THE IONIS STORY

Harnessing Groundbreaking Science and Technology with Relentless Passion to Discover and Deliver New Medicines to Patients

“We are pushing beyond what was thought possible to develop and deliver new RNA- and DNA-targeted therapies that will have a direct and meaningful impact on people living with serious diseases.”

— Brett P. Monia, Ph.D.,

Chief Executive 
Officer

Brett P. Monia, Ph.D. Black & White

WHO WE ARE

Over the last three decades, we have advanced science and discovered medical breakthroughs that have redefined life for people with serious diseases.

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Genetic Therapy Icon
GROUNDBREAKING SCIENCE AND INNOVATION

As a pioneer in RNA therapeutics, we have achieved firsts that were once thought impossible, including turning a rare, progressive and deadly childhood genetic disease into a chronic condition and developing the first medicine for a genetic cause of amyotrophic lateral sclerosis (ALS). We discovered and developed five currently marketed medicines for serious diseases, including breakthrough medicines for neurologic and cardiovascular diseases. With our groundbreaking science and technology, we have enhanced the profiles of our RNA-targeted medicines and unlocked new opportunities in emerging areas of genetic therapy. Historically, we worked with other biopharmaceutical companies as partners to deliver these medicines to patients who need them.

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OUR PIPELINE

In 2020, Ionis leadership made the bold decision to shift the company to directly serving patients by launching our products independently as a fully integrated biopharmaceutical company. In the course of just a few years, we’ve built a wholly owned, rich, late stage pipeline of potentially life-changing RNA-targeted medicines for people in critical need of significant advances, particularly in our focus areas of neurology and cardiology. We also continue to engage with partners on promising programs outside of our core areas of focus to advance investigational medicines for patients in other therapeutic areas with high need.

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PATIENTS & ADVOCACY

We are at an exciting time in Ionis’ trajectory as we prepare to launch our first wholly owned medicines while we pursue innovative science and advance breakthrough technology. As we look to the future, our aim is to discover, develop and deliver a steady cadence of important breakthrough medicines for patients and provide them with the resources and support they need to live fuller lives.

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OUR CULTURE

Our bold breakthroughs in medicine that are changing the course of human health are due to our stellar team. We work with limitless energy, ambition and passion, continually asking questions to learn and discover more and push the boundaries of science. To build on our rich history and achieve even more successes, we’re always looking for people who are similarly driven, inquisitive and want to pursue a fulfilling career.

TIMELINE

1978

  • The Field of Antisense Oligonucleotides is born – Antisense RNA is first used to inhibit protein production

1989

  • First Generation Chemistry chosen – Antisense oligonucleotides with phosphorothioate (PS) substitutions are more stable and able to distribute broadly throughout the body, making selected RNase H1 based antisense as a key mechanism
  • Ionis founded by Stanley T. Crooke, M.D., Ph.D.
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Stanley T. Crooke, M.D., Ph.D.

1991

  • Ionis IPO
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Ionis IPO

1992

  • Classification of potential antisense mechanisms

1993

  • First proven in vivo antisense activities
  • Chimeric (gapmer) antisense medicines with proven pharmacological advantages demonstrated

1995

  • 2′-0-methoxyethyl (2’MOE) discovered, making antisense medicines more RNA-like, with greater affinity for targets, greater potency
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2'-0-Methoxyethyl

1998

  • First Antisense Drug Reaches the Market – Ionis-discovered Vitravene (fomivirsen) is approved as a treatment for CMV-induced retinitis in immunocompromised patients with AIDS

1999

  • RNase H1 characterized enabling design of optimized antisense therapies

2000

  • Ionis initiates cardiovascular program

2003

  • Human genome project completed, revolutionizing the diagnosis and treatment of human diseases
  • Ionis initiates neurological disease program

2010

  • Selection of constrained ethyl (cET) as generation 2.5 chemistry, providing enhanced potency and broad distribution to multiple tissues

2013

  • Approval of KYNAMRO
  • Demonstrated increased uptake of therapies in the liver by linking a GalNAc molecule to the antisense oligonucleotide
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Kynamro (mipomersen sodium) Logo

2014

  • Akcea, Ionis’ first commercial affiliate, founded
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Akcea Therapeutics Logo

2015

  • Further identification of mechanisms of cell uptake and intracellular distribution

2016

  • Approval of SPINRAZA
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Spinraza (nusinersen) Logo

2017

  • Akcea IPO

2018

  • Approval for TEGSEDI
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Tegsedi (inotersen) Logo

2019

  • EU approval for WAYLIVRA
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Waylivra (volaneorsen) Logo

2020

  • Brett Monia, Ph.D., appointed CEO and shifts business strategy prioritizing Ionis’ wholly owned pipeline

2023

  • Approval of QALSODY
  • Approval for WAINUA
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Qalsody (tofersen) Logo
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Wainua (eplontersen) Logo

About Akcea

Akcea Therapeutics, Inc. is a wholly owned subsidiary of Ionis Pharmaceuticals, Inc., the leader in RNA therapeutics. Akcea commercializes TEGSEDI® (inotersen), which is available in the E.U. and Brazil, and WAYLIVRA® (volanesorsen), which is available in the E.U.

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Akcea Therapeutics Logo

Empowering our People and Communities

We are dedicated to fostering an inclusive culture that drives excellence, embraces diversity and supports our communities. Learn more about our commitment to diversity, equity and inclusion (DEI).