Ionis is the leading company in RNA-targeted drug discovery and development with a pipeline of first-in-class or best-in-class medicines with the potential to provide high value for patients with significant unmet medical needs. Over the last 30 years, Ionis has created an efficient and broadly applicable drug discovery platform using its proprietary antisense technology.

Ionis has created a large pipeline of first-in-class or best-in-class drugs, with over forty drugs in development. Including SPINRAZA® (nusinersen), an approved treatment for patients with spinal muscular atrophy, or SMA, a severe motor-neuron disease that is the leading genetic cause of infant mortality. Ionis is well-suited as a partner for RogCon given their leading position in antisense technology and proven success in rare, genetic disorders and neurological disease.

The research collaboration between The Florey and RogCon is critical to ensure RogCon is at the forefront of scientific and medical knowledge on SCN2A mutations. A large group of scientists at the Ion Channels and Human Diseases Laboratory at the Florey Institute have played a pivotal role in RogCon’s work to research and understand SCN2A mutations through a multidisciplinary approach including ion channel biophysics, mouse transgenesis, genetic analysis, computational modeling and in vitro and in vivo electrophysiology.