PIPELINE
Despite significant investment, gene therapy is still in its early stages of development, requiring safer and titratable therapeutics.
Expression & Control
Current gene therapy modalities exhibit variable expression profiles, with viral vectors’ “one and done” dosing and RNA treatments’ transient expression profiles requiring frequent dosing. The applicability of rAAV-based gene therapies is further limited to small viral capsid capacity.
msDNA can accommodate large genetic payloads, exhibits durable and controllable expression profiles, and requires fewer doses per year
Immunogenicity & Safety
AAV and LVV gene therapies are associated with immunogenicity and safety risks on account of their viral origins.
Conventional DNA vectors bring immunogenicity risks due to unnecessary backbone sequences, which may also negatively impact the quality of final drug products. Conventional and novel circular DNA vectors pose an insertional mutagenesis risk due to their circular topology.
Other backbone-free DNA minivectors, manufactured using enzymatic reactions, are produced at lower fidelity in comparison to msDNA.
msDNA is free of immunogenic backbone sequences and exhibits higher sequence fidelity due to its in vivo production system
