"A new era for treating neurological diseases" HDF 2018 Symposium, Dr. C. Frank Bennett, Ionis
Channel: HereditaryDiseaseFoundation
Date: 2019-01-29

The mood within the room was electric with excitement and hope as Dr. C. Frank Bennett, senior vice president for research and franchise leader for neurological programs at Ionis Pharmaceuticals, took the podium at the Hereditary Disease Foundation October 22, 2018 Gala. He gave a mesmerizing talk
about his work developing antisense therapeutics for neuromuscular and neurological diseases. His pioneering work brought us the world’s first approved treatment for spinal muscular atrophy (SMA). In 2005, Frank started a program to develop antisense therapies for Huntington’s disease. IONIS-HTTrx is designed to treat patients with HD and is the first and only drug to demonstrate reduction of the neurotoxic abnormal huntingtin protein, the underlying cause of HD, in patients. Ionis is partnering with Roche for phase 3 of the trial, which is set to begin in the U.S. and abroad soon!! This is a groundbreaking advance for the field of HD!