Ionis Pharmaceuticals

Ionis Pharmaceuticals is a pioneer in RNA therapeutics. They have discovered and developed five currently marketed medicines for serious diseases, including breakthrough medicines for neurologic diseases. With their groundbreaking science and technology, they have enhanced the profiles of their RNA-targeted medicines and unlocked new opportunities in emerging areas of genetic therapy. Historically, they have worked with other biotechnology companies as partners to deliver these medicines to patients who need them.

In March of 2014, CMTA entered an alliance with Ionis Pharmaceuticals (Ionis), in the development of Anti-Sense-Oligonucleotide (ASO) therapies. Specific ASOs were designed which proved effective in altering PMP22 RNA levels in two different rodent models of CMT1A. These studies showed that lowering PMP22 leads to functional improvements in CMT1A. While the Ionis therapeutic candidate was not suitable for use in patients, the pivotal work has attached other companies into the space who currently have candidates in preclinical development.