Humanizing Drug Discovery
In the past 30 years, genetics and genomics have exponentially expanded our understanding of human biology and disease. That understanding has the greatest potential benefit for society when it catalyzes the discovery and development of new medicines with the potential to transform the lives of patients in need.
David Altshuler will discuss two recent examples of the combination of genetic insights into human biology and the invention of new treatment modalities. Specifically, he will focus on protein-folding correction for cystic fibrosis and investigative CRISPR-based gene-editing approaches for sickle cell disease and beta thalassemia.