AAV vector. A new gene is inserted into a cell using the AAV protein shell. Once inside the nucleus, the new gene makes functional protein to treat a disease.<unk> , sold under the brand name Glybera, is a gene therapy treatment designed to reverse lipoprotein lipase deficiency (LPLD), a rare recessive disorder, due to mutations in LPL, which can cause severe pancreatitis. It was recommended for approval by the European Medicines Agency in July 2012 and approved by the European Commission in November of the same year. It was the first marketing authorisation for a gene therapy treatment in either Europe or the United States. The drug is administered via a series of injections into the leg muscles – as many as 60, all in one session. It is a one-time treatment intended to last at least ten years. Glybera gained infamy as the "million-dollar drug" and proved commercially unsuccessful for a number of reasons. Its cost to patients and payers, together with the rarity of LPLD, high maintenance costs to its manufacturer , and failure to achieve approval in the US, led to withdrawing the drug after two years on the EU market. As of 2018, only 31 people worldwide have ever been administered Glybera, and has no plans to sell the drug in the US or Canada.
Alipogene tiparvovec
Valoctocogene roxaparvovec
Neo-Viagra-Y.R.
Odalasvir
Enadenotucirev
Fosamprenavir
Onasemnogene abeparvovec
Elbasvir