Gapmer

Binding of the gapmer to the target has a higher affinity due to the modified RNA flanking regions, as well as resistance to degradation by nucleases.

[9] The drug, developed by Ionis Pharmaceuticals and marketed by Genzyme Corporation, is administered via subcutaneous injection in the form of a mipomersen sodium solution.

[4] Kynamro targets the mRNA product of the APOB gene, which codes for the Apolipoprotein B-100 protein, a component of low-density lipoprotein (LDL).

Kynamro is reported to have an elimination half-life of approximately 1–2 months[9] Tegsedi, developed and marketed by Ionis Pharmaceuticals, was approved by the FDA in October 2018 for the treatment of hereditary transthyretin amyloidosis (hATTR).

[12] The functional consequences of gapmer off-target effects can vary widely, depending on the proteins affected and the extent of the down-regulation.

[9] Side effects of Inotersen include thrombocytopenia, glomerulonephritis, injection site reactions, nausea, headache, fatigue, and fever[10]