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MTOR-targeting antisense oligonucleotide
- Development stage
- Preclinical
- Lead developer
- SoVarGen
- 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
- Administration
- Intrathecal, Parenteral
01
Overview
MTOR-targeting antisense oligonucleotides are RNA-based therapeutics designed to selectively bind to and degrade MTOR mRNA, thereby reducing the expression of the mechanistic target of rapamycin (mTOR) protein. This approach is being investigated for the treatment of mTORopathies, such as focal cortical dysplasia (FCD) type II, hemimegalencephaly, and tuberous sclerosis complex (TSC), which are characterized by hyperactivation of the mTOR pathway in the brain. By downregulating mTOR expression locally in the brain (e.g., via intrathecal administration), these antisense oligonucleotides aim to suppress spontaneous seizures and ameliorate associated neuropathological symptoms with a potentially improved safety profile compared to systemic mTOR inhibitors like everolimus. A prominent candidate in this class is SVG105, developed by Sovargen in collaboration with Angelini Pharma.
- Other names
- MTOR-targeting antisense oligonucleotide
02
Targets
mTOR (Mammalian target of rapamycin kinase)
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