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Spinraza, the First Approved Drug for Spinal Muscular Atrophy (HHS Only)

Air date: Friday, October 19, 2018, 12:00:00 PM
Time displayed is Eastern Time, Washington DC Local
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Description: NCI’s Center for Cancer Research (CCR) Grand Rounds

Adrian Krainer, Ph.D. was born in Montevideo, Uruguay. He received a B.A. in Biochemistry from Columbia University (1981) and a Ph.D. in Biochemistry from Harvard University (1986). Dr. Krainer joined Cold Spring Harbor Laboratory as the first Cold Spring Harbor Fellow in 1986, became a faculty member in 1989 and a full professor in 1994. He also serves as Program Chair of Cancer and Molecular Biology. Beginning with his doctoral studies with Prof. Tom Maniatis, he has been studying fundamental aspects of pre-mRNA splicing mechanisms and alternative-splicing regulation in normal and disease contexts. He and his collaborators developed powerful ways to correct splicing defects and modulate alternative splicing, based on antisense technology, so far resulting in the invention of the first approved treatment for the neuromuscular disease spinal muscular atrophy (SMA). Dr. Krainer is a Pew Scholar in the Biomedical Sciences, a member of the American Academy of Arts & Sciences, an NIH MERIT Award recipient, a former President of the RNA Society, and a recipient of the New York Intellectual Property Law Association Inventor of the Year Award (2017), the FE Bennett Memorial Award of the American Neurological Association (2017), and the Herbert & Esther Bennett Brandwein Award in Genetic Research (2018). Spinraza, the SMA antisense oligonucleotide drug he invented and developed with Ionis Pharmaceuticals and Biogen, received the Prix Galien USA Award to the Best Biotechnology Product (2017), recognizing outstanding scientific innovation and contributions which improve human health..
Author: Adrian R. Krainer, Ph.D., Professor, St. Giles Professorship of Neuroscience Cold Spring Harbor Laboratory
Runtime: 1 hour