Plasmid-based gene therapies – a small but growing pipeline

Non-viral methods to deliver therapeutic genes in vivo can circumvent the problem of viral vector-targeting immune responses that diminish efficacy, prevent re-dosing and sometimes cause inflammation and liver toxicity. Among the leading approaches to non-viral gene delivery is an old method that’s making a comeback.

Once abandoned for lower transfer efficiency than AAV vectors, plus the potential for efficacy-limiting immunogenicity, plasmid gene delivery technology is resurfacing through use of synthetic DNA constructs that use regulatory elements to improve efficiency and remove pathogenic sequences from the plasmids. ...