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SVG105

Development stage: Preclinical
Lead developer: SoVarGen
Modality: Spiegelmers → RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, DNA Vaccines → Plasmid DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Vaccine mRNA → mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Aptamers → DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapy Plasmids → Plasmid DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Protein Replacement mRNA → mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Editing mRNA → mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Natural RNA Aptamers → RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, Unmodified DNA Aptamers → DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics, miRNA Mimics → MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Long Non-coding RNA (lncRNA) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, miRNA Inhibitors → MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small 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
Administration: Parenteral, Intracerebral

Overview

SVG105 is a next-generation **antisense oligonucleotide (ASO)** nucleic acid therapy for patients with **refractory epilepsy**, specifically those with **focal cortical dysplasia (FCD)** who do not respond to existing drugs[1][2][3][5][9]. Its mechanism involves directly targeting and modulating the expression of the **MTOR gene (mammalian target of rapamycin)**, aiming to suppress seizures by blocking the underlying genetic cause of the disease[1][2][9]. Unlike symptomatic treatments, SVG105 aims for disease-modifying effects with the potential to be first-in-class for these indications. It is being developed by SoVarGen and has been licensed for global development (excluding Korea, China, and Taiwan) to Angelini Pharma[1][2][5][9]. Routes of administration under investigation involve parenteral/intracerebral injection to bypass the blood-brain barrier[1]. Currently, it is in preclinical (non-clinical) development with expected entry into clinical trials around 2027[1][2][3][5][9].

Targets

mTOR (Mammalian target of rapamycin kinase)

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