Research Highlights
CMT1A Precision Therapeutics
CELLTIUM is dedicated to developing precision therapeutics for Charcot–Marie–Tooth disease (CMT), the most common group of inherited peripheral neuropathies.
The most common CMT subtype, CMT1A, is caused by a duplication of the 17p12 region that includes the PMP22 gene, leading to progressive damage of peripheral myelin sheaths. Despite advances in research, no disease-modifying therapy capable of fundamentally altering the course of CMT1A has been approved to date.
Leveraging precision neurotechnology that directly targets myelin pathology, CELLTIUM aims to develop CMT1A-tailored therapies that go beyond symptomatic relief and truly modify disease progression.
Challenges & Opportunities
Why CMT1A Drug Development Is So Difficult
Although the genetic cause of CMT1A -- a duplication of the 17p12 region containing PMP22 -- is clearly defined, the precise function of PMP22 and its downstream pathogenic pathways are not fully understood. This makes it extremely challenging to apply conventional target-based drug discovery strategies.
To date, agents such as SARM1 inhibitors and HDAC6 inhibitors have primarily targeted neuronal and axonal pathology. These approaches do not directly correct the core pathology of CMT1A, which lies in Schwann cells and peripheral myelin damage. Moreover, many existing candidates have been developed mainly for symptomatic relief (e.g., muscle function or pain control), or have positioned CMT1A merely as a secondary indication for drugs originally designed for other neurological disorders.
CELLTIUM seeks to overcome these limitations by pursuing a new precision therapeutic strategy that directly targets peripheral myelin pathology at the root of CMT1A.
Platform Technology
PRECISION™ HTS Platform
CELLTIUM’s PRECISION™ HTS (High-Throughput Screening) platform is built on disease-relevant models that faithfully reflect CMT1A pathology, including long-term peripheral nerve myelination co-culture systems and CMT1A mouse models.
Key features include:
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Long-term peripheral nerve myelination co-culture:
An in vitro system that enables prolonged observation of peripheral myelin formation. We quantitatively evaluate myelin damage induced by PMP22 overexpression and myelin restoration achieved by candidate compounds.
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In vivo peripheral nerve functional assessment:
In CMT1A mouse models, we assess functional recovery of candidate compounds through myelin morphometry, nerve conduction studies (NCS), and behavioral assays.
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Multiparametric biomarker-based profiling:
We integrate myelin metrics, axonal integrity markers, and safety biomarkers to precisely characterize the mechanism of action, efficacy, and PK/PD properties of our lead candidates.
Through the PRECISION™ HTS platform, CELLTIUM is discovering first-in-class candidates with robust myelin-restorative activity in experimental systems that closely mirror the pathophysiology of CMT1A.
