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siRNA (SLC38A1)
- Development stage
- Preclinical
- Lead developer
- Albert Einstein College of Medicine
- 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, Subcutaneous
01
Overview
siRNA (SLC38A1) is a small interfering RNA therapeutic candidate designed to silence the expression of the SLC38A1 gene, which encodes a non-canonical glutamine transporter (also known as SNAT1). Research presented at AACR 2025 indicates that SLC38A1 is selectively overexpressed in long-term hematopoietic stem cells (LT-HSCs) of patients with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML), contributing to poor prognosis. By mediating the degradation of SLC38A1 mRNA, this siRNA reduces glutamine transport and intracellular levels of glutamate and aspartate, thereby inhibiting the proliferation and colony-forming ability of malignant cells. This approach is being investigated as a strategy to target the metabolic reprogramming of MDS stem cells and prevent transformation into AML.
- Other names
- SLC38A1 siRNASLC-38A1 siRNASLC 38A1 siRNA
02
Targets
SAT1 (Spermidine/spermine N1-acetyltransferase 1)
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