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CALAA-02
- Development stage
- Discontinued
- Lead developer
- Calando Pharmaceuticals
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
CALAA-02 is an investigational, first-in-class nanoparticle-based therapeutic designed for the systemic delivery of small interfering RNA (siRNA). Developed by Calando Pharmaceuticals, it utilizes a four-component delivery system: a linear, cyclodextrin-containing polymer (CDP), a polyethylene glycol (PEG) steric stabilizing agent, and human transferrin (Tf) as a targeting ligand to bind to transferrin receptors (TfR) overexpressed on cancer cells. The therapeutic payload is a synthetic siRNA duplex targeting the M2 subunit of ribonucleotide reductase (RRM2), an enzyme essential for DNA synthesis and repair. Upon binding to TfR and internalization via endocytosis, the siRNA is released into the cytoplasm, where it mediates the cleavage of RRM2 mRNA through the RNA interference (RNAi) pathway, leading to the inhibition of tumor growth. CALAA-02 was the first targeted RNAi therapeutic to enter clinical trials for the treatment of solid tumors.
- Other names
- CALAA02CALAA-02CALAA 02
02
Targets
RRM2TFRC (Transferrin Receptor)
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