Gene editing holds the potential for a transformative approach to the therapy of CNS disorders. The emergence of novel gene-editing tools such as CRISPR, TALEN, Zinc Finger Nucleases and Homologous Recombination opens new possibilities in the development of disease relevant animal models and, more importantly, in highly specific therapeutic interventions.

In order for these technologies to come to fruition, several challenges need to be addressed, including efficient delivery to target areas and selective editing of neuronal and non-neuronal cell populations.

This workshop will introduce the potential applications of the gene-editing technology in rare neurodegenerative diseases from preclinical study design to therapeutic potential.

Join the session to:

• Uncover protocol advancements for advanced in vitro/vivo editing with optimized specificity
• Learn about the different applications of CRISPR, TALEN and AAV-edited transgenic mice in a biomedical research setting
• Overcome the current limitations of using gene editing tools for insertions, for higher efficiency of desired edits

Ottavio Vitolo, VP Clinical Development, Homology Medicines Inc

Whilst microglia have long been implicated in the pathology of neurodegenerative disease, recent accumulating evidence has pointed to these cell types as a driving factor in neuronal damage and disease progression for AD, PD and others.
Although different models have been established to study these diseases and cell type strategies, their effectiveness has been limited. In order to understand the interplay of underlying mechanisms and to investigate new therapeutic modalities, there is a need to increase human-relevance and accuracy. Recent technological advances, including IPSCs, now grant access to substantial quantities of disease-pertinent neurons, both with and without predisposing mutations.

• Join this session to:
  Understand the accumulation of genetics and human biology implicating innate immune function as being integral to diseases such as Alzheimer’s and Parkinson’s
• Discuss of mechanisms and the biological pathways linked to the human genetics of these diseases
• Therapeutic targets and levers – where could we intervene to alter microglial phenotypes and modify disease progression?

Samuel Hasson, Principal Investigator, Neuroscience, Pfizer

Emerging data suggests that there is a link between changes in the gut microbiome and neurological diseases and disorders. This exciting bi-directional communication via the proposed gut-brain axis or GBA, has catapulted microbiota into the scientific spotlight for CNS disorders. However, with the size and complexity of the Gut-Brain Axis, there still remains a number of fundamental questions to allow scientific researchers to forge a successful path in demonstrating causality for a number of disease indications and ultimately developing target specific therapeutics.

As these two hot areas of research come together, now is the time to understand how we can continue to fuel this dynamic relationship.

Join this session to:

• Receive an overview of recent scientific advances in unravelling the gut-brain connections and potential pathways to harness these findings for novel therapeutic approaches
• Consider different approaches to modulation or supplementation of the microbiome
• Discuss the challenges associated in developing microbiota-associated therapeutics and strategies to overcome them
• Hear perspectives of microbiota-associated therapeutics in the personalized medicine era

Patrice Garnier, CEO, Amabiotics