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Drug intelligence / Profile preview
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
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