Don't miss these highlights from Day 2:
Panel Discussion: Explaining Clinical Failures – Is Efficacy or Delivery
- Which CNS drug delivery technologies are looking the most
promising for the clinic?
- With recent clinical failures, is efficacy or delivery likely to be
- What’s next for CNS drug delivery approaches?
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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