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BLOC1S1 splice-switching oligonucleotide
- Development stage
- Preclinical
- Lead developer
- Institute of Molecular and Cell Biology
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal, Intracerebroventricular
01
Overview
BLOC1S1 splice-switching oligonucleotide is an antisense oligonucleotide (ASO) therapeutic designed for the treatment of amyotrophic lateral sclerosis (ALS). It functions by targeting the BLOC1S1 (also known as GCN5L1) mRNA to induce splice-switching that leads to nonsense-mediated decay (NMD), thereby depleting the BLOC1S1 protein. BLOC1S1 is a regulator of mitochondrial acetylation that is upregulated in ALS patients; its depletion has been shown to reduce mitochondrial hyper-acetylation, improve mitochondrial respiration, and rescue disease phenotypes in patient-derived motor neurons and animal models. The lead candidate, SSO 2232.401, was developed by researchers at the A*STAR Institute of Molecular and Cell Biology in Singapore.
- Other names
- BLOC1S1 SSOBLOC-1S1 SSOBLOC 1S1 SSOGCN5L1 splice-switching oligonucleotideGCN-5L1 splice-switching oligonucleotideGCN 5L1 splice-switching oligonucleotide
02
Targets
Biogenesis of lysosome-related organelles complex-1 subunit 1 pre-messenger RNA (BLOC1S1 pre-mRNA)
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