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RNAi Therapeutics: From Eureka! Moment to a Potential New Class of Medicines
February 28, 2018
How far science and technology have come! At Alnylam, we believe we’re on the verge of a landmark moment in medicine and I can’t help but express my excitement for what lies ahead: the potential to transform the lives of patients living with rare diseases that have had devastating impact on their lives.
The science, called RNA interference (RNAi), led to the development of small interfering RNAs (siRNAs) as therapeutics, the first of which may soon be approved. The mechanism of RNAi is different from any other type of medicine. What began with research on the humble petunia flower and, later, worms, led to a once-in-a-lifetime Eureka! moment—the discovery of RNAi and the confirmation that it worked in human cells. While this finding was revolutionary and recognized as such with a Nobel Prize several years later, that discovery was only the beginning. It provided the means for an idea, a distant vision of something that had never been done before. That moment proved to be the starting line, the place where it all began: 15 years of dedicated effort, bold investment, and immense optimism that was bolstered by breakthroughs and stubborn persistence in the face of setbacks.
Like new and transformational classes of medicines before RNAi—vaccines, monoclonal antibodies, and CAR-T cell therapy—these medicines in development have the potential to have an immense impact on society by providing new options for rare diseases that were once considered untreatable, and give hope to tens of thousands of patients around the world. It just doesn’t get any more exciting than that!
And it doesn’t happen without purposeful people. At Alnylam, we’ve been truly fortunate to work with remarkable individuals, all of whom responded with a resounding “challenge accepted!” during each stage of this journey. Their stories will be acknowledged in the coming months through this New Class of Medicineswebpage. In the meantime, here’s a glimpse at who they are and why they’ve been so critical to our success:
Dreamers. Scientists who elucidated the biology of RNAi and realized that this discovery had the potential to transform the way we treat disease. This was the origin of the development of RNAi therapeutics.
Pioneers. The people who have been with Alnylam since the beginning and who dared to transform the possibility of RNAi into a reality. They persisted in the face of astounding challenges and shaped the vision of what this new discovery could mean for medicine. Their determination opened doors for RNAi to become a powerful new way to develop medicines.
Believers. The people who supported Alnylam through all the round-table meetings, red-eye flights, and clinical trial readouts, faced the optimism and the doubt head-on, and made the choice to keep fighting for the potential of RNAi therapeutics.
Heroes. The brave people living with devastating diseases and the healthcare and advocacy communities working to help them. Together, they are working to change lives, and they will be the first to realize the promise—and the hope—of a whole new class of therapies.
Over the next few months, I invite you to follow Alnylam on Twitter and LinkedIn to stay up to date on our progress. In the meantime, thank you for joining us on this journey!
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