Drug intelligence / Profile preview

siRNA (PYCR1)

Development stage
Preclinical
Lead developer
Vrije Universiteit Brussel
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
Administration
Intravenous
01

Overview

siRNA (PYCR1) is a small interfering RNA designed to silence the expression of Pyrroline-5-carboxylate reductase 1 (PYCR1), an enzyme critical for the conversion of glutamate to proline. In the context of multiple myeloma (MM), PYCR1 is upregulated in both tumor cells and the bone marrow microenvironment (stroma), particularly under hypoxic conditions. By inhibiting PYCR1, this siRNA aims to disrupt proline metabolism, which is essential for tumor cell proliferation and the structural integrity of the extracellular matrix (ECM). Preclinical studies have demonstrated that PYCR1 knockdown in stromal cells reduces their protective effect on myeloma cells and limits tumor growth when combined with standard-of-care agents like bortezomib.

Other names
PYCR1 siRNAPYCR-1 siRNAPYCR 1 siRNAsiRNA targeting PYCR1
02

Targets

PYCR1/PYCR2 (Pyrroline-5-carboxylate reductases 1/2)

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