Adrian Krainer is the St. Giles Foundation Professor at Cold Spring Harbor Laboratory. His lab studies the mechanisms and regulation of messenger RNA splicing in human cells, and the role of splicing dysfunction in genetic diseases and cancer. His lab is also engaged in the preclinical development of antisense-oligonucleotide drugs that target RNA splicing or other RNA-processing steps. Together with Ionis Pharmaceuticals and Biogen, Adrian’s lab developed an antisense drug called nusinersen (Spinraza), which was approved by the FDA in 2016 as the first treatment for spinal muscular atrophy, a neurodegenerative disease that was the leading genetic cause of infant mortality. His awards and accolades include election to the National Academy of Sciences, the National Academy of Medicine, the National Academy of Inventors, and the American Academy of Arts & Sciences; the Life Sciences Breakthrough Prize, the Lifetime Achievement Award of the RNA Society, the International Prize for Translational Neuroscience, the Speiser Award in Pharmaceutical Sciences, the Takeda Pharmaceuticals Innovators in Science Award, the Ross Prize in Molecular Medicine, the Wolf Prize in Medicine, the Gabbay Award in Biotechnology & Medicine, the Watanabe Prize in Translational Research, the Albany Prize in Medicine & Biomedical Research, and an honorary doctorate from Tel Aviv University. Adrian served as President of the RNA Society in 2014, and currently serves on the advisory boards of several scientific centers and networks, non-profit foundations, and biotechnology companies in the U.S., Europe, and Latin America. He is a co-founder and Director of Stoke Therapeutics.