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Session 5 Track B: Progress In Developing Rna Targeted Drugs For the Treatment of Neurological Diseases
Session Chair(s)
Lois Freed, PhD
Director, Division of Pharmacology/Toxicology-Neuroscience (DPT-N), CDER, FDA, United States
Scott Henry, PhD
Senior Vice President, Nonclinical Development, Ionis Pharmaceuticals, Inc., United States
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.
Speaker(s)
Martin Lamb
Principal Investigator, Comparative Pathology, Biogen, United States
Big Vacuoles; Small Problem De-Risking Hippocampal Neuron Macrovesicular Vacuolation
Jeff Allen, PhD
Associate Director, Toxicology, Alnylam Pharmaceuticals, Inc., United States
Recent Developments for Delivery and PD Activity of siRNAs in the CNS
David Carbone, PhD
Toxicologist, FDA, United States
Nonclinical Programs for ASO Development: A Regulatory Perspective
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