Characterization of plasmids or nucleic acid constructs is fundamental to the development of gene therapy approaches. We are committed to providing automated and highly sensitive analytical methods to fully measure and characterize nucleic acids and plasmids, which are essential for the discovery and manufacture of gene therapies for rare diseases. Our transferable and reproducible workflows can provide the accuracy you need and save you time.

Background

Gene therapy is an emerging therapeutic modality that treats diseases by manipulating the expression of genetic material. It has significant potential for curing rare diseases that cannot be cured by conventional therapies. The process of gene therapy varies depending on the in vivo and in vitro routes, but regardless of the type of gene therapy approach, the construction of a plasmid vector, i.e., the insertion of the target gene into the plasmid by means of genetic engineering, using principles such as recombination, is the most fundamental and essential step. Therefore, comprehensive RNA, DNA, and plasmid analyses are essential for the successful development of gene therapies.

DNA and RNA Analysis

Current traditional methods for analyzing RNA and DNA samples are complex, time-consuming, and often fall short of requirements, which affects the downstream development of gene therapies. Our company offers better solutions to help you analyze a wide range of nucleic acids with high accuracy and resolution.

• Accurate analysis of double-stranded DNA (dsDNA)
  Analysis of dsDNA is an important part of the manufacturing of nucleic acids for gene therapy. Traditional agarose gel and microchip-based methods have limitations in terms of size accuracy, segment resolution, reproducibility, and long-size coverage. We have established capillary electrophoresis-laser induced fluorescence detection (CE-LIF)-based workflows to help customers accurately analyze dsDNA over a wide size range of 100 bp to 15 kb. 
• Analysis of CRISPR/Cas9-induced insertions and deletions
  With the increased importance of CRISPR/Cas9 in gene therapy, it is necessary to understand the impact on target genes and potential off-target effects to address safety concerns. We offer SWATH, an MS-based strategy to quantify target products and analyze the overall impact of gene editing on protein expression.
• RNA purity analysis
  Analyzing RNA purity and quality is critical to ensure the safety and efficacy of gene therapy compounds based on these molecules. To further address more specific user needs, we have optimized the CE-LIF method for assessing RNA quality to achieve higher resolution and to provide flexibility for further method modifications.

Plasmid Analysis

Our dedicated research team works with you and provides flexible solutions to meet your needs for in-depth characterization of plasmids used for gene therapy.

• Plasmid purity analysisand degradation monitoring
  Plasmid DNA is not only an efficient gene delivery vector in gene therapy but also an invaluable intermediate in the manufacture of AAV and lentiviral vectors. With CE-LIF, we help our customers quickly, sensitively, accurately, and reproducibly quantify plasmid DNA isoforms, including supercoiled, open circular, and linearized plasmid isoforms. We provide our customers with the detection of various plasmid topologies formed upon exposure to biological or chemical stresses to detect plasmid purity, analyze stability, and monitor plasmid degradation.
• Recombinant DNA sequencing
  It is necessary to verify the fidelity of the nucleotide sequences encoding expression products in the production of gene therapy products. With the GeXPTM system, we provide our customers with sequencing services for various recombinant DNA in plasmid vectors to confirm the sequence of DNA samples with high fidelity.

We can provide expert nucleic acid and plasmid purity/integrity analysis at every step of gene therapy development to enhance the likelihood of the successful development of highly effective gene therapies for rare diseases.

Reference

Collins, M.; Thrasher, A. Gene therapy: progress and predictions. Proceedings of the Royal Society B: Biological Sciences, 2015, 282(1821): 20143003.