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siRNA-KMT2A::AFDN
- 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, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
siRNA-KMT2A::AFDN is an experimental RNA interference (RNAi) therapeutic consisting of a small interfering RNA (siRNA) encapsulated in lipid nanoparticles (LNPs). It is designed to target the *KMT2A::AFDN* (formerly *MLL-AF6*) oncogenic fusion gene, which results from the t(6;11) chromosomal translocation. This fusion is a driver in a subset of acute myeloid leukemia (AML) cases associated with poor prognosis in both pediatric and adult patients. By inducing the degradation of the fusion mRNA, the therapeutic aims to downregulate the fusion protein, leading to the suppression of downstream targets like SHARP1, restoration of cellular differentiation (indicated by decreased CD117 expression), and inhibition of leukemic cell proliferation. The research is primarily conducted by academic institutions including the Princess Máxima Center for Pediatric Oncology and Newcastle University, with various LNP formulations being optimized for bone marrow delivery.
- Other names
- siRNA targeting KMT2A::AFDNsiRNA-KMT2A-AFDNsiRNA-KMT-2A-AFDNsiRNA-KMT 2A-AFDN
02
Targets
KMT2A fusion (Histone-lysine N-methyltransferase 2A (KMT2A) fusion protein)
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