Plasmids for improved AAV titers and empty full ratios

By employing a rep plasmid with specific rep-binding elements, the production of recombinant adeno-associated viral vectors is enhanced, addressing the challenges of low titers and high empty capsid ratios, and improving the efficiency and cost-effectiveness of rAAV vector production for gene therapy.

WO2025087987A1 Publication Date: 2025-05-01SARTORIUS STEDIM CELLCA GMBH
8 Cites 0 Cited by

Patent Information

Application Number
PCT/EP2024/079978
Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
Priority Date
2024-06-28
Filing Date
2024-10-23
Publication Date
2025-05-01

AI Technical Summary

Technical Problem

Current methods for producing recombinant adeno-associated viral (rAAV) vectors face challenges in achieving high titers and full particle ratios, leading to inefficiencies in gene therapy applications due to high levels of empty capsids and impurities.

Method used

The use of a rep plasmid containing an adeno-associated virus replication protein coding sequence, a poly adenylation signal sequence, and a minimal element with recombinant rep-binding elements downstream of the poly adenylation signal sequence, which enhances rAAV vector production by increasing the concentration of viral genomes and full capsids.

Benefits of technology

This approach results in improved rAAV vector production, reducing the number of empty capsids and increasing the transduction efficiency, thereby making the process more cost-effective and suitable for gene therapy applications.

✦ Generated by Eureka AI based on patent content.
Patent Text Reader

Abstract

The invention relates to a rep plasmid comprising at least one adeno-associated virus replication protein coding sequence encoding at least one functional rep protein, at least one polyadenylation signal sequence downstream the adeno-associated virus replication protein coding sequence, and a minimal element comprising one or more rep-binding elements downstream the polyadenylation signal sequence. In another aspect, the invention provides a transgene plasmid comprising a CMVie promotor, a transgene, SV40 PolyA, a 5' and a 3' inverted terminal repeats derived from J01901.1 AAV2; wherein the transgene plasmid is selected from one of the following: a conventional single-stranded genome recombinant adeno-associated virus, or a self-complementary genome recombinant adeno-associated virus.
Need to check novelty before this filing date? Find Prior Art