Cell and gene therapy
Cell and gene therapy represents a revolutionary leap forward in the development of next-generation drugs. These innovative therapies hold the promise of treating, and even potentially curing, a wide range of diseases by targeting their underlying genetic causes. We are committed to advancing the field of cell and gene therapy through our cutting-edge gene-delivery characterisation platform. Our comprehensive approach helps ensure that we can effectively develop and optimise oligonucleotide-based therapies, addressing critical aspects of their delivery and efficacy.
Our platform provides detailed insights into various crucial parameters, including delivery mechanisms, biodistribution patterns, tissue-targeting and gene-editing efficacy, immune response dynamics and tolerability. Nuvisan offers a suite of specialised services to support the development of nucleic acid therapeutics, including:
- Model and cellular assay generation including CRISPR
- Validation with human induced pluripotent stem cell (iPSC) derived models
- Studies in immunodeficient, immunocompetent and humanised or transgenic mouse models
- Bioluminescence/fluorescence in vivo imaging for real-time monitoring of RNA/DNA delivery
- Gene delivery detection methods, including single-cell sequencing and RNAscope.
Our platform offers comprehensive evaluation of therapeutic uptake and delivery. We quantify fluorophore-labelled RNA/DNA to assess uptake efficiency and utilise fluorescent Gal9 reporter assays for precise assessment of endosomal escape. Additionally, we evaluate GFP expression from delivered RNA using a versatile assay system that can be adapted to various readouts and cell lines or tissues, including iPSC-derived models.
Enhance your assay with cutting-edge readouts such as next-generation sequencing and single-cell sequencing. Our in vivo platform includes robust biodistribution and efficacy assessments, utilising advanced reporter systems and ex vivo analyses. These include bioluminescence imaging, fluorescence imaging and various oncology models, along with single-cell sequencing, target mRNA quantification, protein expression, RNA in situ hybridisation, flow cytometry analysis and immunohistochemistry assessment. We help ensure the safety and effectiveness of your therapeutics through comprehensive tolerability assessments, including cytokine profiling, liver enzyme profiling and complement activation.