What is Glybera and how is it administered?
Sep 02, 2016 · Glybera is the trade name of a medical treatment called alipogene tiparvovec, which was created by UniQure NV in the Netherlands for treating patients with a rare and unusual medical condition called lipoprotein lipase deficiency, which tends to be abbreviated as LPLD for ease of use. Although it is far from being the first gene therapy treatment, Glybera is notable in …
Is Glybera the first gene therapy treatment?
Sep 17, 2018 · 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.
How long does Glybera last?
Jan 03, 2013 · Glybera: The story of gene therapy. This article or excerpt is included in the GLP’s daily curated selection of ideologically diverse news, opinion and …
What is the history of Glybera?
Glybera should only be prescribed and given under the supervision of a doctor with expertise in treating lipoprotein lipase deficiency and in gene therapy. Glybera is given as a single treatment involving multiple injections into the muscles of the upper and
Why was Glybera discontinued?
How much does Glybera cost?
Is Glybera still on the market?
How is Glybera administered?
The drug normalises the metabolism of fat in the blood and thereby prevents episodes of pancreatitis. The drug is administered in the form of an injection into the leg muscle.Nov 7, 2012
Who manufactured glybera?
What disease does Luxturna treat?
What are the FDA approved gene therapies?
- ABECMA (idecabtagene vicleucel) ...
- ALLOCORD (HPC, Cord Blood) ...
- BREYANZI. ...
- CARVYKTI (ciltacabtagene autoleucel) ...
- CLEVECORD (HPC Cord Blood) ...
- Ducord, HPC Cord Blood. ...
- GINTUIT (Allogeneic Cultured Keratinocytes and Fibroblasts in Bovine Collagen) ...
- HEMACORD (HPC, cord blood)
What is gene therapy simple definition?
Is gendicine FDA approved?
What gene does glybera target?
When was glybera approved?
How does glybera work?
Glybera contains alipogene tiparvovec as the active substance. The drug works by breaking down the chylomicron particles present in the blood. It delivers a normal LPL gene into the body for correcting the LPL deficiency. The drug normalises the metabolism of fat in the blood and thereby prevents episodes of pancreatitis.
Is Glybera approved in Europe?
Glybera is currently the only gene therapy to be approved in Europe. Cerepro, another gene therapy developed by Ark Therapeutics, was rejected in the regulatory process in December 2009. uniQure is expecting to launch Glybera in the European market by mid-2013.
What is the cause of lipoprotein lipase deficiency?
Lipoprotein Lipase Deficiency is caused by chylomicron particles which transport fat through the blood. Triglycerides are a type of fat in the bloodstream which can cause recurrent pancreatitis. Image courtesy of Xvazquez. Glybera (alipogene tiparvovec) is a gene therapy indicated for the treatment Lipoprotein Lipase Deficiency (LPLD).
What is a tricyceride?
Triglycerides are a type of fat in the bloodstream which can cause recurrent pancreatitis. Image courtesy of Xvazquez. Glybera (alipogene tiparvovec) is a gene therapy indicated for the treatment Lipoprotein Lipase Deficiency (LPLD). It was developed by Amsterdam Molecular Therapeutics (AMT), which was acquired by uniQure in April 2012.
What is alipogene tiparvovec?
Glybera (alipogene tiparvovec) is a gene therapy indicated for the treatment Lipoprotein Lipase Deficiency (LPLD). It was developed by Amsterdam Molecular Therapeutics (AMT), which was acquired by uniQure in April 2012. In October 2012, uniQure received marketing authorisation approval for Glybera from the European Commission (EC) ...
When did UniQure get approval for Glybera?
In October 2012, uniQure received marketing authorisation approval for Glybera from the European Commission (EC) for treating patients with LPLD who have chronic and acute pancreatitis attacks. Glybera is the first gene therapy to be licensed in Europe.
What is a LPLD?
Lipoprotein Lipase Deficiency (LPLD) Lipoprotein Lipase Deficiency (LPLD) is rare genetic condition that elevates the levels of fat in the blood. The disease occurs due to alterations in LPL genes, which are responsible for breaking down of a protein-lipid complex called chylomicron.
Overview
The marketing authorisation for Glybera has expired following the marketing-authorisation holder’s decision not to apply for a renewal.
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When was Glybera approved?
After over two years of testing, Glybera was approved in the European Union in 2012. However, after spending millions of euros on Glybera's approval, AMT went bankrupt and its assets were acquired by uniQure.
Who developed Glybera?
Glybera was developed over a period of decades by researchers at the University of British Columbia (UBC). In 1986, Michael R. Hayden and John Kastelein began research at UBC, confirming the hypothesis that LPLD was caused by a gene mutation. Years later, in 2002, Hayden and Colin Ross successfully performed gene therapy on test mice to treat LPLD; their findings were featured on the September 2004 cover of Human Gene Therapy. Ross and Hayden next succeeded in treating cats in the same manner, with the help of Boyce Jones.
How long does a gene therapy last?
It is a one-time treatment intended to last at least ten years.
What is AAV1 vector?
The adeno-associated virus serotype 1 (AAV1) viral vector delivers an intact copy of the human lipoprotein lipase (LPL) gene to muscle cells. The LPL gene is not inserted into the cell's chromosomes but remains as free floating DNA in the nucleus. The injection is followed by immunosuppressive therapy to prevent immune reactions to the virus.
Who is the founder of AMT?
Meanwhile, Kastelein—who had, by 1998, become an international expert in lipid disorders—co-founded Amsterdam Molecular Therapeutics (AMT), which acquired rights to Hayden's research with the aim of releasing the drug in Europe.
What is glybera used for?
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).
Is Glybera a gene therapy?
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.
What is gene therapy?
image caption. Gene therapies alter a patient's DNA. 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 ...
Lipoprotein Lipase Deficiency
Glybera Mechanism of Action
- Glybera contains alipogene tiparvovec as the active substance. The drug works by breaking down the chylomicron particles present in the blood. It delivers a normal LPL gene into the body for correcting the LPL deficiency. The drug normalises the metabolism of fat in the blood and thereby prevents episodes of pancreatitis. The drug is administered i...
Clinical Trials of Europe’s First Approved Gene Therapy
- AMT conducted a Phase II/III clinical study on Glybera between February 2009 and April 2011. It was an open label, single group assignment which enrolled five patients with LPLD. The primary outcome of the study was finding the reduction of triglyceride (TG) concentrations in 12 weeks time. The secondary outcome measures included finding the reduction in chylomicrons, determi…
Marketing Glybera in Europe
- Glybera is currently the only gene therapy to be approved in Europe. Cerepro, another gene therapy developed by Ark Therapeutics, was rejected in the regulatory process in December 2009. uniQure is expecting to launch Glybera in the European market by mid-2013. The drug will be available in all 27 European member states. uniQure has also applied for the approval of Glybera in the US, C…