First therapy in the western world to correct errors in a person’s genetic code approved
November 5, 2012
Lab creation of the adeno-associated virus (AAV) vector, which packages the gene that codes for a protein called lipoprotein lipase (LPL) (credit: uniQure)
European regulators have approved the first therapy in the western world that can correct errors in a person’s genetic code, according to Amsterdam-based uniQure (formerly Amsterdam Molecular Therapeutics),
Europe has approved Glybera for treatment of Lipoprotein Lipase Deficiency (LPLD), a very rare, inherited disease. Patients with LPLD are unable to metabolize the fat particles carried in their blood, which leads to inflammation of the pancreas (pancreatitis).
Glybera introduces a normal, healthy LPL gene into the body so that it can make functional LPL protein. The LPL gene is packaged in a delivery vector derived from adeno-associated virus (AAV), serotype 1, which has a natural propensity towards muscle cells.
Glybera will be available in the second half of 2013, according to uniQure, which is preparing to apply for regulatory approval in the U.S., Canada, and other markets.
Comments (26)
by Martin
I keep checking on this website hoping to find stories like this :-) This really warms my heart
by InvestorPro2012
What an amazing breakthrough. To think what the next five to ten years holds for the field of medicine is truly exciting. I’m confident that new treatments for cancer are just around the corner.
by GatorALLin
Makes me think we are just a very complicated computer code and repairs and then upgrades are just around the corner. If we are 1% there at year 7 of 14 total, then maybe the half way point and right on track…….(grin).
…so then how long before they can bring back a woolly mammoth or just bring Evils and Einstein (ok, just a clone…. I know he would not be the same exactly).
Hurry up…this future thing sounds cool.
by Jason Silva fanboy
Wow, this is awesome, sooner than I’d have thought. Bring it on.
But … did somebody in the non-western world manage this earlier?
by Editor
Yes. On October 16, 2003, Shenzhen SiBiono GenTech (Shenzhen, China), obtained a drug license from the State Food and Drug Administration of China (SFDA; Beijing, China) for its recombinant Ad-p53 gene therapy for head and neck squamous cell carcinoma (HNSCC)—a cancer that accounts for about 10% of the 2.5 million annual new cancer patients in China, sold under the brand name Gendicine. — Nature Biotechnology 22, 3 – 4 (2004)
doi:10.1038/nbt0104-3 http://www.nature.com/nbt/journal/v22/n1/full/nbt0104-3.html (open access)
by Vin
Wow, shades of ‘Post Mortal Syndrome’ the sci-fi novel already? Too surreal, man.
by Gabriel
This is beyond awesome – I cannot believe something like this has happened already….it feels so soon — Kurzweil DID say this decade would be when genetics reaches it’s peak, but that therapies are already being approved is incredible. We’re living in such an amazing time when so many things seem to be happening all at once
by Mr.X
@Gabriel: This is like the -the past was better- thing.People say this at all times.Everyone lives/lived in amazing times- maybe living is amazing (in wealthy societies)!?
by Aus
I have marfan syndrome, a genetic disorder that causes me to have weak connective tissue throughout my body. It also causes serious heart problems.
I am cautiously optimistic that one day I’ll have a strong, healthy, and youthful body. I predict decades but I think we’ll get there.
by Mr.X
Europe rules!
by Beatriz Valdes
There are some, I for one, whose metabolism creates mor Tryglicerides than is normal, and it is genetic. Would this new Glycera cure this condition? Pancreatitis could develop in these cases.
by Bob
I hope this really works. It will pave the way to curing all kinds of diseases.
by monty
When do people envision something like this being brought to cancer patients and other diseases?
by Dave
Things like this are already in testing for cancer. Using viruses to target certain cells is not a new idea… just not feasible yet on a large or practical scale.
by YourNameHere
Another great discovery. And BTW “Will be available by second half of 2013″
This is fast. Wow.
by judge
The promise of practical genetic modification for genetic based conditions has officially arrived- onward and upward!!!!
by melajara
@Editor, could you please check if this therapy is addressing the somatic cells only or the germ line too.
This is important as the former case is addressing the person only but without any effect to his/her potential descendents when the later will translate on an “inherited” therapy for the descendents.
Of course, it is much more difficult to have this “definitive” type of therapies accepted by defensive ethical committees.
by Editor
I sent a note. No mention of germline research on their website that I could find.
by Editor
“No germline” — uniQure CEO
by melajara
Thanks for the precision Amara
by Bri
We are in the first phase. Genetic research like this has already happened for other diseases, but they were only proof of principal tests. They will wait and see how this pans out, before proceeding with other cures and genetic therapeutic vectors.that’s why they choose these very rare afflictions. There is no other hope, so if things go wrong, there won’t be as much negative impact. Soon enough we will have nanobots inserting the corrective genes into the necessary cells.
by James
This is absolutely huge. These first treatments to fix our genetic code will rightly focus on people with life threatening diseases, but they will also scale up the technologies. Also, and I don’t mean to sound callous here, but these treatments for people with genetic diseases will allow medical science to iron out the bugs and make future treatments more effective. So when treatments to enhance healthy people come along, probably early next decade, we can be confident they’ll work, and work well. I look forward to seeing how effective this is.
by blair
YES
by Gorden Russell
I don’t see you as the least bit callous, James. I have suffered from a genetically inherited disease since infancy. As an asthmatic, I have had to struggle just to breathe. I can tell you that a severe asthma attack is just one step down from being water boarded. Yet I have often had to put up with ridicule from healthy people who think my gasping for breath is funny. Now that is really callous. I’ve always envied you healthy people your every clear breath, and lamented to see you put cigarette smoke into your healthy lungs. When I meet a smoker who is suffering from emphysema, I feel so bad for him. He is living through an attack that never ends.
by TheDonald
Yeah, that’s true. In general, the new and advanced therapies start out at the extreme ends of a bell curve and generally move to the centre (for the mainstream), becoming more cheaper and robust.
by Karl
You are right, this is really huge and very exciting. I am looking forward for the time I will be able to enhance myself through genetic engineering and nanotechnology. It is interesting to note that Ray Kurzweil predictions are proving to be accurate also in which regards to timing (sorry for eventual English errors: it is a foreign language for me).