On this Rare Disease Day—and as is the case each year on February 28—I’m reminded of why I founded Global Genes nearly a decade ago. At that time, I was just coming to understand the magnitude of rare diseases and how devastating they can be for families, as I witnessed with my friends and their entire family. It’s crazy to think that, for the 350 million people on this planet affected by one of the 7,000 rare diseases, treatments are available for less than 5% of those diseases. We as a society need to do better. And although currently the number of treatments is small, the good news is that major advances in science and technology are bringing hope to patients, and that more treatments are on the horizon.
We are inspired by and grateful for the work that many of our biotechnology partners are doing, and their commitment to advancing efforts in rare disease.
We are only at the beginning of what I believe is a new era in modern drug discovery and development. Advances in science mean we can better understand how diseases that have baffled us in the past—particularly rare diseases—work at their core. Advances in technology mean we can target diseases in incredibly precise ways.
We are inspired by and grateful for the work that many of our biotechnology partners are doing, and their commitment to advancing efforts in rare disease. RNA interference (RNAi) is one of those areas of innovation that has the potential to make a huge impact on rare disease therapy development. We in the patient community are optimistic that advances like this will open new doors to treatment for a long line of diseases where no options and little hope currently exist.
As Global Genes discusses often, much of the success and forward movement that we now see within biotech happens as a result of the increased engagement and partnerships with patients and patient advocacy organizations. Patients have an increasing role in drug development and have become critical participants in seed-funding early research and designing and participating in clinical trials. They have become important strategic partners throughout the drug development cycle. There are a few companies that are doing this well, showing us what’s possible when an entire community puts its most determined hearts and smartest minds behind tackling the seemingly impossible. Alnylam is one of those companies that has stayed the course to transform the promise of the science. Patients don’t get a day off, but through their determination they have served as partners, advisors, and clinical trial participants helping to advance this emerging new way to treat disease.
We know that there are many challenges that drug developers face when working to bring innovative therapies to market. We know that it isn’t easy, and that it takes persistence and tenacity to see it through. Join Global Genes in applauding the work of our industry partners who are working tirelessly to bring hope to patients through their work. Head to Alnylam and Global Genes on Twitter for more.