FORMULATIONS FOR TREATING AND PREVENTING AMYOTROPHIC LATERAL SCLEROSIS Russian patent published in 2024 - IPC A61K31/7115 A61K31/7125 A61K31/712 C12N15/113 A61P25/28 

FIELD: biotechnology; medicine.

SUBSTANCE: disclosed are uses of a pharmaceutical composition containing a fixed dose of 100 mg of an antisense oligonucleotide for treating or preventing amyotrophic lateral sclerosis (ALS) associated with a mutation in superoxide dismutase 1 (SOD1) gene, in a human subject. Pharmaceutical composition is a pharmaceutical composition for intrathecal administration. Antisense oligonucleotide nitrogen base sequence consists of SEQ ID NO: 1, wherein each of nucleosides 1–5 and 16–20 is modified 2'-O-methoxyethylribose nucleoside, and each of nucleosides 6–15 represents 2'-deoxynucleoside, wherein internucleoside bonds between nucleosides 2–3, 4–5, 16–17 and 18–19 are phosphodiester bonds, and internucleoside bonds between nucleosides 1–2, 3–4, 5–6, 6–7, 7–8, 8–9, 9–10, 10–11, 11–12, 12–13, 13–14, 14–15, 15–16, 17–18 and 19–20 are phosphorothioate bonds, and each cytosine is 5-methylcytosine.

EFFECT: inventions provide effective treatment of ALS with rapidly progressing subtypes of mutations without serious adverse events.

15 cl, 8 dwg, 5 ex