We like to think that we understand a lot about disease and the molecular basis of disease and exactly how to address, particularly, single-gene disorders with gene therapy. But the fact is, we still have a lot to learn.
Katherine High, MD President, Therapeutics, AskBio
We are going to see more gene therapy treatments cross the finish line in the next two years. Finally, for the first time, patients will have hope that they might be able to live a relatively normal life.
Jill Weimer, PhD Senior Director of Therapeutic Development, Sanford Research
Michael R. Yeaman, PhD Professor and Chief of Molecular Medicine, UCLA School of Medicine; Director, Institute for Infection & Immunity, Lundquist Institute at Harbor-UCLA; Chair, Medical Sciences, Guthy-Jackson Charitable Foundation By understanding the process through which the immune system differentiates between self and non-self, we have opportunities to solve, not just autoimmune disease, but infection, cancer, transplant rejection and many other conditions where the immune system is functioning in a way that’s not optimal.
We are always trying to unravel the mysteries of human biology, but it turns out that it’s really hard to do. When it comes to rare diseases, we are in a race against the clock; we are also in a race against the mistakes of nature.
John F. Crowley Chairman of the Board and CEO, Amicus Therapeutics
I am very optimistic. The first drug law in the U.S. was in 1906. In 2016, President Obama signed The 21st Century Cures Act . The word patient didn’t appear for over 100 years in U.S. drug laws. Now, we are inviting patients to have a seat at the table.
Frank J. Sasinowski, JD, MPH Director, Hyman, Phelps & McNamara
Peter Marks, MD, PhD Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration It’s time for global, worldwide trials. There’s no way we will do justice to people with rare diseases globally unless we do these. There simply aren’t enough patients to succeed otherwise.
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