Translating Mechanistic Insights into Precision Therapeutics
CELLTIUM translates deep neurobiological insights into precision therapies for CMT1A and related inherited peripheral neuropathies. Powered by our proprietary PRECISION™ platform, we are advancing a pipeline of targeted drug candidates designed to restore nerve function and move beyond symptom management.
Precision CMT1A Pipeline
CMT1A, the most common subtype of Charcot-Marie-Tooth disease, is caused by a 1.5-Mb duplication on chromosome 17p11.2 encompassing the PMP22 gene. This genetic alteration drives Schwann cell and myelin dysfunction, leading to progressive peripheral nerve impairment and motor and sensory deficits.
Using disease-relevant discovery models, we identify compounds that restore myelination and nerve function by addressing core features of CMT1A biology. This approach has generated a growing pipeline of targeted candidates with distinct mechanisms of action and first-in-class potential. Our lead program, CTM26, has demonstrated compelling preclinical efficacy in severe CMT1A mouse models, while follow-on programs with distinct mechanisms and non-overlapping chemistry continue to expand the pipeline.
PRECISION™ Neuro-Phenomic Discovery Platform
PRECISION™ is our proprietary neuro-phenomic discovery engine integrating peripheral nerve models, high-content phenotypic screening, and AI-driven multimodal analytics to accelerate target identification, candidate prioritization, and lead optimization.
By directly linking disease biology to phenotypic outcomes, PRECISION™ enables the discovery of differentiated therapeutic opportunities for CMT1A and related neuropathies. The platform has already generated multiple promising programs and continues to expand our precision pipeline.
Advancing Toward the Clinic
Our lead CMT1A program, CTM26, is advancing through IND-enabling studies to support future regulatory submissions and clinical development.