Explore the Agenda
8:00 am Check In, Coffee & Light Breakfast
Workshop A
9:00 am Defining Heterogeneous Disease Biology to Unlock Precision Therapies in Myasthenia Gravis, Chronic Inflammatory Demyelinating Polyneuropathy & Multifocal Motor Neuropathy
This workshop is a mechanism-focused deep dive into disease heterogeneity across MG, CIDP, MMN and GBS, bringing together drug developers to interrogate immunologic origins, autoantibody uncertainty and responder variability. By aligning biological nuance with development strategy, attendees will move beyond buzzwords around precision medicine and confront the mechanistic gaps that are currently limiting predictive biomarker validation and subtype stratification. Participants will leave with a clearer framework for integrating disease pathophysiology into patient selection, sequencing decisions and biomarker development, ultimately accelerating precision-driven clinical progress.
Key Questions to be Addressed:
- Dissecting how AChR-positive, MuSK-positive and seronegative MG differ mechanistically and determining how those differences should influence therapeutic positioningInterrogating the uncertain immunologic origins of CIDP and MMN to clarify where immunomodulatory approaches are biologically justified versus potentially misaligned
- Examining why heterogeneity is limiting predictive biomarker development and defining what evidence would be required to validate assays end-to-end for clinical use
- Debating how to biologically stratify patients before Phase III rather than retrospectively explaining nonresponse
- Exploring whether collaborative industry approaches are required to close development gaps in rare, heterogeneous populations
12:00 pm Lunch Break & Networking
Workshop B
1:00 pm Bridging In Vitro & In Vivo Preclinical Gaps to Improve & De- Risk Clinical Translation Across Peripheral Neuropathies & MG
This workshop will critically evaluate the translational limitations of in vitro and in vivo models in MG, CIDP, GBS and MMN. While MG may be comparatively easier to model, CIDP and GBS rely on complicated induction systems that do not reliably reflect human immunologic origin, heterogeneity or anatomical distribution. By directly interrogating how current models oversimplify antibody-driven pathology, fail to capture unknown autoantibody subsets (particularly in CIDP/MMN), and may not represent disease compartments such as spinal involvement, developers can recalibrate go/no-go decisions earlier in the pipeline. Attendees will redefine what constitutes meaningful translational evidence, ensuring that preclinical immune modulation signals correlate with functional muscle improvement and clinically relevant biomarkers before advancing assets into heterogeneous patient populations.
Key Questions to be Addressed:
- Analyzing how current in vivo induction methods fail to accurately reflect human disease mechanisms, particularly in CIDP and GBS
- Comparing situations in which developers bypass animal models entirely and evaluating when that strategy is justified
- Embedding clinically relevant biomarkers into preclinical assays to improve forward and reverse translation
- Defining what constitutes a translatable efficacy signal rather than an isolated model-specific response
- Preventing the premature elimination of candidates due to poor model representativeness rather than flawed human biology