From Bench to Bedside: Bioanalysis and Translational Science

Keynote: Delivering Biotherapeutics to the CNS via Different Routes of Administration: State-of-the-Art

Keynote Speaker(s)

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  Robert Thorne, PhD (he/him/his)

  Denali Fellow & Adjunct Associate Professor
  Denali Therapeutics & University of Minnesota
  San Francisco, California

Moderator(s)

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  Steven Louie, M.S. (he/him/his)

  Director
  Medicilon -US Corp
  Lexington, Massachusetts

There is currently a shortage of disease-modifying treatments for Alzheimer’s, Parkinson’s and other CNS diseases, despite these disorders representing among the most significant unmet health needs of our time. Our ability to effectively tap into the vast potential that protein, oligonucleotide, and gene therapies have for treating CNS disorders has been sharply limited by the typically insufficient brain exposure that results after their systemic or central administration. Even many orally administered small molecules suffer from brain exposure limitations. Among the primary reasons for these limitations are the physical and biochemical barriers that exist at key CNS interfaces, including the blood-brain barrier at the level of the cerebrovasculature, other specialized barriers between the blood and cerebrospinal fluid, and further obstacles hindering drug exchange between cerebrospinal fluid and CNS tissue. CNS drug delivery research lies at the crossroads between many different fields, including physiology, pharmacology, pharmaceutical science, neuroscience, neurosurgery, engineering, genetics, and vascular biology, among others. Dr. Thorne's talk will provide an overview of the background and top level considerations needed to understand the nature of the CNS delivery challenge for macromolecules and will also highlight where the field is at today with regards to different routes of administration and new technology.