Pre-Conference Workshop Day

Tuesday, February 21, 2023

8:00 am WORKSHOP A – Optimizing Therapeutic Delivery Across the Blood-Brain Barrier to Reach Neurodegenerative Targets


Amplifying the complexity in CNS research, safely accessing deep brain tissues with sufficient compound quantity is an ongoing difficulty for many therapeutics. Large molecules unable to penetrate the BBB have limited the potential for drugs achieving therapeutic effect on target cells. Several methodologies can be utilized to bring drugs more effectively into the CNS, from shuttle technologies to mechanical delivery. Matching the most appropriate methodology to therapeutic needs and understanding safety and scaling concerns is pivotal to producing clinically efficacious therapies.

Take part in a discussion-based deep dive into:

  • Understanding the challenges with therapeutics penetrating the blood brain barrier
  • Exploring new molecular methods to deliver therapeutics across the blood brain barrier
  • Assessing novel mechanical technologies used to deliver CNS therapies

11:30 am WORKSHOP B – Translational De-Risking Toolkit – Enhancing Utility of Preclinical Models of Alzheimer’s & Parkinson’s Disease


*Speaker to be confirmed, Atuka

Despite technological and scientific progress, a significant challenge in drug development for neurological disorders remains – capturing the complexity of the human brain in laboratory models. Historically, translation has been ineffective and contributed to a catalog of failures in human studies. To continue trialing new drug treatments, an optimized approach must be taken to utilize the currently available methodologies in early research and increase the chance of success at clinical phases.

Attend this practical, solution-focused workshop to:

  • Outline current rodent models, their limitations, and optimal functionality
  • Understand the implications of transgenic models on pathophysiology
  • Improve ability to review data gleaned in preclinical studies and translate into likely clinical effect

3:30 pm WORKSHOP C – Tackling the Genome to Identify New Neurodegenerative Targets

  • Karen He Scientist, Disease Genetics, Janssen


As our knowledge of the human genome increases, the scope of potential targets grows increasingly unwieldy. With largely unexplored genetic links to disease processes, this is a critical stage in linking genetics to Alzheimer’s, Parkinson’s Disease, and related disorders. With a multitude of triggers and pathways in these complex diseases, endless opportunities to match new compounds and repurposed drugs to those at risk or affected.

Connect with experts to have an engaging discourse on vital questions:

  • What is the underlying biology linking newly discovered genetic influences on neurodegenerative disorders?
  • How could these be brought into discovery programs to develop new compounds?
  • Where are the algorithmic approaches that can be taken to use information on genetics in predicting those at the highest risk?