Next generation gene control solutions for enhanced gene therapy development
Laverock’s gene silencing technology can be incorporated into both viral and non-viral gene therapy approaches, to address a range of different disease types. Silencing RNAs can be rapidly developed for single or multiplex targeting, with allele / mutation specificity, as part of a gene silencing / suppression only or silence and replace strategy. Our technology is uniquely tunable, and can utilise miRNAs native to the cell type of interest to ensure processing fidelity.
Laverock’s gene silencing technology provides unparalelled control, capturing the full dynamic range of gene expression. When combined with multiplexing and allele specific targeting our approach allows a large number of genetic disease classes to be addressed, with precision and high specificity.
Our computational platform allows us to rapidly design silencing RNAs based on the miRNA profile of the cell type of interest and the target gene sequence(s). Our screening capabilities then progress designs into pooled and arrayed screens identifying sRNAs with the exact silencing potency required and a highly specific profile. sRNAs are validated in single and multiplex contexts giving confidence of performance in the intended gene therapy application.
