When I first joined Ionis more than 35 years ago, we were a small group of scientists chasing what many considered to be an impossible dream: creating an entirely new way to design medicines with genetic precision. At the time, antisense technology – an approach that uses short, chemically-modified synthetic strands of DNA or RNA to modulate gene expression – was largely uncharted territory and a burgeoning area of pharmacological exploration. While many companies walked away from the field, we stayed. The science was bold, the risks were high and many questioned our vision.
Over the decades, I’ve had a unique vantage point; I’ve held various roles within the company and seen the evolution of our science and business. One of the biggest lessons I learned is that the way medicines are brought to patients is just as important as the science behind them.
Making a Strategic Shift
In the early years, Ionis’ business model was built on partnerships – we focused all our efforts on the research, discovery and early development of innovative new therapies and then licensed those innovations to trusted partners. And for three decades, we did just that: advancing RNA-targeted medicines and drug discovery and development while racking up several firsts along the way, including the first treatment for spinal muscular atrophy. We had success with this approach, as it enabled us to finance the company and build a strong pipeline, to validate our technology, while offloading risk and expense associated with late-stage development and commercialization.
Over time and as Ionis evolved, however, I recognized the limitations of this model. Despite creating medicines with extraordinary potential, we lacked control over how efficiently and effectively our medicines got into the hands of patients or how they were positioned in the marketplace. We were relying on partners to deliver our medicines, yet we held the clearest understanding of our technology and what our patients needed most. This realization became the cornerstone of my vision as I stepped into the role as Ionis’ chief executive officer.
Becoming a Fully Integrated Biotech for Maximized Patient Impact
Since then, we’ve redefined our strategy so that we can focus on the full development cycle of a medicine – from research to commercialization. This enables us to have a greater, more meaningful impact on the patients we serve. It allows us to tailor clinical trial designs to better fit patient needs, respond faster to clinical research findings and develop market strategies that more closely align with Ionis’ core values. This autonomy also provides greater value for our stakeholders because our investments and returns stay within Ionis, fueling further advancements and reinvestments into our science and technology, as well as our incredible workforce, who are bringing our medicines to life.
The work to get us here has been no small feat. We’ve built a robust commercial organization. We’re using real-world insights to shape our clinical pipeline. We’ve established expertise in commercial and late-stage development, including our regulatory and manufacturing practices. What hasn’t changed is our unwavering curiosity, passion for science and the strong spirit and culture that got us here. This is, and has always been, the foundation of Ionis.
This is Just the Beginning—Working Tirelessly to Deliver More Life-Changing Medicines for Patients We’ve made tremendous progress for patients living with serious diseases through our first independent launches of two new medicines in the U.S.: the first approved medicine for familial chylomicronemia syndrome and the first RNA-targeted medicine for hereditary angioedema. Together, these therapies are already changing lives and bringing new hope to people with these devastating conditions. Beyond that, our pipeline continues to advance at every stage and across many diseases — most recently with positive Phase 3 data in severe hypertriglyceridemia (sHTG) and in Alexander Disease (AxD). These achievements reflect our strong scientific heritage and our vision to deliver better outcomes to patients with urgency – and they offer a glimpse of the future we are creating.
Every day, we’re taking learnings from these launches to continue scaling for growth and widen the potential impact we can make on patients around the world. We’re advancing an expansive pipeline with promising developments on the horizon – anticipating meaningful clinical trial data for investigational therapies in serious diseases like Angelman syndrome, multiple system atrophy and prion disease. The benefits we are seeing for patients continue to inspire our science and strengthen our perseverance, further enabling us to continue delivering groundbreaking medicines for patients in need.
I’m more optimistic than ever about the unprecedented opportunities that lie ahead. This is more than a professional responsibility to me and my colleagues; it’s a passion that has shaped my entire career. With our fully integrated model and a team of more than 1,200 of the brightest minds from academia, industry and medicine, we are accelerating the pace at which we can deliver new medicines to the people who need them most.
The road ahead won’t be without challenges – no journey worth taking ever is – but I’m confident in our scientific expertise, operational strength and ability to push the boundaries of what’s possible in RNA-targeted medicines. Together, we’re setting our sights on enabling better futures for people living with serious diseases.