Ionis Pharmaceuticals

Ionis Pharmaceuticals is a biotechnology company focused on developing RNA-targeted drugs. Its technology platform in the antisense space allows for the control of protein production and reduction at a genetic level. Ionis´ antisense drugs are designed to interact precisely with RNA, allowing them to create treatments that disrupt the disease process and change its course. The company – in collaboration with its partners – has brought three drugs to the market already and currently has more than ten drug candidates in mid- to late-stage clinical development. The three approved drugs are Spinraza (partnered with Biogen) for spinal muscular atrophy, Tegsedi for hereditary transthyretin amyloidosis polyneuropathy and Waylivra for familial chylomicronemia syndrome. Antisense therapies change the process of producing protein before it even begins. To build a protein, a cell must make a copy of the DNA, which contains specific instructions for how to make that particular protein. This copy, called messenger RNA (mRNA), carries the instructions to the part of the cell where proteins are made. Antisense therapies are designed to seek out, bind to and destroy a mRNA in a highly specific manner, so that the amount of disease-causing protein is dramatically decreased. Antisense therapies can also treat diseases caused by too little protein by increasing the production of the protein and thereby restoring the protein to normal levels.



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