2018 Lorne Genome Conference

Professor Adrian Krainer

RNA Based Therapeutics

Prof. Adrian Krainer received a B.A. in Biochemistry from Columbia (1981) and a Ph.D. in Biochemistry from Harvard (1986). He joined Cold Spring Harbor Laboratory as a Fellow in 1986, became full Professor in 1994, and Chair of Cancer & Molecular Biology in 2011. His lab investigates fundamental aspects of RNA splicing in normal and disease contexts. They developed powerful ways to correct splicing defects, so far resulting in the invention of the first approved treatment for the neuromuscular disease spinal muscular atrophy (SMA). Prof. Krainer is a Pew Scholar in the Biomedical Sciences, a member of the American Academy of Arts & Sciences, an NIH MERIT Award recipient, and a recipient of the NY Intellectual Property Law Association Inventor of the Year Award (2017) and the FE Bennett Memorial Award of the American Neurological Association (2017). Spinraza, the SMA drug he invented and developed with Ionis Pharmaceuticals and Biogen, received the Prix Galien USA Award to the Best Biotechnology Product (2017), recognizing “extraordinary achievement in scientific innovation that improves the state of human health”.