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THN1
- Development stage
- Preclinical
- Lead developer
- Thera Neuropharma
- 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
THN1 is a self-delivering RNA interference (sd-rxRNA) therapeutic candidate developed by Thera Neuropharma for the treatment of Amyotrophic Lateral Sclerosis (ALS). The drug utilizes a proprietary sd-rxRNA technology, originally acquired from RXi Pharmaceuticals, which consists of chemically modified asymmetric RNAi compounds that do not require a delivery vehicle for cellular uptake. THN1 is designed to silence the expression of superoxide dismutase 1 (SOD1) and TAR DNA-binding protein 43 (TDP-43), both of which are central to the neurodegenerative processes in ALS. By reducing the levels of these pathogenic proteins, THN1 aims to modify the course of the disease. It is currently in preclinical development.
- Other names
- sd-rxRNA SOD1-TDP-43
02
Targets
SOD (Manganese Superoxide Dismutase)TARDBP (TAR DNA-binding protein 43)
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