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di-siRNA SOD1
- Development stage
- Preclinical
- Lead developer
- Atalanta Therapeutics
- 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
- Intraventricular
01
Overview
di-siRNA SOD1 is a chemically stabilized, divalent small interfering RNA (siRNA) therapeutic designed to silence the expression of the superoxide dismutase 1 (SOD1) gene. The drug utilizes a unique scaffold that links two siRNAs together, enhancing central nervous system (CNS) tissue penetration and prolonging silencing activity. By targeting and degrading SOD1 mRNA via the RNA-induced silencing complex (RISC), it reduces production of toxic mutant SOD1 protein implicated in amyotrophic lateral sclerosis (ALS), particularly familial ALS caused by SOD1 mutations. Preclinical studies demonstrate that intraventricular administration of di-siRNA SOD1 in ALS mouse models significantly extends survival, slows disease progression, and prevents neuropathology compared to antisense oligonucleotide approaches[1][7]. This modality offers an improved strategy for treating dominantly inherited neurological disorders linked to toxic gain-of-function proteins.
- Other names
- divalent siRNA SOD1di-siRNA^SOD1^
02
Targets
SOD (Manganese Superoxide Dismutase)
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