Genetherapy

The Biotech Revolution (2007) – Video

November 3rd, 2012

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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The Biotech Revolution (2007) - Video

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

November 3rd, 2012

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

Recommendation and review posted by Bethany Smith

European Union Approves New Gene Therapy Drug

November 3rd, 2012

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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Europe backs first gene therapy

November 3rd, 2012

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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First gene therapy drug given European license

November 3rd, 2012

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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UPDATE 1-Europe approves high-price gene therapy

November 3rd, 2012

* 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

Recommendation and review posted by Bethany Smith

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

November 3rd, 2012

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

Recommendation and review posted by Bethany Smith

Europe approves high-price gene therapy

November 3rd, 2012

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.