Products Onasemnogene bebeparvovec was approved in the United States in 2019 as a suspension for intravenous infusion (Zolgensma). Structure and properties It is a gene therapy using the -gene, in which adeno-associated serotype 9 (AAV9) viruses are used as vectors. The gene is contained in the form of double-stranded DNA in the capsid of the … Onasemnogen-Abeparvovec
Products Voretigenneparvovec was approved in the United States in 2017, in the EU in 2018, and in many countries in 2020 as a concentrate and solvent for the preparation of a solution for injection (Luxturna). Structure and properties Voretigenneparvovec is the capsid of an adeno-associated viral vector serotype 2 (AAV2). It contains the cDNA of … Voretigenneparvovec
Products Alipogentiparvovec has been approved in the EU as a solution for injection since 2012 (Glybera). It was the first gene therapy to be released in Europe. The drug’s high price is controversial. Structure and properties The drug contains a highly active variant of the lipoprotein lipase gene (S447X) in a viral vector. This consists … Alipogentiparvovec
