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SG005

Development stage: Preclinical
Lead developer: Ionis Pharmaceuticals
Modality: Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics

Overview

SG005 is an antisense oligonucleotide (ASO) therapeutic candidate being developed by Shenji Changhua for the treatment of Amyotrophic Lateral Sclerosis (ALS). It is specifically designed to target the FUS (Fused in Sarcoma) gene transcript. In patients with FUS-mutated ALS, the FUS protein mislocalizes and forms toxic aggregates in motor neurons; SG005 aims to reduce the expression of this protein by modulating or degrading the FUS mRNA. This approach is intended to mitigate the neurotoxicity associated with FUS proteinopathy. The drug is currently in preclinical development.

Targets

FUS (Fused in sarcoma)

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