Ionis Pharmaceuticals, Inc.

Today, our CEO Brett P. Monia, PhD?had the incredible honor of ringing the Nasdaq?Opening Bell to celebrate our recent FDA approval for the treatment of FCS - our first independent medicine! For more than three decades, we’ve been at the forefront of RNA-targeted medicine, pioneering breakthroughs that were once thought impossible. And now we’re poised for even greater impact as a fully integrated commercial-stage biotechnology company! Today’s celebration wouldn’t have been possible without the dedication of our employees, the trust of our partners, and—most importantly—the resilience of the patients we serve. To the Ionis team, our investors and our supporters: thank you for believing in the power of what we do.

In the past week, we had the pleasure of partnering with two exceptional organizations to share our passion for science and biotechnology and help educate and prepare a new generation for careers in related fields. First, we joined industry peers to facilitate interactive activities for underserved?students at MANA de San Diego STEAM Pi day celebration. On Tuesday, we hosted the?Young Women In Bio?career panel at our Carlsbad campus—a platform for accomplished professionals to share their career journeys, insights, and advice, inspiring and guiding young women. It was a privilege to partner with Scientella and?Aroga Biosciences to share our passion and knowledge with such energetic and engaged future scientists.?

DYK: people living with transthyretin amyloidosis (#ATTR) can experience multiple symptoms that may seem unrelated? In March, we join the community in recognizing #AmyloidosisAwarenessMonth. ATTR amyloidosis symptoms may vary from person to person and progress over time, so education about this #raredisease may lead to quicker diagnosis, identification of a treatment plan and a better journey for patients. Learn more about ATTR amyloidosis: https://lnkd.in/etb8dfX4

HCPs: Unsure how to distinguish familial chylomicronemia syndrome (FCS) from other causes of elevated triglycerides? A clinical diagnostic scoring tool is now available that can help with diagnosis of this #raredisease on the basis of common clinical and laboratory tests. To learn more and access the North American FCS Scoring Calculator: https://lnkd.in/ecHN_XyD

We were honored to join top academics, biotech innovators, and up-and-coming organizations at RNA at the Bench and Bedside IV at the end of last year. This event continues to be a catalyst for collaboration, knowledge-sharing, and inspiration. At Ionis, we're proud to be part of this dynamic community and see real momentum year over year as these conversations and partnerships continue to shape future innovations in science and medicine. https://lnkd.in/e4NRU2Cn

We’re “Lighting the Night” red to mark #AmyloidosisAwarenessMonth and join in increasing awareness of this group of #rarediseases caused by the buildup of abnormal proteins (called amyloids) in tissues and organs throughout the body. Transthyretin amyloidosis (#ATTR) can be difficult to recognize – sometimes taking more than three years to accurately diagnose – because its symptoms are varied and may resemble those of other conditions. As a progressive disease, ATTR diagnosis delays can have lasting consequences. That’s why education and advocacy are critical to ensuring timely diagnosis and earlier treatment. Learn more about ATTR amyloidosis: https://ow.ly/PPl350VgviJ

Today we announced a new license agreement with Ono Pharmaceutical Co., Ltd for the exclusive global rights for our investigational RNA-targeted medicine for people living with polycythemia vera (PV).? ? Read more about today’s news:?https://lnkd.in/eb2jVq4C ? #raredisease????

The European Commission has approved Ionis and AstraZeneca’s medicine for the treatment of hereditary transthyretin-mediated amyloidosis in adults with stage 1 or stage 2 polyneuropathy. Learn more about this #raredisease news: https://lnkd.in/exDGJ9_Y

As we celebrate the women in our lives?this #InternationalWomensDay, 2025’s theme, #accelerateaction, is a reminder of the work we still must do to achieve gender equality. This week, EmpowHER, our women’s employee resource group, hosted a virtual panel for all Ionis employees recognizing the contributions of women who came before us and spurring us to support the women in our lives through?mentorship, advocacy and allyship. Participants related stories of their professional and personal?experiences that prove how far individual actions?go toward promoting belonging.? ? What’s one commitment you’re making to accelerate action this year??

Living with hereditary angioedema (#HAE) can mean having to make daily trade-offs and regular accommodations. We hosted a virtual round table discussion in partnership with the Hereditary Angioedema Association - HAEA?to hear from individuals living with HAE about how they manage the mental load that comes with living with a #raredisease and find grace for themselves along the way.? ?? Visit HAEA.org for additional resources and perspectives from the HAE community.???