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SPTLC1 antisense oligonucleotide
- Development stage
- Preclinical
- Lead developer
- Leal Therapeutics
- 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
- Subcutaneous, Intrathecal
01
Overview
SPTLC1 antisense oligonucleotides are experimental RNA-based therapeutics being investigated for the treatment of SPTLC1-related hereditary sensory neuropathy type IA (HSN1A) and juvenile amyotrophic lateral sclerosis (ALS). Developed by researchers at University College London and patented through UCL Business PLC, these agents employ allele-specific silencing to selectively target and degrade mutant *SPTLC1* mRNA transcripts. This approach aims to reduce the accumulation of neurotoxic 1-deoxysphingoid bases (1-deoxySBs)—metabolites that cause axonal degeneration—while maintaining the levels of the wild-type Serine Palmitoyl Transferase Long Chain base subunit 1 protein. Preclinical research has demonstrated the efficacy of LNA (locked nucleic acid) gapmer ASOs, including GalNAc-conjugated variants for systemic administration, in lowering toxic sphingolipid levels in animal models.
- Other names
- SPTLC1 ASOSPTLC-1 ASOSPTLC 1 ASOSPTLC1-targeting antisense oligonucleotideSPTLC-1-targeting antisense oligonucleotideSPTLC 1-targeting antisense oligonucleotide
02
Targets
SPTLC1 (Serine palmitoyltransferase long chain base subunit 1)
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