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siMA6
- Development stage
- Preclinical
- Lead developer
- Princess Máxima Center for Pediatric Oncology
- 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
siMA6 is a small interfering RNA (siRNA) designed to target the unique fusion breakpoint sequence of the MLL-AF4 (KMT2A-AFF1) fusion gene, specifically the e9-e4 variant. This fusion gene is a hallmark of high-risk t(4;11) acute lymphoblastic leukemia (ALL), particularly in infants. By silencing the expression of the MLL-AF4 oncogene, siMA6 inhibits the proliferation, clonogenicity, and self-renewal of leukemic cells, and induces apoptosis. To overcome the poor pharmacokinetic and pharmacodynamic properties of naked siRNA, researchers have investigated encapsulating siMA6 in lipid nanoparticles (LNPs), such as sphingomyelin-rich LNPs (LNP-SM), to enhance cellular uptake and therapeutic efficacy.
- Other names
- KMT2A-AFF1 siRNAKMT-2A-AFF1 siRNAKMT 2A-AFF1 siRNAMLL-AF4 siRNAMLL-AF-4 siRNAMLL-AF 4 siRNAsiMA6 siRNAsiMA-6 siRNAsiMA 6 siRNA
02
Targets
KMT2A fusion (Histone-lysine N-methyltransferase 2A (KMT2A) fusion protein)
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