Research Highlights
Pioneering Precision Therapeutics for CMT1A
CELLTIUM is dedicated to developing precision therapeutics for Charcot–Marie–Tooth disease (CMT), the most common inherited peripheral neuropathy.
CMT1A, caused by duplication of the PMP22 gene (17p12), leads to progressive demyelination and axonal loss. Despite substantial unmet medical need, there are currently no approved disease-modifying therapies (DMTs) capable of altering the disease course.
Leveraging proprietary precision neurotechnology, CELLTIUM is developing CMT1A-tailored interventions that directly target myelin pathology. Our goal is to move beyond symptomatic relief and fundamentally modify disease progression.
Challenges & Opportunities
Overcoming the Barriers of CMT1A Drug Discovery
Although the genetic driver of CMT1A (PMP22 duplication) is known, the underlying pathogenic mechanisms remain complex, limiting the success of conventional target-based discovery.
Current therapeutic strategies primarily focus on neuronal or axonal preservation. While these approaches offer neuroprotective benefits, they may not fully address the root cause: Schwann cell dysfunction and myelin damage. Furthermore, reliance on repurposed drugs often leaves critical unmet medical needs for CMT1A patients.
CELLTIUM addresses these limitations with a precision therapeutic strategy that directly targets peripheral myelinopathy, treating the disease at its core.
Platform Technology
The PRECISION™ High-Content Screening (HCS) Platform
CELLTIUM’s PRECISION™ HCS platform integrates proprietary disease models with machine learning and automation. It faithfully recapitulates CMT1A peripheral nerve pathology, bridging the gap between bench and bedside.
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Proprietary Co-Culture System: An advanced in vitro system that enables quantitative assessment of myelin damage and restoration in a stable, long-term culture environment.
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Rigorous In Vivo Validation: Candidates undergo comprehensive testing in CMT1A transgenic models, including myelin morphometry, nerve conduction studies (NCS), and behavioral assays.
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Multi-Parametric Profiling: Efficacy data and safety biomarkers are integrated to define mechanism of action (MoA) and PK/PD profiles, ensuring high clinical translatability.
Through this platform, CELLTIUM identifies first-in-class small molecules and antibody–drug conjugates (ADCs) with robust myelin-restorative activity.
