DIA/FDA Oligonucleotide-Based Therapeutics Conference

The approval of Spinraza for the treatment of spinal muscular atrophy has demonstrated that locally administered RNA targeting drugs have the potential to treat a broad array of neurological disease. The challenge is to fully understand how these compounds are interacting with the CNS compartment and how studies in animals can inform the design of patient clinical trials. This session will review progress toward developing antisense and siRNA drugs for the treatment of CNS diseases. The focus will be on nonclinical safety testing, systemic, tissue exposure evaluation, and regulatory considerations.