Case study – Gene therapy vectors

SPARTA^® analysis of lentiviral vectors – overcoming variation in vector performance

Gene therapy is one of the most promising directions in the fields of personalised and nano-medicine. Viral vectors, such as lentiviruses, can be used to transfer nucleic acids into target cells. This genetic cargo can then be integrated into the host cell genome to effect repair of a defective or mutated gene.

Due to the cellular production process of the viruses, extensive purification is essential to ensure a safe and effective treatment is obtained. In addition, incomplete packaging of the viral vectors may lead to variations in vector performance and could result in additional immunogenic burden.

These, amongst other related issues, are key examples that SPARTA^® can address by analysing these novel gene therapy formulations. The single particle analysis of vectors can help solve critical issues in both the early R&D stages of new formulation optimisation, as well as for the quality control (QC) of existing formulations.

SPARTA^® was used to analyse more than 1500 single lentiviral vectors and the resulting compositional information was used to extract the relative capsid and RNA cargo contents. This revealed homogeneous loading of 96.5% (blue) of vectors with 3.5% showing significantly lower loading (orange).

Penders J. Stevens M.M. et al. In preparation