Adrian R. Krainer

Cold Spring Harbor Laboratory

2019 Breakthrough Prize in Life Sciences

For the development of an effective antisense oligonucleotide therapy for children with the neurodegenerative disease spinal muscular atrophy.


I am grateful to the Breakthrough Prize Committee for this amazing honor, and to Ionis, Biogen, my lab members, collaborators, and SMA patients, families, clinicians, and researchers for their contributions to the work this prize recognizes. I especially thank: nusinersen’s co-inventor Yimin Hua, who did the lion’s share of the preclinical development in my lab; Luca Cartegni, whose earlier mechanistic research enabled our discovery; and Cold Spring Harbor Laboratory, the NIH, St. Giles Foundation, the Muscular Dystrophy Association, and the Spinal Muscular Atrophy Foundation. I am indebted to my mentors Cathy Squires (Columbia), Tom Maniatis (Harvard), and Rich Roberts (CSHL). It has been an amazing privilege to devote my career to curiosity-driven research, and to successfully convert the resulting insights into a drug that is extending and improving thousands of lives. I thank my parents, who always encouraged my brother and me to study; my father and my mother’s parents were Holocaust refugees from Romania and Hungary, who emigrated to Uruguay. I was fortunate that the US welcomed me as a foreign student, and later as an immigrant. I am proud of my children, Emily, Andrew, and Brian. And I am lucky to be married to Kate, who makes me happy and a better person.

Adrian R. Krainer