Adrian Krainer, Ph.D. is the St. Giles Foundation Professor at Cold Spring Harbor Laboratory (CSHL), and Deputy Director of Research of the NCI-Designated CSHL Cancer Research Center. He received a Ph.D. in Biochemistry from Harvard University in 1986, followed by postdoctoral research at CSHL.
His research focuses on the mechanisms and regulation of mRNA splicing, and how splicing dysregulation contributes to cancer and genetic diseases, as well as on the preclinical development of antisense therapeutics. One notable success was the development of nusinersen (Spinraza), in collaboration with Ionis Pharmaceuticals and Biogen, as the first approved drug for spinal muscular atrophy. The group has worked on the role of splicing dysregulation in breast, skin, pancreatic, liver, brain, and blood cancers, and is currently developing targeted antisense therapeutics for hepatocellular carcinoma and midline glioma.
Dr. Krainer is a co-founder, director, and chair of the SAB of Stoke Therapeutics. He is a member of the National Academy of Sciences, the National Academy of Medicine, the National Academy of Inventors, and the American Academy of Arts & Sciences. He is a recipient of the Life Sciences Breakthrough Prize, the Lifetime Achievement Award of the RNA Society, the International Prize in Translational Neuroscience, the Bennett Brandwein Award for Genetics Research, the Benett Award of the American Neurological Association, the Speiser Award in Pharmaceutical Sciences, the Ross Prize in Molecular Medicine, the Takeda Pharmaceuticals Innovators in Science Award, and the Wolf Prize in Medicine.
He was appointed to Health Research Science Board service in 2021.