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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

  • Supervisory Pharmacologist, 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

  • Principal Investigator, Comparative Pathology
  • Biogen, United States
Jeff  Allen, PhD

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

Jeff Allen, PhD

  • Associate Director, Toxicology
  • Alnylam Pharmaceuticals, Inc., United States
David  Carbone, PhD

Nonclinical Programs for ASO Development: A Regulatory Perspective

David Carbone, PhD

  • Toxicologist
  • FDA, United States