The life-changing science of Ionis Pharmaceuticals’ antisense technology platform has once again been recognized by the scientific community. Dr. C. Frank Bennett, senior vice president of research and franchise leader for neurological programs at Ionis, will receive the prestigious Breakthrough Prize in Life Sciences. Dr. Bennett will be recognized for his contribution to the discovery and development of the first approved drug for the treatment of people with spinal muscular atrophy (SMA), a devastating neurodegenerative disease that is the leading genetic cause of infant death. Dr. Bennett will share this award with Dr. Adrian Krainer of Cold Spring Harbor Laboratory.
SMA is characterized by loss of motor neurons in the spinal cord and lower brain stem, resulting in severe and progressive muscular atrophy and weakness. The Breakthrough Prize acknowledges the discoveries of Drs. Bennett and Krainer related to antisense technology and the natural process of RNA splicing leading to discovery of the first drug to treat SMA — nusinersen, marketed by Biogen as SPINRAZA. The drug was approved by the FDA in 2016 and is now available to patients around the world. SPINRAZA is one of many antisense therapies approved or in development for a broad range of diseases. Dr. Bennett and his team’s work in developing nusinersen has also paved the way for new potential therapies for other neurodegenerative diseases, such as Huntington’s disease, amyotrophic lateral sclerosis (ALS), spinocerebellar ataxias, Parkinson’s disease and Alzheimer’s disease, all being developed using Ionis’ proprietary antisense technology.
“I’m humbled and deeply honored to accept the Breakthrough Prize and am proud to share it with Adrian. This achievement recognizes the work, not of one or two individuals, but the collaborative effort of many. It began with the vision and leadership of Dr. Stanley T. Crooke, founder of Ionis Pharmaceuticals and continued with dedicated scientists, patients who participate in clinical programs and their caregivers. At Ionis, we know that sick people depend on us. That knowledge fuels our passion to discover and develop new, innovative therapies that can help people live better, healthier lives,” said Dr. Bennett.
Drs. Bennett and Krainer will receive the award at a televised gala ceremony held in Silicon Valley on November 4.
Founded in 2013, Breakthrough Prizes recognize the world’s leading scientists in life sciences, fundamental physics and mathematics. The life sciences prize honors transformative advances toward understanding living systems and extending human life. The awards are sponsored by Sergey Brin, Priscilla Chan and Mark Zuckerberg, Pony Ma, Yuri and Julia Milner, and Anne Wojcicki.