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The inventor and developer of a drug, nusinersen (Spinraza), are among the winners of the Breakthrough Prize in Life Sciences. Adrian Krainer (pictured) of Cold Spring Harbor Laboratory and Frank Bennett, Senior Vice President of Research at Ionis Pharmaceuticals, share the $ 3 million prize. The Breakthrough Awards, which are in their seventh year, feature as the world's largest science prize and are sponsored by Sergey Brin, Priscilla Chan and Mark Zuckerberg, Ma Huateng, Yuri and Julia Milner and Anne Wojcicki.
Nusinersen, marketed by Biogen (NASDAQ: BIIB), is used to treat a rare childhood illness called spinal muscular atrophy (SMA) and was the first drug approved by the FDA for this serious and potentially fatal genetic disorder.
SMA causes muscle loss that can lead to death and is caused by a defect in a gene called SMN1. Krainer has found a way to compensate for this error by increasing the production of a protein made from a related gene, SMN2. He and his team worked with researchers at San Diego-based Ionis (NASDAQ: IONS), led by Bennett, to develop a small molecule of RNA called an antisense oligonucleotide (ASO) that binds to the molecule. SMN2 RNA and increases the production of functional SMN protein. . Ionis began testing nusinersen in humans in 2011, has partnered with Biogen to continue its development and the drug has been approved by the FDA in 2016.
Krainer recently co-founded a Boston-based start-up, Stoke Therapeutics, which works on ASO objects for other genetic diseases.
Other winners of this year's Breakthrough Life Sciences awards are Angelika Amon, of MIT, for her work on the effect of abnormal numbers of chromosomes, Xiaowei Zhuang, of Harvard, for her invention of a technique known as 39, very high resolution imaging that revealed new cell structures, and Zhijian. "James" Chen of the Southwestern Medical Center at the University of Texas, for his research on how cells detect and respond to foreign DNA. Jennifer Doudna and Emmanuelle Charpentier, the main discoveries of the CRISPR-Cas9 gene editing, are among the previous winners.
See here for the winners of the Basic Physics and Mathematics Awards.
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