumor draining lymph nodes whereby this pathway promotes peripheral tolerance to tumor associated antigens (TAAs). When hijacked by developing cancers in this manner, the IDO pathway may facilitate the survival, growth, invasion and metastasis of malignant cells expressing TAAs that might otherwise be recognized and attacked by the immune system. NewLink has a number of active programs directed at synthesizing IDO pathway inhibitors. These small-molecule, anti-tolerogenic product candidates are intended to counteract this key mechanism by which tumors evade immune-mediated destruction.

About NewLink Genetics Corporation

NewLink Genetics Corporation is a biopharmaceutical company focused on discovering, developing and commercializing novel immunotherapeutic products to improve treatment options for cancer patients. NewLink's portfolio includes biologic and small molecule immunotherapy product candidates intended to treat a wide range of oncology indications. NewLink's product candidates are designed to harness multiple components of the immune system to combat cancer without significant incremental toxicity, either as a monotherapy or in combination with other treatment regimens. NewLink's lead product candidate, algenpantucel-L (HyperAcute Pancreas) is being studied in a Phase 3 clinical trial in surgically resected pancreatic cancer patients (under a Special Protocol Assessment with the U.S. FDA) as well as in a separate study in locally advanced pancreatic cancer patients. NewLink has recently launched an adaptive design Phase 2B/3 clinical trial of tergenpumatucel-L (HyperAcute Lung) in patients with non-small cell lung cancer. NewLink is developing indoximod, a small molecule, orally bioavailable product candidate from NewLink's proprietary indoleamine-(2,3)-dioxygenase, or IDO, pathway inhibitor technology. NewLink is studying indoximod in various chemotherapy and immunotherapy combination studies independently and in collaboration with the National Cancer Institute. For more information please visit http://www.linkp.com. Patient information is available at http://www.pancreaticcancer-clinicaltrials.com.

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements of NewLink that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release are forward-looking statements, within the meaning of The Private Securities Litigation Reform Act of 1995. The words "anticipate", "believe", "estimate", "expect", "intend", "may", "plan", "target", "potential", "will", "could", "should", "seek", or the negative of these terms or other similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. These forward-looking statements include, among others, statements about: the prospects of algenpantucel-L, indoximod and our other HyperAcute product candidates and related clinical trials. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements that NewLink makes due to a number of important factors, including risks relating to: the initiation of clinical trials and the completion of enrollment; adverse general economic and industry conditions; and those risks discussed in "Risk Factors" and elsewhere in NewLink's Annual Report on Form 10-K for the period ended December 31, 2012, Form S-3 Registration Statement filed December 28, 2012 and in its other filings with the Securities and Exchange Commission. The forward-looking statements in this press release represent NewLink's views as of the date of this press release. NewLink anticipates that subsequent events and developments will cause its views to change. However, while it may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. You should, therefore, not rely on these forward-looking statements as representing NewLink's views as of any date subsequent to the date of this press release.

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NewLink Genetics Presents Preclinical Data on New IDO Pathway Inhibitor, NLG919, at AACR 2013 Annual Meeting

Step Two of PMD #39;s System
Step Two: The PMD Calming Neuro Neutralizing Toner balances pH levels and renews essential skin cells for a more youthful appearance. Our innovative formula ...

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Step Two of PMD's System - Video

Malaria and Sickle Cell Disease
Part of the Salsassin excerpt series.

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Malaria and Sickle Cell Disease - Video

Gene Therapy PSA

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Gene Therapy PSA - Video

BAVUBUKA FOUNDATION - LUGA FLOW DREAMS EPISODE # 5

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BAVUBUKA FOUNDATION - LUGA FLOW DREAMS EPISODE # 5 - Video

ACGT today announced that seed money provided for innovative T-Cell immunotherapy cell and gene therapy research has led to two successful Clinical Trials for treatment of Acute Lymphoblastic Leukemia resulting in remissions in both children and adults who had no other hope of recovery.

Stamford, Connecticut (PRWEB) April 08, 2013

According to Michel Sadelain, MD, PhD, ACGT Scientific Advisory Council member and Research Fellow, and senior author of the Acute Lymphoblastic Leukemia study published March 21st in the journal Science Translational Medicine, This is a very exciting finding for patients and a major achievement in the field of targeted immunotherapy. Sadelain explained that these successful treatments would not have been possible had not been for the startup funding from ACGT. It wasnt easy to get funding to support such innovative, unproved therapies. The grant from ACGT was our first major grant to launch our T-cell program. Dr. Sadelain is the Director of the Center for Cell Engineering and Gene Transfer Expression Laboratory at Memorial Sloan-Kettering Cancer Center.

Dr. Sadelain, and Dr. Carl H. June, Perelman School of Medicine at the University of Pennsylvania, were each awarded $1 million in ACGT Clinical Investigator grants in 2004 to fund research using immune-mediated gene therapy for the treatment of lymphoma or leukemia. They were the first two Clinical Translational grants that ACGT awarded. Just last week, ACGT received 49 letters of intent, with a majority focused on T-cell therapy research, in response to its Request for Applications for 2013 Clinical Translational grants that will be awarded this Spring.

Dr. June, ACGT Scientific Advisory Council member and leader of the team that successfully treated two young leukemia patients, Emma Whitehead and Maddie Major, and seven other adult leukemia patients at the Abramson Cancer Center of the University of Pennsylvania (Penn) also commented on the cutting-edge research ACGT supported: "Funding was not available from the National Institutes of Health (NIH) for gene therapy (in 2004). ACGT was the first to be there, to say that cell and gene therapies can be a new platform therapy, and giving literally lifesaving grants to scientists who cannot get them from the federal system."

ACGT is the only not-for-profit in the U.S. solely dedicated to cancer cell and gene therapy treatments for all types of cancer. 100% of contributions go directly to research. ACGT has funded 41 grants since its founding in 2001 totaling almost $24 million to fund both basic research and clinical translation. ACGT's Scientific Advisory Council, comprised of 16 renowned physicians and researchers, conducts a rigorous review process. Seventeen ACGT funded research projects have been approved for human clinical trials, 11 of which are underway. To donate, please visit http://www.acgtfoundation.org or call 203.358.8000.

# # #

Press Contact:

Deborah Burns, 203.257.3163

deborah(at)burnscommunications(dot)net

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ACGT Provides Seed Money for T-Cell Immunotherapy Cell and Gene Therapy Research; Successful Clinical Trials for ...

PARIS, April 8, 2013 /PRNewswire/ --

GenSight Biologics, a biopharmaceutical company focused on the development of ophthalmic therapeutics using gene therapy, announced today the closing of a 32 million Series A financing.

The financing was co-led by Novartis Venture Fund, Abingworth LLP, Versant Ventures and Index Ventures. GenSight will use the funds to develop a gene replacement therapy for Leber's hereditary optic neuropathy (LHON) and an optogenetic therapy for retinitis pigmentosa (RP). The lead product is expected to enter the clinic in 2013 in LHON patients.

Bernard Gilly, co-founder and CEO of GenSight is a successful bio-entrepreneur, was formerly CEO of Fovea Pharmaceuticals (which was acquired by sanofi-aventis in 2009), a partner at Sofinnova Partners and CEO of Transgene, a publicly traded gene therapy company. GenSight co-founder, Professor Jos-Alain Sahel, is Chairman of the Vision Institute in Paris. Botond Roska, a specialist in the structure and function of neural circuits at the FMI (Basel), has been pioneering the field of optogenetics. Jean Bennett, Professor of Ophthalmology, University of Pennsylvania, and also a co-founder of GenSight, has successfully conducted gene therapy trials to treat a rare retinal degeneration. The other co-founders of GenSight are Connie Cepko Professor of Genetics at Harvard (Boston), Ernst Bamberg Professor at The Max Planck Institute (Frankfurt), Luk Vandenberghe at the Schepens Eye Research Institute(Boston) and Serge Picaud at the Vision Institute (Paris).

Bernard Gilly commented: "Gene therapy is coming of age and ophthalmology is one of the most promising indications in particular because of the safety and efficacy demonstrated in certain trials. GenSight has a unique, proprietary approach to targeting the mitochondria in LHON developed by Dr Corral-Debrinsky and Jose Sahel at the Vision Institute, and exclusive access to key intellectual property from Novartis for using optogenetics to treat RP patients. Our outstanding links to leading ophthalmology physicians and scientists on both sides of the Atlantic, our strong link with the Foundation Fighting Blindness, our partnership with AFM/Genethon and our experienced management team are key to successfully moving our products through clinical developments to proof of efficacy."

"We are delighted to be working with GenSight and the other investors to assist the company in progressing its highly innovative technology and gene-based therapeutics towards the clinic. Gene therapy represents a new frontier in targeting ophthalmic diseases, and GenSight's expertise and capabilities are outstanding," said Florent Gros of Novartis Venture Fund, who will join the board of directors, along with Dr Genghis Lloyd-Harris (Abingworth) and Dr Guido Magni (Versant).

GenSight is based in Paris, France on the campus of the Vision Institute. Please visit our website http://www.gensight-biologics.com

Notes for Editors

About Novartis Venture Fund

The Novartis Venture Funds manages over $850 million in committed capital. NVF invests in companies which have the potential to change a core therapeutic field or explore new business areas that will be critical to patient care. Our primary interest is in the development of novel therapeutics and platforms as well as medical devices, diagnostics, and delivery systems. The Funds invest for financial objectives at all stages, but prefers to invest in the early-stages of company development. With ten investment professionals located in Basel, Switzerland and Cambridge, MA the team has extensive experience in pharmaceutical R&D and venture capital.

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GenSight Biologics Raises €32m in a Series A Financing for Ophthalmic Gene Therapy

Stem Cell Therapy for Pets - Teddy Before After
Amazing before and after footage of Teddy, an 11 year old dog who received stem cell therapy for his arthritis and received an unexpected benefit to his hear...

By: NewmanVets

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Stem Cell Therapy for Pets - Teddy Before

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

Contact: Aileen Sheehy press.office@sanger.ac.uk 44-012-234-96928 Wellcome Trust Sanger Institute

Researchers have uncovered the gene at the root of a human blood group that has remained a mystery for the past 60 years. They showed that a genetic deletion on this gene is responsible for the lack of this blood group in some people.

With the discovery of the gene behind the Vel blood group, medical scientists can now develop a more reliable DNA test to identify people who lack this group. This will reduce the risk of severe, and sometimes life threatening, destruction of the Vel-positive donor red blood cells in patients with antibodies against Vel.

The genetic basis of nearly all 34 blood group systems has been resolved over the past century, but identification of the underlying gene of the Vel blood group has withstood persistent attempts since it was first identified 60 years ago. It is estimated that one in 5000 people are Vel-negative, and routine blood transfusions for patients with antibodies against Vel can lead to kidney failure and even death.

"This is really exciting as it shows how the power of modern genomics technologies can directly benefit patient care," says Professor Willem Ouwehand , who heads one of the NHS Blood and Transplant research teams at both the University of Cambridge and the Wellcome Trust Sanger Institute. "This is also a milestone in blood group genetics and the end of long and astounding journey of discoveries in blood group genetics which started with Landsteiner from Austria and Fisher, Coombs and Morgan from England."

The discovery by the team would not have been possible without the colleagues from the blood transfusion services of Denmark, England and the Netherlands who undertook the Herculean effort of identifying the 65 individuals that lacked the Vel blood group by testing the red blood cells from nearly 350,000 donors with antibodies against Vel.

They then sequenced the coding fraction of the genomes of five donors who lack the Vel group to identify the underlying gene.

The team showed that the gene SMIM1 malfunctions in Vel-negative people. SMIM1 is found on chromosome 1 and specifies a small protein, five times smaller than the average human protein. This provides a direct explanation why a discovery by other routes has proven so challenging.

"It has been a remarkable feat to go from gene discovery to function in less than two months", continues Professor Ouwehand.

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Final chapter to 60-year-old blood group mystery

SOUTH PLAINFIELD, NJ--(Marketwired - Apr 7, 2013) - GENEWIZ, Inc., leading global genomics service provider, announced the launch of OncoGxOne Discovery cancer panels, proprietary gene panels designed specifically to assay genomic aberrations for all major cancer types.

Designed and developed by experts with the most recent research data available, GENEWIZ OncoGxOne Discovery cancer panels are the most comprehensive cancer-specific gene panels on the market, and have the ability to detect all types of genomic aberrations, including point mutations, Indels, gene fusions, and copy number variance (CNV).

"With the launch of OncoGxOne Discovery cancer panels, GENEWIZ has the opportunity to drive advancements in cancer research, offering solutions for biomarker discovery, therapeutic target discovery, and novel mutation identification," stated Dr. Guanghui Hu, Vice President of Translational Genomics.

"Using the latest next generation sequencing and bioinformatics technologies, OncoGxOne Discovery cancer panels can detect not only low-frequency variations and gene fusions in both exon and intron regions, but also CNV with great accuracy," Hu asserted. "The resulting data has the ability to stratify patients in clinical trials, as well as make a significant impact on personalized cancer treatment."

For more information about GENEWIZ OncoGxOne Discovery cancer panels, visit GENEWIZ at the American Association for Cancer Research (AACR) Annual Meeting this week in Washington, D.C.

About GENEWIZ, Inc. GENEWIZ, Inc. is a global contract research organization (CRO) specializing in genomic services, including DNA sequencing, gene synthesis, molecular biology, genomic, bioinformatics, and GxP/CLIA regulatory-compliant services. GENEWIZ leads the industry in outsourcing partnerships with pharmaceutical, biotechnology, clinical, academic, and government institutions.

Headquartered in South Plainfield, NJ, GENEWIZ, Inc. is a privately-held global enterprise with locations in Boston, MA; San Diego, CA; Washington, DC Metro; Research Triangle Park, NC; and Seattle, WA. International locations include Beijing, China; Suzhou, China; and London, United Kingdom.

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GENEWIZ, Inc. Launches OncoGxOne(TM) Discovery Cancer Panels

Dangers of "Diversity" | NationalProtectionism.com
We are told time and again that "diversity" is a great strength for our nations, but this video will examine the dangerous reality of non-white immigration a...

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Dangers of "Diversity" | NationalProtectionism.com - Video

Christian Jones bin flip
Christian Jones kickflips over a bin at flo.

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Christian Jones bin flip - Video

(GEAR event at UPenn for girls in high school; Credit: Justin Udo)

By Justin Udo

PHILADELPHIA (CBS) University of Pennsylvanias Society of Women Engineers hosted a workshop called Girls in Engineering and Related Sciences or GEARS. Its an effort to excite young women about a future in engineering

More than 80 girls from 9th-12 grade gathered at the University of Pennsylvania to participate in the day-long program.

Hilary Grosskopf is the Co-chair of University of Pennsylvanias Society of Women Engineers.

They get to experience four different workshops in different field of engineering.

The workshops include a bridge design competition, a glimpse into genetic engineering, a biopharmaceutical manufacturing workshop, and computer design program course.

The young women say this program is making them look at engineering in a whole new light.

Before I came in I really didnt know anything about computers. But I learned how to use part of a computer program which was really cool, I didnt know I could do that.

The students participating in the program received insight and advice into the engineering field by experts who work at Boeing, MWH Global and other companies on the engineering forefront.

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UPenn’s Society Of Women Engineer’s Hosts Education Event For Young Women

Better Living: Open Doors to Health- Patient Navigators
Specially trained patient navigators in the Open Doors to Health program help patients understand the importance of colonoscopies and walk them through the p...

By: PartnersHealthCare

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Better Living: Open Doors to Health- Patient Navigators - Video

Better Living: Domestic Violence Prevention Program
When a woman is a victim of domestic abuse, she can find assistance through the Brigham and Women #39;s Hospital Passageways and the Women #39;s After Care Clinic.

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Better Living: Domestic Violence Prevention Program - Video

Quit Everything and Train
Sign up Grow Stronger Newsletter: http://hulsestrength.com/go/youtube Elliott #39;s Other Channel: http://www.youtube.com/user/elliottsaidwhat Elliott #39;s Facebook...

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Quit Everything and Train - Video

Breast Cancer Answers Weekly Hangout On Air

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Breast Cancer Answers Weekly Hangout On Air - Video

PS3 BF3: Goodie Mob - Cell Therapy : Busta Rhymes - Break Ya Neck : Scarface - Homies Thugs
I do not own the copyright to the music, and no infringement intended. For entertainment purposes only* a little bit of rush on kharg island with rush_n_ata...

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PS3 BF3: Goodie Mob - Cell Therapy : Busta Rhymes - Break Ya Neck : Scarface - Homies

Michael Dickman, pictured with his fiancee Kerry Bright, suffers from the rare medical condition bladder exstrophy. Picture: Chris Scott Source: Sunday Herald Sun

VICTORIAN researchers have made a world-first medical discovery that could help people with a rare, but incredibly severe medical condition.

The research team, led by Monash University and Southern Health Professor of paediatric surgery Wei Cheng, found a mutation in the P63 gene, which increases the risk of bladder exstrophy.

Babies born with the condition have a split in their abdomen and pubic bone, which exposes their bladder, putting them at risk of infection, renal failure and bladder cancer.

Prof Cheng said babies undergo surgery to repair the bladder, close the hole and reattach the pelvic bones, but often suffer lifelong complications.

The research was sparked by the chance discovery of a deformed mouse, which had a strange bubble in the bladder area.

Prof Cheng proved it was bladder exstrophy and started exploring a genetic link.

Prof Cheng and Monash Institute of Medical Research's Dr Simon Wilkins and Dr Susan Zhang co-ordinated a worldwide DNA collection over six years, asking surgeons from Canada, Malaysia, USA, Australia, Spain, India, Bangladesh and China, to collect DNA from patients.

Examination of the DNA led to the discovery of the genetic link.

They found 11 mutations in total; three were associated with an increased risk of BE.

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Gene hope for rare condition

Genetic Engineering Overview

By: Danielle Garza

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Genetic Engineering Overview - Video

Errors Of The Human Body Official US Release Trailer #1 (2013) - Michael Eklund Thriller HD
Subscribe to TRAILERS: http://bit.ly/sxaw6h Subscribe to COMING SOON: http://bit.ly/H2vZUn Like us on FACEBOOK: http://goo.gl/dHs73 Errors Of The Human Body ...

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Errors Of The Human Body Official US Release Trailer #1 (2013) - Michael Eklund Thriller HD - Video

Supply-side policies - AS Economics
FACEBOOK PAGE: https://www.facebook.com/MultiplexinggamerTutorials Tutorial on Supply side policies for AQA AS level Economics. Requested by Rad.

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Supply-side policies - AS Economics - Video

The Legislatures Public Health Committee this week gave favorable approval to a measure that would require labeling of all food products containing genetically modified organisms (GMO) products where the genetic makeup has been altered through genetic engineering. On the surface, it sounds like a no-brainer. Who wouldnt want to know what were eating? The problem, however, is that the bill, if enacted, would only be effective within the borders of Connecticut. Several other states are considering similar legislation, but those measures would have no impact here, and our legislation would have no impact anywhere else. Stan Sorkin, president of the Connecticut Food Association, contends that if such labeling were necessary and he and others contend it isnt that such a mandate should be issued by the Federal Food and Drug Administration on a national scale, not piecemeal by individual states. The FDA, however, has denied requests to mandate GMO labeling, saying there is no evidence of the GMO products being unsafe. If there is no public threat, then clearly there is no need for the mandate. But where also is the harm in labeling it as such? Sorkin is correct in that Connecticuts large supermarkets, small groceries and other food-related businesses would be unfairly and unnecessarily harmed with higher costs of doing business if such mandates were applied only to them. Paul Pescatello, president and CEO of Connecticut United for Research Excellence (CURE), also contends that labeling GMO sends the wrong message to the very bio-science industry that Connecticut is hoping to attract to the state. Labeling, he contends, unfairly implies to the public that there is something to be concerned about when if fact there is overwhelming scientific evidence proving otherwise. Unfortunately, in todays society, mistrust of government and corporate America is the bigger threat than any genetically altered product. Such staunch opposition to labeling only feeds into that distrust. Capitalizing on the scientific evidence, and voluntary labeling, would seem the more appropriate course of action. Thats our opinion. Wed like to hear yours. Email us your thoughts at letters@norwichbulletin.com

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Our View: GMO labeling is better if voluntary