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LDHA siRNA
- Development stage
- Unknown
- Lead developer
- Novo Nordisk
- 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, Intravenous
01
Overview
LDHA siRNA refers to small interfering RNA (siRNA) molecules designed to silence the *LDHA* (Lactate Dehydrogenase A) gene. LDHA is a critical enzyme in the glycolytic pathway, responsible for the conversion of pyruvate to lactate. In the liver, it also facilitates the conversion of glyoxylate to oxalate. By utilizing the RNA interference (RNAi) pathway, LDHA siRNA reduces the expression of the LDHA protein. This therapeutic approach is primarily utilized to treat primary hyperoxaluria type 1 (PH1) by reducing hepatic oxalate production, with nedosiran (Rivfloza) being the most prominent clinical example. Additionally, LDHA siRNA is a significant research tool in oncology, where it is studied for its ability to inhibit the Warburg effect (aerobic glycolysis) in various cancers, including lymphoma, pancreatic cancer, and colorectal cancer, where LDHA is frequently overexpressed.
- Other names
- LDHA-targeting siRNAsiLDHA
02
Targets
LDH (L-lactate dehydrogenase A chain)
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