Workshop A: Pioneering Modalities for ALS: Unpacking the Gene Therapy Toolbox & Beyond
Time: 9:00 am
day: Pre Conference Day Workshop Track A
Details:
From gene editing and augmentation to emerging delivery systems and knockdown strategies, the session will provide a comprehensive exploration of the gene therapy landscape. Participants will discuss which technologies are best suited for targeting specific ALS mutations, the challenges of sporadic versus hereditary forms, and the broader application of current platforms. By analyzing both successes and setbacks, such as the lessons from C9orf72 and SOD1, the workshop aims to define a roadmap for optimizing genetic medicines “for ALS.”
- Examine the potential of gene editing, viral and non-viral delivery, and vectorized antibodies, and their relevance in various use cases to address ALS pathologies effectively
- Strategize on targeting more complex mutations e.g C9orf72 requiring balancing gain- and loss-of-function approaches within the gene therapy framework
- Identify how current platforms and delivery technologies can overcome challenges in targeting sporadic and hereditary ALS populations
- Evaluate the potential of peripheral administration for gene therapies to explore comparable efficacy and safety
- Navigate future directions and regulatory requirements for gene therapy in ALS, exploring new modalities and technologies to enhance therapeutic impact and accessibility
