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Dr. Bennett is responsible for preclinical antisense drug discovery and antisense technology research at Ionis Pharmaceuticals. He is also the franchise leader for neurological programs at Ionis. He has been involved in the development of antisense oligonucleotides as therapeutic agents, including research on the application of oligonucleotides for inflammatory, neurodegenerative diseases and cancer, oligonucleotide delivery, pharmacokinetics and medicinal chemistry. Dr. Bennet is a co-recipient of the 2019 Breakthrough Prize in Life Sciences for his contributions to the discovery and development of SPINRAZA® (nusinersen) and the 2018 Hereditary Disease Foundation’s (HDF) Leslie Gehry Brenner Prize for Innovation in Science for his leadership and continued commitment to developing antisense therapies for Huntington’s disease (HD). Dr. Bennett has published more than 200 papers in the field of antisense research and development and he is an inventor on more than 175 issued patents.
The healthy individual participant is at the center of population sequencing studies. Spinal muscular atrophy (SMA) is a severe pediatric neuromuscular disorder due to a defect in the Survival Motor Neuron 1 (SMN1) gene that presents with a diverse range of phenotypes of motor impairment and related comorbidities – effecting approximately 1 in 10,000 live births. Research has focused on strategies that restore sufficient levels of needed full-length SMN protein. This resulted in FDA approval of Spinraza, which is using antisense oligonucleotides to cause more full-length SMN production from SMN2 genes, and the new gene therapy Zolgensma, that is based on transferring SMN1 genes into the body. The session will review the two different approaches and the challenges to bring them to the clinic.