AAV-BASED VIRAL VECTORS AND PATHS OF APPLICATION THEREOF Russian patent published in 2023 - IPC A61K48/00 C12N15/864 

FIELD: pharmaceuticals.

SUBSTANCE: group of inventions relates to a composition and an application of viral particles, namely, to a composition containing AAV9-based viral vectors, and to a method for application thereof for treating patients with spinal muscular atrophy (SMA). Method for treating spinal muscular atrophy (SMA) in a human patient involves intrathecal administration of an AAV9-based viral vector containing a polynucleotide encoding a survival motor neurone (SMN) protein. Pharmaceutical composition for intrathecal administration for treating spinal muscular atrophy (SMA) contains an AAV9-based viral vector containing modified ITR AAV2, a chicken beta-actin (CB) gene promoter, an immediate early cytomegalovirus (CMV) gene enhancer, a modified late intron 16S SV40, a polynucleotide encoding the survival motor neurone (SMN) protein, bovine growth hormone (BGH) polyadenylation signal, and unmodified ITR AAV2.

EFFECT: improved treatment of SMA, including type II and III SMA, which leads to an easier course of progression of the disease, terminated progression of the disease and/or improvement in the functional development.

41 cl, 13 dwg, 16 tbl, 3 ex