Two scientists behind Biogen’s rare disease treatment Spinraza won the richest award in the life sciences on Wednesday, splitting a $3 million prize and a turn on stage at a star-studded ceremony next month.
C. Frank Bennett, a pharmacologist at Ionis Pharmaceuticals, and Adrian Krainer, a biochemist at Cold Spring Harbor Laboratory, picked up a Breakthrough Prize for their work discovering a treatment for the rare and deadly condition spinal muscular atrophy. The drug, developed by Ionis and marketed by Biogen, won Food and Drug Administration approval in 2016.
Before Spinraza, spinal muscular atrophy, or SMA, was the leading genetic cause of death for infants and toddlers. Children with the disease fail to develop along normal timelines, struggling to crawl, sit up, and walk. Many lose the the ability to swallow, and in the worst cases, eventually die from respiratory failure.
Spinraza is a first-of-its-kind treatment that targets the gene responsible for producing a protein called SMN, which is key to motor development. The debilitating effects of the disease result from a deficiency of SMN, and Spinraza works by tinkering with the defective gene and replacing the missing protein.
Bennett and Krainer, who have worked together on SMA research since 2004, will receive their award Nov. 4 at a gala in Mountain View, Calif. The event, hosted by actor Pierce Brosnan, will be broadcast on National Geographic.
The Breakthrough Prize, billed by its organizers as “the Oscars of science,” is funded by the Silicon Valley billionaires behind Google, Facebook, and DST Global. The red-carpet affair, which debuted in 2013, puts researchers on the same stage as celebrities in what its founders say is an effort to elevate the work of scientists.