Pre Conference Workshop Day | Tuesday, June 6 2023


9:00 am Workshop A: Methods in Neurological Genomic Target Identification: Widening the Indication Net to Expand Oligo Portfolios


With oligonucleotides rapidly becoming a more exciting and feasible therapeutic option, the possibility of treating previously undruggable CNS disease is endless. Identifying key, novel and validated targets will be crucial in staying ahead of the curve. From neurodegeneration to neurodevelopmental disorders, broaden your pipeline and understand how to develop novel therapeutic candidates for a range of conditions.

Explore the possibilities of future CNS research and:

• Discuss methods in target identification for oligonucleotide therapies

• Identify potential for further reach of these modalities to treat unserved rare neurodevelopment disorders

• Tackle common problems in genetic and protein targets for CNS disorders from ineffective knockdown to off target effects

• Unlock future potential by reviewing computational and AI methods for predicting phenotypic alterations in molecules to optimize your pipeline

12:15 pm Networking Lunch

12:30 pm Workshop C: Categorizing Clinical Outcome & Biomarker Measures in Oligo Trials for CNS Disease

  • Aaron Yarlas Director, Clinical Outcome Assessments, Ionis Pharmaceuticals


As oligonucleotides are increasingly being demonstrated as safe, viable options for a range of conditions, the requirements to demonstrate clinical efficacy in studies loom. Outlining considerations in clinical outcome scores for common and orphan neurological conditions allows effective and efficient studies.

Advance your knowledge in this practical workshop to:

• Improve your clinical trial design by understanding validated clinical outcome measures for a range of disorders: from neurodegeneration moving from qualitative to quantitative markers, to rare disease with limited historical patient data

• Understand the unique challenges of biomarkers for orphan versus common neurological disorders and strategize steps forward


9:00 am Workshop B: Oligonucleotide CNS Delivery Methods to Breach the Brain Barrier


With delivery to CNS and target brain cells representing the biggest hurdle in development, optimizing through established and novel methodologies is a viable and important component for drug development. However, with different options available for bypassing the blood brain barrier, selecting the most appropriate technology can prove challenging. Get up-to-date with the latest research and explore the pioneers in CNS drug delivery.

Take innovation inspiration from this workshop and:

• Review the different methods of mechanical delivery to the CNS – from industry standard intrathecal to endonasal

• Understand the implications for distribution of therapeutic to intended target tissue in the brain

• Debate the latest data and future research needed to allow further implementation

12:15 pm Networking Lunch

12:30 pm Workshop D: Gene Silencing with CRISPR: Utilizing Platforms to Transform Neuroscience

  • Thomas Gaj Assistant Professor, University of Illinois


CRISPR technology presents an exciting opportunity to enable precise and efficient effects on genomic targets. With successes across a broad range of oncological and hematological work, inspiration is given that these therapeutics can be utilized in the challenging field of neuroscience. This workshop will explore some of the pioneering research done to advance therapeutics and basic translational science in the CNS.

• Draw on expertise in Cas9 and 13 nucleases and single-base editors to understand their potential in targeting genes in the CNS

• Explore the versatility of CRISPR systems in neurological disorders with guided examples in neurodegeneration

• Outline AAV and oligonucleotide guide RNA approaches for effective delivery of CRISPR systems

• Review in-vivo and in-vitro studies and identify the key challenges in these systems in toxicity and translatability and potential solutions