Currently there are multiple genetic based medicines being pursued for rare neurological diseases including antisense technology, gene therapy and gene editing technologies. Antisense oligonucleotides (ASOs are one of the more advanced technologies. ASOs are synthetic, chemical modified nucleic acid analogs designed to bind to RNA by Watson-Crick base paring. Upon binding to the RNA, ASOs modulate the function of the targeted RNA through a variety of mechanisms. Both protein coding, as well as non-coding RNAs, can be targets of ASO based drugs, significantly broadening therapeutic targets for drug discovery compared to small molecules and protein based therapeutics. The approval of nusinersen (Spinraza™) as a treatment for spinal muscular atrophy (SMA) and the recent approval of tofersen (Qalsody) for familial ALS validates the utility of antisense drugs for the treatment of motor neuron diseases. The application of antisense technology as potential therapy for other rare neurodegenerative diseases and neurodevelopmental disorders will be discussed.
About the Keynote Speaker: C. Frank Bennett, Ph.D
Dr. Bennett is the executive vice president and chief scientific officer at Ionis Pharmaceuticals and one of the founding members of the company. He is responsible for continuing to advance Ionis’ technology and expanding the company’s drug discovery platform. Dr. Bennett is also the franchise leader for gene-editing 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. Bennett lead the discovery and development of nusinersen and the discovery of tofersen and tominersen.