11-3 Exploring Mendelian Genetics (Part 1)
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11-3 Exploring Mendelian Genetics (Part 1) - Video

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11-3 Exploring Mendelian Genetics (Part 2)
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11-3 Exploring Mendelian Genetics (Part 2) - Video

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Mind Viruses Genetics
Date: 06 08 2009 To access the full archive and all the latest daily shows please visit and subscribe to this channel: http://www.youtube.com IF you would like to be notified the minute the daily show is released each day why not subscribe here http://www.youtube.com Also please subscribe on TWITTER heres the address or just click the twitter link on the right of the channel page twitter.com FACEBOOK heres the address or just click the facebook link on the right of the channel page http://www.facebook.comFrom:DiscoveryRadioShowsViews:0 0ratingsTime:02:34:33More inEducation

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Mind Viruses

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IRRI Pioneer Interview: Susan McCouch--A Juggling Act
Susan McCouch, an associate geneticist at the International Rice Research Institute (IRRI) in 1990-94 and currently professor of plant breeding and genetics at Cornell University, was truly a pioneer mdash;in more ways than one. She was an integral part of the team at Cornell that developed the first-ever molecular map of rice while, at the same time, being among the first female scientists to break the gender barrier in agricultural research. Back in 1985, when she applied to be a graduate student in Cornell #39;s plant breeding program, she was placed on probation because she was a female with a young child. Even so, during her July 2009 interview on the Cornell campus, she pointed out that, although she experienced difficulties and challenges as everyone does, she didn #39;t really believe that hers at both Cornell and IRRI were centered on gender issues. For the full transcript with links and photos, go to archive.irri.orgFrom:irrivideoViews:0 3ratingsTime:40:04More inEducation

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Race and Genetics - Wiki Article
Race (human classification) Category:Race (human classification) Race and Genetics - Wiki Article - wikiplays.org Original @ http All Information Derived from Wikipedia using Creative Commons License: en.wikipedia.org Author: Unknown Image URL: en.wikipedia.org Licensed under:Creative Commons ASA 3.0, This work is in the public domain in the United States. Author: Unknown Image URL: en.wikipedia.org Licensed under:Creative Commons ASA 3.0, This work is in the public domain in the United States. Author: Varoon Arya Image URL: en.wikipedia.org Licensed under:Creative Commons ASA 3.0, Licensed under the GNU Free Documentation License., GNU Free Documentation License, Creative Commons License Attribution-Share Alike 3.0 Unported This work is in the public domain in the United States. Author: Unknown Image URL: en.wikipedia.org Licensed under:Creative Commons ASA 3.0, This work is in the public domain in the United States. Author: Wobble Image URL: en.wikipedia.org Licensed under:This image is ineligible for copyright and therefore is in the public domain, because it consists entirely of information that is common property and contains no original authorship., This...From:WikiPlaysViews:0 0ratingsTime:47:36More inEducation

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Personality Genetics
Date: 08 02 2012 To access the full archive and all the latest daily shows please visit and subscribe to this channel: http://www.youtube.com IF you would like to be notified the minute the daily show is released each day why not subscribe here http://www.youtube.com Also please subscribe on TWITTER heres the address or just click the twitter link on the right of the channel page twitter.com FACEBOOK heres the address or just click the facebook link on the right of the channel page http://www.facebook.comFrom:DiscoveryRadioShowsViews:0 0ratingsTime:02:32:53More inEducation

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Personality

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Why I don #39;t want SRS and how that doesn #39;t make me less of a woman.
I #39;m really quite frustrated with this idea that trans women who do not want srs are in fact, men. It frustrates me because regardless of how loudly or how confindently you say that to me, you aren #39;t going to be able to convince anyone in my life that I #39;m a man. I have and will always be treated as a woman. Regardless of how you personally feel about my gender that is just something that won #39;t be changed. I also have issues with how people want to state that you aren #39;t a woman until you have SRS. Truth is, post op you still have male genetics and male blood flowing through your body. How much should we allow that to define our transition?From:Kitty KadaversViews:3 0ratingsTime:14:59More inPeople Blogs

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The Hidden Source Episode 25 -TROLL HIDDEN-
Myself ThatOneRebel playing some The Hidden Source! Nobody can see The Hidden only trained eyes can see him well! WATCH AS I MURDER EVERYONE AS THE HIDDEN MAHAAAHAHAHA The objective of The Hidden Source is that the humans aka IRIS must eliminate The Hidden to win vice versa for the Hidden but as the title says he is invisible and only is equipped with a knife capable of a one hit kill depending on server and only three grenades. The hidden has many abilities like taunting, super strength to ram objects into IRIS soldiers or be able to leap super high and hang on buildings. Lastly The Hidden can heal himself by eating bodies but only if they are stabbed to death not PIGSTICKED! (which is the one hit kill move) The IRIS are equipped with four weapons ranging from shotgun to rifle to SMG and a few options of equipment like senors to laser optics on your gun. Game is The Hidden Source which requires Half life 2 engine meaning you have to have Half Life 2 purchased already or another source game to run with STORYLINE In the early 1950s human genetics experimentation was taking its first, tentative steps. Amongst many other black projects, a team of British scientists working at an Infinitum Research experimental station stumbled across some remarkable phenomena involving DNA manipulation. This led to deeper research with dangerously unpredictable results, often leading to human patients losing their lives in irresponsible and immoral experiments. Time passed on, and by the mid ...From:ThatOneRebel55Views:0 0ratingsTime:14:02More inGaming

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The Hidden Source Episode 25 -TROLL HIDDEN- - Video

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Sam Sly Interviewing Luke Sly Pfizer Animal Genetics Employee
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Sam Sly Interviewing Luke Sly Pfizer Animal Genetics Employee - Video

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Kimberly Banks Discusses The Different Genetic Testing Methods
Genetics counselor Kimberly Banks talks about the different methods for genetic testing. Patients usually have the option of either a blood test or mouth swab. Click Here Get The 15 Breast Cancer Questions To Ask Your Doctor http://www.breastcanceranswers.com Breast Cancer Answers is a social media show where viewers submit a question and get the answer from an expert. Submit your question now at, http://www.breastcanceranswers.com This information should not be relied upon as a substitute for personal medical advice, diagnosis or treatment. Use the information provided on this site solely at your own risk. If you have any concerns about your health, please consult with a physician.From:drjayharnessViews:1 0ratingsTime:02:26More inScience Technology

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Kimberly Banks Discusses The Different Genetic Testing Methods - Video

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Statement of Psychiatric Survivors Made at the 2012 Carter Center Symposium
This is a statement composed by 6 psychiatric survivors at the 2012 Carter Center Symposium on Social Inclusion. Dan Fisher reads it at the end of his panel presentation on Integrated Care and Wellness. The conference was highly focused on the disease model of emotional distress. This statement is about our concerns with the disease model approach and how it might be increasing stigma. Many people in the audience doubted our quote saying Thomas Insel of NIMH has admited there is no genetic evidence for mental illness. The exact quote is right here, from JAMA. 2010;303(19): "Where is the missing genetic signal for mental illness? The discovery that large (1 megabase) structural or copy number variants, such as deletions and duplications, are 10-fold more common in autism and schizophrenia is an important clue.3,4 Copy number variants are individually rare,sometimes restricted to a single family or developing de novo in an individual. Although "private mutations" are rare (reminiscent of Tolstoy #39;s dictum that "each unhappy family is unhappy in its own way"), they are in aggregate remarkably common, spread across vast expanses of the genome, and ultimately could explain more genetic risk than common variants. Although many of the genes implicated are involved in brain development, copy number variants do not appear to be specific for illnesses in the current diagnostic scheme. Within families, the same copy number variant may be associated with schizophrenia in one person ...From:Corinna WestViews:0 1ratingsTime:05:09More inNonprofits Activism

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What Can I Learn About My Family History Of Breast Cancer From Genetic Testing?
Genetics counselor Kimberly Banks talks about how genetic testing for breast cancer helps patients understand their risk. Click Here Get The 15 Breast Cancer Questions To Ask Your Doctor http://www.breastcanceranswers.com Breast Cancer Answers is a social media show where viewers submit a question and get the answer from an expert. Submit your question now at, http://www.breastcanceranswers.com This information should not be relied upon as a substitute for personal medical advice, diagnosis or treatment. Use the information provided on this site solely at your own risk. If you have any concerns about your health, please consult with a physician.From:drjayharnessViews:1 0ratingsTime:01:32More inScience Technology

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The Dark Side of the Double Helix: Andrew Hessel at TEDx Marin 2012
A fascinating inside look at the coming world of Synthetic Biology. Andrew Hessel is a futurist and catalyst in biological technologies, helping industry, academics, and authorities better understand the changes underway in life science. Trained in microbiology and genetics, Andrew has continually worked at the forefront of life science in industry and academia. He is faculty at Singularity University, where he co-chaired the Biotechnology and Bioinformatics track and now helps startup biotechnology companies organize and launch. He is also a fellow at the University of Ottawa, Institute for Science, Society, and Policy, and the founder of the world #39;s first cooperative biotechnology company, the Pink Army Cooperative, which is working to create open source therapies for cancer. Andrew has given dozens of invited talks related to synthetic biology, for groups that include Intel Inc., the FBI, and the United Nations. In thespirit of ideas worth spreading, TEDx is a program of local, self-organized events that bring people together to share a TED-like experience. At a TEDx event, TEDTalks video and live speakers combine to spark deep discussion and connection in a small group. These local, self-organized events are branded TEDx, where x = independently organized TED event. The TED Conference provides general guidance for the TEDx program, but individual TEDx events are self-organized.* (*Subject to certain rules and regulations)From:TEDxTalksViews:65 11ratingsTime:19:05More inScience Technology

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Anthropologies Of Modernity: Foucault, Governmentality, And Life Politics
ll4.me Anthropologies Of Modernity: Foucault, Governmentality, And Life Politics This book brings together a range of anthropological writings that are inspired by the French philosopher Michel Foucault and examine Foucaults contribution to current theories of modernity. Treats modernity as an ethnographic object by focusing on its concrete manifestations. Tackles issues of broad interest: from colonialism and globalization to war, genetics, and AIDS. Draws on work from North and South America, Europe, Africa, and South and Southeast Asia. Contributors include James Ferguson, Akhil Gupta, Aihwa Ong, Paul Rabinow, and Rayna Rapp. Publisher: Wiley-Blackwell Illustration: N Language: ENG Title: Anthropologies of Modernity: Foucault, Governmentality, and Life Politics Pages: 00288 (Encrypted PDF) On Sale: 2008-04-15 SKU-13/ISBN: 9780631228264 Category: Social Science : Anthropology - Cultural This book brings together a range of anthropological writings that are inspired by the French philosopher Michel Foucault and examine Foucaults contribution to current theories of modernity.From:michaelsims9866Views:0 0ratingsTime:00:14More inPeople Blogs

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Miscarriage Part V
Most likely cause: random genetic abnormalities. This is unfortunate but it can be totally normal. I will not know the exact cause of my miscarriage, so I #39;m sticking with the idea that it was random and happened because the world of genetics is extremely fragile.From:22ANewBeginning47Views:0 0ratingsTime:07:55More inPeople Blogs

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Miscarriage Part V - Video

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SALT LAKE CITY, Nov. 1, 2012 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (MYGN) announced today that Peter D. Meldrum, President and CEO, is scheduled to present at the 2012 Credit Suisse Annual Healthcare Conference, at 9:30 a.m. Mountain Time on Thursday, November 15, 2012. The conference is being held at the Arizona Biltmore Resort & Spa in Phoenix, Arizona.

The presentation will be available to interested parties through a live webcast accessible on the investor relations section of Myriad's website at http://www.myriad.com.

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

Myriad, the Myriad logo, BRACAnalysis, Colaris, Colaris AP, Melaris, TheraGuide, Prezeon, OnDose, Panexia and Prolaris are trademarks or registered trademarks of Myriad Genetics, Inc. in the United States and foreign countries. MYGN-G

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Myriad Genetics to Present at the 2012 Credit Suisse Annual Healthcare Conference

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Seattle Genetics (Nasdaq: SGEN) is a biotech company focused on the development of monoclonal antibody-based treatments for cancer. The company's antibody-drug conjugate, or ADC, technology treats cancer by sending in an antibody and an attached drug, which bind with cancer cells and stops cell division from the inside. The company currently has one drug, Adcertis, available for sale.

The company has a small field of competitors in the ADC realm. Leading that pack is ImmunoGen (Nasdaq: IMGN) with a targeted antibody payload, or TAP, technology that works similarly to ADC technology, and has led to the company striking several large partnerships to license it. Pfizer (NYSE: PFE) is in the early stages of developing its own ADC technology, even as it works on other drugs created with Seattle Genetics' technology that could become competitors for Adcertis. Medarex, a subsidiary of Bristol-Myers Squibb (NYSE: BMY) , has ADC technology and is developing monoclonal antibody drugs that could compete with Adcertis.

Here are three ways Seattle Genetics is working to expand its revenues past the currently narrow definitions of its sole drug on the market.

Geographic expansionAdcetris is a treatment that's been approved by the Food and Drug Administration for resistant forms of Hodgkin lymphoma and systemic anaplastic large cell lymphoma. The drug was approved last summer, but revenues have failed to ramp up sharply due in part to the domestic limitation of the drug.

Geographic expansion could be just around the corner. A European Commission decision is expected any day now, following a July committee recommendation, which would make the treatment available in 27 countries. Canadian approval is following right behind. Health Canada accepted the New Drug Submissionfor Adcetris back in May, and a decision is expected in early 2013.

Adcetris is a partnership between Seattle Genetics and Millennium, an oncology subsidiary of Takeda. The partnership grants Seattle Genetics the drug rights in the U.S. and Canada, and requires Millennium to pay up to $230 million in milestone payments plus a double-digit percentage of net sales from its global markets.

Expanded useExpanding the approved indications for Adcetris would also boost its revenues. Seattle Genetics' pipeline is half full of trials for new indications. There are two phase 3 trials that deserve closer attention.

The AETHERA trialtests Adcetris versus a placebo in patients at risk for residual Hodgkin lymphoma after an autologous stem-cell transplant. The study has a primary endpoint of progression-free survival, and secondary endpoints include overall survivability and safety. Data is expected in late 2013 or early 2014.

The ALCANZA trialcompares Adcetris to methotrexate or bexarotene in patients with specific mature type of t-cell lymphoma. This trial is still enrolling, so its data will come in behind ATHERA, which finished enrollment in late September.

There are also Adcetris trials in earlier stages. The potential indications include front-line HL when used in conjunction with chemotherapy, front-line HL in elderly populations, and non-lymphoma malignancies.

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3 Things to Watch With Seattle Genetics

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The Biotech Revolution (2007)
Visions of the Future: Episode 1: The Intelligence Revolution (2007) http://www.youtube.com Theoretical physicist and futurist Michio Kaku shows how, in the 21st century, artificial intelligence is going to become as ubiquitous as electricity, how robots with human-level intelligence may finally become a reality, and how we #39;ll even be able to merge our minds with machine intelligence. As the challenges and choices are literally mind-bending, Dr Kaku asks how far we will ultimately go. Episode 2: The Biotech Revolution (2007) http://www.youtube.com Leading theoretical physicist and futurist Michio Kaku looks at the revolution in genetics and biotechnology, which promises unprecedented health and longevity but also raises the spectre of a Frankenstein future of genetically engineered people. Will we, as transhumanists expect, evolve into a new species? Dr Kaku warns that we may end up in a world divided by genetic apartheid. Episode 3: The Quantum Revolution (2007) http://www.youtube.com Theoretical physicist and futurist Michio Kaku shows how quantum physics is giving mankind the power to manipulate the fundamental building blocks of matter. Science fiction ideas like the space elevator, teleportation, invisibility cloaks, or nanosized molecular machines might soon become a reality. But will we use our unprecedented scientific mastery wisely? Visions of the Future: Genetics and Biotechnology promise a future of unprecedented health and longevity: DNA screening could prevent many diseases, gene ...From:Phobosuchus1Views:2 0ratingsTime:58:39More inScience Technology

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LONDON, Nov 2 (Reuters) - European officials have approved the Western world's first gene therapy drug from a small Dutch biotech company, in a milestone for the novel medical technology that fixes faulty genes.

The formal clearance from the European Commission paves the way for a launch next summer of the treatment for an ultra rare genetic disease that will cost around 1.2 million euros ($1.6 million) per patient, a new record for pricey modern medicines.

After more than 20 years of experiments and a series of disappointments, the EU approval of Glybera, which treats the genetic disorder lipoprotein lipase deficiency (LPLD), is a significant boost for the gene therapy field.

Joern Aldag, chief executive of Amsterdam-based uniQure, said more such treatments would follow and argued a high price was justified because gene therapy restored natural body function and did not just offer a short-term fix.

"This provides higher benefit to patients than the classical protein replacement strategy and this is why we think we should be fairly and adequately compensated," he said in a telephone interview on Friday.

Patients with LPLD, which affects no more than one or two people per million, are unable to handle fat particles in their blood and are at risk of acute and potentially fatal inflammation of the pancreas.

The approval follows a positive recommendation from the European Medicines Agency in July.

The privately owned firm is now working with governments on potential pricing strategies, which are likely to vary from country to country, ahead of the commercial roll-out from the second half of 2013.

Aldag said some countries preferred the idea of a one-off payment at the time of treatment but others were interested in an annuity system, which would probably involve charging around 250,000 euros a year for five years.

That kind of annual charge would put Glybera in a similar price range to expensive enzyme replacement therapies for other rare diseases, such as Cerezyme for Gaucher disease from Sanofi's Genzyme unit.

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Europe approves therapy to fix bad genes that will cost $1.6M per patient

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November 2, 2012

Lee Rannals for redOrbit.com Your Universe Online

A drug that has the ability to correct errors in a genetic code has been approved for the first time ever in Europe.

The European Commission has given Glybera authorization to sell the drug next summer for treating an ultra rare genetic disease.

The small Dutch biotech companys drug will cost around $1.6 million per patient, a new record price for medicine.

The drug is a gene therapy for a rare disease that leaves people unable to properly digest fats, which affects one in a million people. As fat builds up in the blood, it leads to abdominal pain and life-threatening inflammation of the pancreas. The only way to manage the condition is with a very low-fat diet.

UniQures drug uses a virus to infect muscle cells with a working copy of the gene that breaks down fats.

Treating patients by replacing a defective gene with a working copy first came to light in 1990, when the worlds first gene therapy clinical tests against a rare condition called severe combined immunodeficiency (SCID) took place.

People who suffer from SCID are unable to cope with infections and usually die in childhood. The researchers trying to use gene therapy against the condition faced a setback in 1999 when an Arizona teenager died in a gene therapy experiment. A few years later, two French boys with SCID developed leukemia after being treated.

The Chinese firm SiBono GeneTech won approval for a gene therapy drug for head and neck cancer in 2003, but no products have been approved in the U.S. or Europe.

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European Union Approves New Gene Therapy Drug

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2 November 2012 Last updated at 07:00 ET By James Gallagher Health and science reporter, BBC News

A treatment which corrects errors in a person's genetic code has been approved for commercial use in Europe for the first time.

The European Commission has given Glybera marketing authorisation, meaning it can be sold throughout the EU.

It is a gene therapy for a rare disease which leaves people unable to properly digest fats.

The manufacturers say it will be available next year.

Gene therapy has a simple premise. If there is a problem with part of a patient's genetic code then change the code.

However, the field has been plagued with problems. Patients have developed leukaemia and in one trial in the US a teenager died.

In Europe and the US, the therapies are used only in research labs.

Glybera is used to treat lipoprotein lipase deficiency. One in a million people have damaged copies of a gene which is essential for breaking down fats.

It means fat builds up in the blood leading to abdominal pain and life-threatening inflammation of the pancreas (pancreatitis).

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Europe backs first gene therapy

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Gene therapy drug given green light for rare disease Glybera to cost 1m ($1.6m) for each patient Drug is first to get approval in Western world EU go-ahead paves way for many more treatments

By Jenny Hope

PUBLISHED: 08:19 EST, 2 November 2012 | UPDATED: 21:14 EST, 2 November 2012

Revolution: Gene therapies can change a person's DNA

Regulators yesterday approved the first therapy in the western world that can correct errors in a persons genetic code.

Europe has approved Glybera to be used against a rare inherited disorder which disrupts fat production in the body.

The treatment uses a virus to counteract LPLD, lipoprotein lipase deficiency, which can led to acute inflammation of the pancreas.

The illness affects around one or two people per million and sufferers have damaged copies of a gene which is essential for breaking down fats. The virus infects muscle cells with a copy of a healthy gene and a one-off treatment is effective.

The treatment was backed by an advisory panel to the European Commission in July and full approval was granted yesterday. The medicine should be available next year.

Professor John Kastelein, of the University of Amsterdam, said the therapy would have a dramatic impact on patients.

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First gene therapy drug given European license

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* European Commission clears Glybera for ultra rare disease

* UniQure drug is first gene therapy to reach market in West

* Glybera likely to cost around $1.6 million per patient

* Final EU approval follows positive recommendation in July (Adds interview with company CEO, pricing details, background)

By Ben Hirschler

LONDON, Nov 2 (Reuters) - European officials have approved the Western world's first gene therapy drug from a small Dutch biotech company, in a milestone for the novel medical technology that fixes faulty genes.

The formal clearance from the European Commission paves the way for a launch next summer of the treatment for an ultra rare genetic disease that will cost around 1.2 million euros ($1.6 million) per patient, a new record for pricey modern medicines.

After more than 20 years of experiments and a series of disappointments, the EU approval of Glybera, which treats the genetic disorder lipoprotein lipase deficiency (LPLD), is a significant boost for the gene therapy field.

Joern Aldag, chief executive of Amsterdam-based uniQure, said more such treatments would follow and argued a high price was justified because gene therapy restored natural body function and did not just offer a short-term fix.

"This provides higher benefit to patients than the classical protein replacement strategy and this is why we think we should be fairly and adequately compensated," he said in a telephone interview on Friday.

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UPDATE 1-Europe approves high-price gene therapy

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AMSTERDAM, The Netherlands, November 2, 2012 /PRNewswire/ --

uniQure announced today it has received approval from the European Commission for the gene therapy Glybera (alipogene tiparvovec), a treatment for patients with lipoprotein lipase deficiency (LPLD, also called familial hyperchylomicronemia) suffering from recurring acute pancreatitis. Patients with LPLD, a very rare, inherited disease, are unable to metabolize the fat particles carried in their blood, which leads to inflammation of the pancreas (pancreatitis), an extremely serious, painful, and potentially lethal condition. The approval makes Glybera the first gene therapy approved by regulatory authorities in the Western world.

"Glybera's approval means LPLD patients, for the first time, have a medical treatment option for a very complex and severe disease," said Professor John Kastelein of the Department of Vascular Medicine at the Academic Medical Center of the University of Amsterdam, the Netherlands. "LPLD leads to acute and recurrent pancreatitis attacks, and in many patients causes early onset diabetes and cardiovascular complications. This therapy will have a dramatic impact on the lives of these patients. Currently their only recourse is to severely restrict the amount of fat they consume. By helping to normalize the metabolism of fat, Glybera prevents inflammation of the pancreas thereby averting the associated pain and suffering and, if administered early enough, the associated co-morbidities."

As part of the approval, patients will receive treatment with Glybera through dedicated centers of excellence and by specially trained doctors. uniQure will also build a patient registry to further improve the understanding of this devastating, under-researched disease and the effects of Glybera treatment. Marketing Authorisation covers all 27 European Union member states. uniQure is preparing to apply for regulatory approval in the US, Canada, and other markets.

"The final approval of Glybera from the EC marks a major step forward in making gene therapies available not only for LPLD but also for a large number of rare diseases with a very high unmet medical need," says Jrn Aldag, CEO of uniQure. "The EC's approval is an important validation of our innovative product platform and offers strong support for our other advanced development programs, which focus on acute intermittent porphyria, Sanfilippo B, hemophilia B and Parkinson's disease."

About Glybera

uniQure has developed Glybera as a therapy for patients with the genetic disorder lipoprotein lipase deficiency, an orphan disease for which no treatment existed. The disease is caused by mutations in the LPL gene, resulting in highly decreased or absent activity of LPL enzyme in patients. This enzyme is needed in order to break down large fat-carrying particles that circulate in the blood after each meal. When such particles, called chylomicrons, accumulate in the blood, they may obstruct small blood vessels. Excess chylomicrons result in recurrent and severe acute inflammation of the pancreas, called pancreatitis, the most debilitating complication of LPLD. Glybera has orphan drug designation in the EU and US. LPL Deficiency affects 1-2 persons per million.

Glybera has been tested in three interventional clinical studies conducted in the Netherlands and in Canada, in which a total of 27 LPLD patients participated. In all three clinical trials, Glybera was well tolerated, with no relevant safety issues observed. Data from these clinical trials indicate that a single dose administration of Glybera resulted in a long-term biological activity of the LPL protein. For further information on LPLD visit http://www.lpldeficiency.com.

Lipoprotein lipase is a key 'first step' enzyme in the metabolism of lipoproteins following fat intake with diet. In clinical studies a transient reduction in triglycerides for up to 12 weeks in individual patients could be observed. Furthermore, Glybera allows expression of the LPL protein in injected muscle which is reflected by the improvement of postprandial chylomicron (CM) metabolism observed in a small subset of patients. Glybera (alipogene tiparvovec) contains the human lipoprotein lipase (LPL) gene variant LPL S447X in a vector. The vector comprises a protein shell derived from adeno-associated virus serotype 1 (AAV1), the promoter, a posttranscriptional regulatory element and AAV2 derived inverted terminal repeats.

Glybera is indicated for adult patients diagnosed with familial lipoprotein lipase deficiency (LPLD) and suffering from severe or multiple pancreatitis attacks despite dietary fat restrictions. The diagnosis of LPLD has to be confirmed by genetic testing. The indication is restricted to patients with detectable levels of LPL protein.

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uniQure's Glybera® First Gene Therapy Approved by European Commission

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LONDON (Reuters) - European officials have approved the Western world's first gene therapy drug from a small Dutch biotech company, in a milestone for the novel medical technology that fixes faulty genes.

The formal clearance from the European Commission paves the way for a launch next summer of the treatment for an ultra rare genetic disease that will cost around 1.2 million euros ($1.6 million) per patient, a new record for pricey modern medicines.

After more than 20 years of experiments and a series of disappointments, the EU approval of Glybera, which treats the genetic disorder lipoprotein lipase deficiency (LPLD), is a significant boost for the gene therapy field.

Joern Aldag, chief executive of Amsterdam-based uniQure, said more such treatments would follow and argued a high price was justified because gene therapy restored natural body function and did not just offer a short-term fix.

"This provides higher benefit to patients than the classical protein replacement strategy and this is why we think we should be fairly and adequately compensated," he said in a telephone interview on Friday.

Patients with LPLD, which affects no more than one or two people per million, are unable to handle fat particles in their blood and are at risk of acute and potentially fatal inflammation of the pancreas.

The approval follows a positive recommendation from the European Medicines Agency in July.

The privately owned firm is now working with governments on potential pricing strategies, which are likely to vary from country to country, ahead of the commercial roll-out from the second half of 2013.

Aldag said some countries preferred the idea of a one-off payment at the time of treatment but others were interested in an annuity system, which would probably involve charging around 250,000 euros a year for five years.

That kind of annual charge would put Glybera in a similar price range to expensive enzyme replacement therapies for other rare diseases, such as Cerezyme for Gaucher disease from Sanofi's Genzyme unit.

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Europe approves high-price gene therapy

Recommendation and review posted by Bethany Smith