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Atalanta MSH3 di-siRNA
- Development stage
- Preclinical
- Lead developer
- Atalanta Therapeutics
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intracerebroventricular
01
Overview
Atalanta Therapeutics is developing a divalent siRNA (di-siRNA) therapeutic targeting MSH3 for the treatment of Huntington's Disease. MSH3 is a DNA mismatch repair protein that promotes somatic CAG repeat expansion in neurons, a process central to the pathogenesis of Huntington's Disease. The therapeutic utilizes a proprietary branched siRNA architecture designed for broad distribution and sustained gene silencing throughout the central nervous system following intracerebroventricular administration. By knocking down MSH3 mRNA through RNA interference, the drug aims to block the expansion of CAG repeats and slow disease progression. This program is being developed in collaboration with Biogen.
- Other names
- MSH3 programMSH-3 programMSH 3 programMSH3-siRNA (di-siRNA)
02
Targets
MSH3 (DNA mismatch repair protein Msh3)
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