Unlocking the potential of RNA therapies in autism spectrum disorder: How definitive human iPSC-derived cells are paving the way

In this webinar, Dr. Rodney Bowling, Chief Scientific Officer, Everlum Bio, will discuss how new GABAergic neuronal models are advancing the screening of antisense oligonucleotide (ASO)-mediated RNA therapies for rare autism spectrum disorders (ASD) affecting children.

ASOs have significant potential for neurological disorders as they target specific RNA sequences, either to block the translation of disease-causing proteins or to encourage the production of therapeutic proteins. Human iPSC-derived GABAergic neurons offer a useful physiologically relevant system to investigate ASO efficiency but their generation is hindered by heterogeneous populations and lot-to-lot inconsistencies.

Dr. Malathi Raman, Senior Product Manager, bit.bio, will explore how ioGABAergic Neurons, a new class of precision reprogrammed hiPSC-derived cells, overcomes these limitations, yielding highly pure, consistent, and functional populations of mature human iPSC-derived inhibitory GABAergic neurons. Dr. Bowling will then explain how this new class of inhibitory neurons are being applied within ASO-based screening assays and drug discovery workflows for rare ASD.

Key learning objectives

• Explore how ASOs can be used to discover and develop drugs for rare ASD affecting children
• Learn about the need for a consistent, physiologically-relevant and scalable in vitro cellular screening model to identify optimal ASOs for an RNA target for ASD
• Understand the design considerations for building optimal ASOs, including modifications to improve ASO binding affinity and effectiveness
• Discover how precision reprogrammed hiPSC-derived ioGABAergic Neurons are being applied within ASO screens to discover personalized oligonucleotide-based therapies for rare ASD

Who should attend?

• Sector: CRO, biotech, academia, and pharma
• Persona: Group leaders, senior scientists, innovation leads, and researchers
• Disease indication: Neurodevelopmental disorders, rare diseases affecting children, autism spectrum disorders

Certificate of attendance

All webinar participants can request a certificate of attendance, including a learning outcomes summary, for continuing education purposes.