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CTX-001
- Development stage
- Unknown
- Lead developer
- Vertex
- Modality
- Small Molecules
- Administration
- Intravenous
01
Overview
CTX-001 is a first-in-class small molecule iron mobilizer being developed by **Cajal Therapeutics** (formerly Cajal Neuroscience) to restore iron homeostasis and iron transport. The drug is designed to address functional iron deficiency in **Chronic Kidney Disease (CKD) anemia** by mobilizing iron across cellular membranes, thereby making it available for essential metabolic and cellular processes. As the lead program in Cajal Therapeutics' pipeline, CTX-001 utilizes a novel mechanism of action to restore iron balance, representing a potential shift in the treatment of systemic iron-related disorders. The drug is currently in Phase 1 development.
02
Targets
TF (Transferrin)
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