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PYCR1 inhibitor (siRNA)
- Development stage
- Preclinical
- 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
01
Overview
PYCR1 inhibitor (siRNA) refers to small interfering RNA (siRNA) molecules designed to target and knock down the expression of pyrroline-5-carboxylate reductase 1 (PYCR1). PYCR1 is a key mitochondrial enzyme that catalyzes the final, rate-limiting step in the biosynthesis of proline from glutamine. In various cancers, including multiple myeloma, breast cancer, and renal cell carcinoma, PYCR1 is frequently overexpressed and plays a critical role in supporting tumor cell survival, proliferation, and resistance to chemotherapeutic agents (such as bortezomib or docetaxel) under hypoxic conditions. Knockdown of PYCR1 using siRNA in vitro and in preclinical models impairs proline synthesis, disrupts mitochondrial function, reduces the protective effects of the tumor microenvironment (such as bone marrow stromal cells), and enhances sensitivity to therapies like bortezomib.
- Other names
- PYCR1 siRNAPYCR-1 siRNAPYCR 1 siRNAsiPYCR1siPYCR-1siPYCR 1
02
Targets
AGO2 (Argonaute RISC catalytic component 2)PYCR1/PYCR2 (Pyrroline-5-carboxylate reductases 1/2)
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