gene therapy drugs

Onasemnogen-Abeparvovec

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

Alipogentiparvovec

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