ministring DNA (msDNAᵀᴹ) Platform

msDNA carries a highly versatile expression cassette that can be tailored to accommodate promoters, genes of interest and regulatory sequences

Plasmids, along with other DNA vectors, such as minicircles and linear derivatives, play a central role as starting materials, intermediates, drug substances, and drug products in the manufacturing of various therapeutics. These applications encompass DNA and mRNA vaccines, gene-editing templates, and gene therapies employing both viral and non-viral vectors.

 

msDNAᵀᴹ Features

  • msDNAᵀᴹ is a highly adaptable platform designed to have a high degree of customizability, accommodate large mono- or poly-cistronic genetic payloads, and be effective across a range of applications and tissue targets and with a size up to 20-25 kb.

  • msDNAᵀᴹ has demonstrated prolonged and durable gene expression even after single systemic injection, when compared to conventional plasmid vectors. msDNA’s proprietary Super Sequences (SSeq) enhance cellular trafficking & nuclear uptake​

  • msDNAᵀᴹ has a strong safety profile, due to the absence of immunogenic pathogenic sequences, linear conformation and proprietary sequence design. msDNAᵀᴹ has demonstrated increased tolerability and redosability; it also features an in-built safety switch that prevents potentially oncogenic chromosomal integration.

 
 

Transgene expression from the msDNATM vector in vivo can be sustained and/or increased by re-dosable administration.

When redosability & efficacy of msDNATM were evaluated in a dose-dependent study for 16 weeks after multiple injections (3x):

  • msDNATM showed higher and more consistent Luciferase expression compared to the plasmid DNA control
  • msDNATM showed smooth and incremental reaction to injections
  • msDNATM might provide control over dose-response

msDNAᵀᴹ permits systemic redosability and has durable gene expression after multiple dosing​

 

Sustained transgene expression in vivo of reporter gene test article: Luciferase expression in BalbC mice after intravenous administration of 2.0 mg/kg of a msDNATM vector into the tail vein at a first time point (day 0) and re-dosed with administration of a 3.0 mg/kg msDNATM vector at a second time point of 7 days and a third dose of 3.0 mg/kg msDNATM on 21 days. Six arm study (n=5 per arm): 1. msDNATM-LNP, 2. parental plasmid-LNP, 3. naked msDNATM, 4. naked Parental plasmid, 5. control #1: empty LNP, 6. control #2: PBS