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Dr C. Frank Bennett, has been announced as a recipient of the Breakthrough Prize in Life Sciences in recognition of his contribution to the discovery and development of the first drug approved for the treatment of spinal muscular atrophy (SMA).
Dr. C. Frank Bennett, senior vice-president of research and franchise leader for neurological programs at Ionis Pharmaceuticals, has been announced as a recipient of the Breakthrough Prize in Life Sciences in recognition of his contribution to the discovery and development of the first drug approved for the treatment of spinal muscular atrophy (SMA). Sharing the award with Dr Bennett will be Dr. Adrian Krainer of Cold Spring Harbor Laboratory.
Bennett and Krainer discovered the first drug to treat the rare genetic disease, SMA, using Ionis' antisense technology and the natural process of RNA splicing. The drug, Spinraza, is marketed by Biogen and approved by FDA in 2016, is currently available for patients worldwide.
SMA is a neurodegenerative disease characterised by a loss of motor neurons in the spinal cord and lower brain stem, resulting in severe and progressive muscular atrophy and weakness. It is a leading genetic cause of infant death, with most of those who are severely affected by the disease dying by the age of 18 months.
"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," said Dr. Bennett in a press release. "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."
The first of the Breakthrough Prizes (in Fundamental Physics) was founded in 2012 by Yuri Milner. A year later, in 2013, the Breakthrough Prize in Life Sciences was founded, which is sponsored by Sergey Brin, Priscilla Chan and Mark Zuckerberg, Pony Ma, Yuri and Julia Milner, and Anne Wojcicki. The life sciences prize honors transformative advances towards understanding living systems and extending human life.
Source: Ionis Pharmaceuticals