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Bethesda North Marriott Hotel and Conference Center

Oct 28, 2019 7:00 AM - Oct 30, 2019 12:30 PM

5701 Marinelli Road, , North Bethesda, MD 20852 , USA

DIA/FDA Oligonucleotide-Based Therapeutics Conference

Session 5 Track B: Progress In Developing Rna Targeted Drugs For the Treatment of Neurological Diseases

Session Chair(s)

Lois M. Freed, PhD

Lois M. Freed, PhD

Director, Division of Pharmacology/Toxicology-Neuroscience (DPT-N), CDER

FDA, United States

Scott  Henry, PhD

Scott Henry, PhD

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.


Martin  Lamb

Big Vacuoles; Small Problem De-Risking Hippocampal Neuron Macrovesicular Vacuolation

Martin Lamb

Biogen, United States

Principal Investigator, Comparative Pathology

Jeff  Allen, PhD

Recent Developments for Delivery and PD Activity of siRNAs in the CNS

Jeff Allen, PhD

Alnylam Pharmaceuticals, Inc., United States

Associate Director, Toxicology

David  Carbone, PhD

Nonclinical Programs for ASO Development: A Regulatory Perspective

David Carbone, PhD

FDA, United States


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