uniQure Terug naar discussie overzicht

een behandeling van 1,6 mln.

1. sp1946 29 september 2013 20:27

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   Jim Kozubek: The Birth of ‘transhumans’
   September 29, 2013 01:00 AM
   By Jim Kozubek
   Boston
   UniQure BV, the Dutch company that won approval to market the first gene therapy drug in Europe, is now pacing to bring the first gene therapy drug to market in the United States. The drug maker this fall will begin building a 55,000 square foot manufacturing plant in Lexington, Mass., after the town wooed it with a package of tax savings. The first gene therapy drugs will be upon us in the United States within three years.
   Transhumans? People with shiny, new synthetic genes that replace defective copies of those genes, or people who have fish DNA sewn up into their genomes? Yes, they’re already walking among us.
   Probably numbering in the hundreds, they are part-human, part-synthetics who are the products of gene therapy trials. Now the Food and Drug Administration is weighing approvals for the first market applications for genetic engineering in humans.
   I’d say it’s time to embrace gene therapy. I’m not alone. “The anti-determinists want to say that DNA is a little side-show, but every disease that’s with us is caused by DNA and can be fixed by DNA,” the biologist George Church told me.
   Indeed, gene therapy drugs are what investors might call “disruptive technology” since they radically upset insurance models, and indeed, how doctors think about treating a disease.
   When it hits the market in Europe for the first time this fall at an estimated $1.6 million, Glybera will be the most expensive drug in the world.
   “The idea is that Glybera is a one-time treatment,” Jörn Aldag, CEO of UniQure, told me. “If we look out five years, there should be tens approved, if not hundreds” of gene therapy drugs, which Aldag called the new “routine treatment modality.”
   The field suffered staggering setbacks in 1999 when 18-year-old Jesse Gelsinger died in a gene therapy trial. Gelsinger had an enzyme deficiency disease, and an adenovirus was used to deliver a replacement gene to his genome. But it touched off a massive immune system response called a “cytokine storm.” The FDA suspended gene therapy trials in the early 2000s and investment disappeared.
   But small improvements have made the technology safer. UniQure’s drug, Glybera, makes use of an adeno-associated virus which is less “immunogenic” than the one used to treat Gelsinger, and slips inside cells along with its cargo, a shiny new gene — in this case, a copy of a gene that builds lipoprotein lipase. It is a type of gene therapy that is “non-integrative” since the gene is not actually incorporated into the genome. Other gene therapy tools, or “expression cassettes,” do integrate new DNA into our genomes and have been engineered to be gentler, and less likely to trigger side-effects, like a cancer.
   And, George Church’s group is one of several labs working on the development of tiny molecular scissors that can actually make edits to our genomes with high precision. His mechanism, called CRISPR, may be used to treat conditions where specific mutations are known to cause a disease, such with Huntington’s Disease, where repeats of the motif CAG in the HTT gene are known to drive the disease.
   The biologist Richard Dawkins once said that groups and individuals mingle in loose federations, like “clouds in the sky or dust-storms in the desert,” while “genes, like diamonds, are forever.” If the publishing of the first draft of human genome in 2001 was like decoding an ancient, antediluvian script, a tool now exists to rewrite the Tablets of Stone.
   In our lifetimes we will be confronted with new ethical choices on whether to edit our genes. Will we try to do all sorts of fancy things, aligning ourselves with the Kurzweil Singularity movement, the sad, comical spectacle that it is? The Time magazine cover story “Can Google Solve Death” became part of that spectacle when it noted that, for CEO Larry Page, solving cancer “may not be a big enough task.”
   On the other hand, will we recoil from genetic engineering with the gut reaction that being “transhuman,” synthetically modifying our genes, somehow makes us “less than human”? Much is at stake. For many dangerously sick children, genetic engineering may be the most powerful, if not the only, tool. Not only can we use it. I think we must.
   Earlier this year, a researcher from Japan, Masashito Tachibana, wrote a landmark article in the journal Nature (“Towards germline gene therapy of inherited mitochondrial diseases”). It laid the groundwork for gene engineering in the “germline” cells that get passed to future generations.
   It would allow for the birth of the first transhumans. And future offspring would forever carry this additional code. Do we want that? These are open questions. We will confront them in our very generation.
   
   Jim Kozubek, of Cambridge, Mass., is a computational biologist and a science writer. He is writing a book on gene therapy and genetic engineering in humans.
   
   
2. [verwijderd] 29 september 2013 21:37

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   $1,6 mln per behandeling?
   
   Dat zijn "amerikaanse" bedragen.
   
   Gr
   
   Frank
3. flosz 30 september 2013 08:58

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   Linkje erbij: www.providencejournal.com/opinion/com...
   
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   Jörn Aldag, UniQure’s chief executive, said he would probably charge €250,000 a year for five years for the drug, a similar amount to Cerezyme, another “orphan drug” for a very rare disease. That would indicate a total bill of €1.25m per patient.
   www.iex.nl/Forum/Topic/1286507/2/Morg...
   
   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.
   www.iex.nl/Forum/Topic/1290716/uniQur...
   
   Most expensive medicine: Glybera breaks Guinness world record
   Amsterdam, The Netherlands -- Glybera, developed by private Dutch firm uniQure, the first gene therapy to win approval in the West, is expected to arrive soon with a price tag of more than a million dollars, setting the new world record for the Most expensive medicine, according to the World Record Academy: www.worldrecordacademy.com
   Glybera is the first gene therapy approval in an independently regulated healthcare market.