AGENTS AND METHOD OF PRODUCING VIRAL VECTORS AND VERSIONS OF THEIR USE Russian patent published in 2024 - IPC C12N15/86 A61K39/12 A61K48/00 

FIELD: pharmaceutics.

SUBSTANCE: group of inventions relates to methods of producing and purifying AAV virus particles and use thereof. Method of producing viral vector based on AAV involves culturing adhesive cells; their transfection with plasmid(s) to ensure production of viral vector based on AAV; lysis of adhesive cells with recovery of viral vector based on AAV; acidification and clarification of the obtained cell lysate; purification of the obtained product using cation-exchange chromatography (CEX); filtration using tangential flow filtration (TFF); ultracentrifugation in a buffer with cesium chloride CsCl and collection of viral vectors based on AAV. Method of treating type I spinal muscular atrophy comprises administering a composition comprising an AAV vector to a patient, where the AAV-based vector contains a polynucleotide coding a motor neuron survival protein (SMN).

EFFECT: group of inventions provides obtaining a pharmaceutical product based on AAV, with low content of empty capsids, low content of host cell protein and/or low content of contaminating DNA, while maintaining high activity.

40 cl, 36 dwg, 36 tbl, 22 ex