Enduring Purpose, Expanding Reach
BioMarin was founded in 1997 to make a big difference in small patient populations. In the years that followed, we have established a significant foundation for growth. Going our own way has led to countless breakthroughs, bettering the lives of those living with rare genetic diseases.
As we reflect on the progress of the last quarter century, we take this opportunity to also look forward to what’s next for BioMarin. In our continued pursuit to maximize impact for patients, we continue to ask ourselves: what more can we do? The outcome of this ongoing self-assessment has helped bring clarity to how we can best deliver on our purpose – to transform lives through genetic discovery.
Same Approach, Greater Impact
Our science-driven, patient-forward approach has propelled our last 25 years of drug development and will continue to do so for the next 25 years and beyond. Our historic commitment to rare and ultra-rare communities remains unwavering, while enabling us to do more across other patient communities. Going forward, we will continue to apply our approach in rare diseases while also expanding to larger genetic disorders and genetic subsets of more common conditions to make an even greater impact.
Applying our knowledge to make a transformative impact is not just a calling, but an obligation to those who will benefit most. With each success, we recognize that “those who will benefit most” is an ever-expanding group of individuals for whom we work every day. As such, our expansion beyond rare diseases is a means to further deliver on our deep-rooted and continuing commitment to patients.
Together, our four core attributes enable our people to achieve their full potential and the full potential of our science.
- Genetics: We have unparalleled expertise in genetics and molecular biology and a clear understanding of underlying disease mechanism. Together, this enables us to explore diverse modalities and discover the best approach that will yield the greatest effect.
- Assessable: To yield the greatest effect we must first be able to adequately evaluate our progress. When designing studies, we use readily assessable endpoints that offer a clear efficacy signal and reliability translate into clinical benefit for patients.
- Targeted: We work tirelessly to develop therapies that directly or proximally address the root cause of genetic disorders and genetic subsets of other conditions – whether common or rare.
- Transformational: Regardless of which condition we aim to treat, we will never settle for a marginal effect. Every step we take is to maximize impact on patients’ lives by profoundly improving the way they feel, function, and survive.
The people of BioMarin are proof that science and emotion are not mutually exclusive. We are on a dispassionate search for scientific truth while driven by the emotional connection and commitment we have forged with patients.
The resulting sense of urgency is what propels us forward every day. It motivates us to maximize the impact of genetic innovation. It gives us the courage to be curious. It encourages us to adapt our ways of working to optimize productivity, efficiency, and focus. It compels us daily to pursue our purpose.
The end goal has always been better lives and now we can reach more. And the more people we reach, the more our impact can grow.
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