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AKR1C3 siRNA
- Development stage
- Preclinical
- Lead developer
- Alnylam 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
- Administration
- Subcutaneous
01
Overview
AKR1C3 siRNA is a small interfering RNA therapeutic candidate developed by Alnylam Pharmaceuticals designed to silence the expression of the AKR1C3 gene (also known as HSD17B5). AKR1C3 is an aldo-ketoreductase enzyme that plays a critical role in the peripheral activation of androgens, converting precursors such as androstenedione and 11-ketoandrostenedione into potent androgens like testosterone and 11-ketotestosterone, respectively. By utilizing an adipose-targeted delivery approach, this siRNA aims to reduce circulating androgen levels without affecting adrenal-derived hormones like DHEA. Preclinical data in cynomolgus monkeys demonstrated significant knockdown of AKR1C3 mRNA and a corresponding reduction in circulating testosterone, suggesting potential utility in treating Polycystic Ovarian Syndrome (PCOS) and other hyperandrogenic conditions.
- Other names
- HSD17B5 siRNAHSD-17B5 siRNAHSD 17B5 siRNA
02
Targets
AKR1C3 (Aldo-keto reductase family 1 member C3)
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