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Company Profile

As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, proprietary antisense technology platform with the potential to treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs – such as children and adults with spinal muscular atrophy (SMA). We created SPINRAZA®(nusinersen)* and are proud to have brought new hope to the SMA community by developing the first and only approved treatment for this disease. 

Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 drugs with the potential to treat patients with cardiovascular disease, rare diseases, neurological diseases, infectious diseases and cancer. We created TEGSEDI™ (inotersen) the world's first RNA-targeted therapeutic approved for the treatment of polyneuropathy of hereditary transthyretin (TTR) amyloidosis (ATTR) in adult patients that our affiliate Akcea Therapeutics is commercializing. Together with Akcea, we are also bringing new medicines to patients with cardiometabolic lipid disorders.

*Spinraza is marketed by Biogen.

Upcoming Events

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Date Event Details
Summary Toggle 02/27/19 11:30 am EST
Summary Toggle 06/15/19 12:15 pm CEST

Bridging The Gap: Translating Genomic Discovery Into Human Therapeutics 


Bridging the Gap


European Society of Human Genetics Annual Conference 
Swedish Exhibition & Congress Center
Mässans Gata/Korsvägen
412 94 Göteborg, Sweden

 - Registration for the ESHG conference is required
 - The first 50 registrants for the Bridging The Gap corporate Satellite Symposium will receive a free gift
 - Registration link coming soon
 - Lunch will be provided


European Society of Human Genetics Annual Conference

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