Don't miss these highlights from Day 2:

Panel Discussion: Explaining Clinical Failures – Is Efficacy or Delivery
to Blame?

  • Which CNS drug delivery technologies are looking the most
    promising for the clinic?
  • With recent clinical failures, is efficacy or delivery likely to be
    the cause?
  • What’s next for CNS drug delivery approaches?
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Led by:

Sophie Parmentier Batteur, Director, Neurodegenerative Diseases Discovery, Merck
Allan Jensen, Senior Director, Biotherapeutic Discovery, Lundbeck
Andrew Goodearl, Senior Director, Biologics Discovery Group, AbbVie

Beyond small molecules and protein therapeutics: new modalities for treatment of CNS diseases.

  • New modalities is a new norm in CNS drug development.
  • Targeting transcriptome with oligonucleotides and genome with gene therapies was shown effective in treating previously untreatable diseases such as spinal muscular dystrophy.
  • Distribution of the new modalities drugs plays a key role in achieving a desired effect.
  • Oligonucleotides cannot penetrate blood-brain barrier.
  • Intrathecal administration directly into CSF allows targeting all CNS cell types and pursue oligonucleotides for wide variety of neurodegenerative diseases.

Talk by: Natasha Penner, Director, Clinical Pharmacology and Pharmacometrics, Biogen

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