WORKSHOP TRACK A
DISCOVERY & PRECLINICAL
WORKSHOP TRACK B
CLINICAL & TRANSLATIONAL UTILITY
Designed for CSOs, preclinical researchers, discovery scientists, directors of biology and research, novel modality scientists, in vivo/in vitro modeling experts, and many more; connect with an intimate group of discovery and preclinical experts for a more discursive session
Tailor-made for medical directors, CMOs, clinical program leads, translational and clinical development heads, clinicians, and more; share expertise from diverse clinical challenges and innovation in ALS
9:00 am Workshop A: Pioneering Modalities for ALS: Unpacking the Gene Therapy Toolbox & Beyond
Synopsis
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
9:00 am Workshop B: Decoding Clinical Endpoints in ALS: Revolutionizing Drug Development with Emerging Biomarkers, ALS-FRS-R & Digital Endpoints
Synopsis
This workshop explores the ALS Functional Rating Scale-Revised (ALSFRS-R) as a cornerstone clinical endpoint, addressing its limitations and strategies to enhance its impact. We’ll investigate the role of digital biomarkers, such as wearables and smartphone-based assessments, alongside other primary and secondary endpoints like respiratory function, survival, and quality of life. Attendees will gain actionable insights into building a robust, multidimensional framework for trial design, aligned with regulatory requirements, to accelerate ALS drug development and approvals.
- Critically examine the ALSFRS-R to analyze the strengths, limitations, standardization concerns and key considerations when using ALSFRS-R as a clinical endpoint, including its sensitivity to disease heterogeneity
- Evaluate the role of wearables, smartphone-based assessments, and AI-driven analysis as digital endpoints in capturing nuanced progression signals for ALS
- Integrate diverse endpoints to discuss the interplay of primary, secondary, and exploratory endpoints—such as respiratory function, survival rates, and quality-of-life measures—in building a comprehensive efficacy profile
- Streamline approvals to align endpoint selection with regulatory expectations, fostering more efficient pathways for drug approval
12:00 Lunch & Networking
1:00 pm Workshop C: To What Extent Does TDP-43 Underlie ALS Pathology? Evaluating Novel Target Discovery & Validation to Address the Breadth of ALS Pathology
Synopsis
This workshop will delve into TDP-43’s role as a key driver of ALS pathology and explore broader mechanisms contributing to disease heterogeneity. Attendees will critically assess TDP-43 as a therapeutic target, how well TDP-43 accounts for all ALS pathology in sporadic ALS, identify other pathological drivers such as neuroinflammation and metabolic dysfunction, and refine preclinical strategies to validate novel targets.
The discussion will address the challenges of model selection, biomarker development, and the integration of TDP-43-targeted therapies into a multifaceted ALS treatment landscape.
- Evaluate TDP-43’s role in ALS pathology and its interaction with non-neuronal mechanisms, including neuroinflammation, mitochondrial dysfunction, and lipid metabolism
- Explore the utility of TDP-43 biomarkers and preclinical models in the context of target validation and preclinical development of novel drug mechanisms beyond TDP-43
- Discuss the positioning of TDP-43-targeted therapies within a broader ALS treatment landscape to address disease heterogeneity
- Refine preclinical strategies for better target validation and enhanced predictability of therapeutic efficacy in humans.
1:00 pm Workshop D: Establishing Adaptive & Decentralized Models to Accelerate Therapeutic Discovery in ALS
Synopsis
This workshop will explore the transformative potential of adaptive and decentralized clinical trial designs in ALS research. By integrating platform trials, multi-arm designs, and decentralized tools, these approaches aim to enhance trial efficiency, broaden patient access, and improve the trial experience for participants. Discussions will focus on optimizing recruitment, leveraging interim data for trial modifications, and utilizing digital tools to reduce patient burden while ensuring regulatory alignment.
- Evaluate adaptive trial designs, including platform trials and response-adaptive randomization, optimize recruitment, improve data collection, and enhance trial efficiency
- Explore strategies to improve patient engagement, streamline enrollment, and diversify participation while maintaining trial integrity
- Assess decentralized trial methodologies such as remote monitoring, self-reported ALS-FRS-R, and home visits to mitigate patient burden and foster inclusivity
- Examine operational challenges and regulatory requirements for implementing these models effectively in ALS research