Next generation gene control
We utilise the cell’s own microRNA (miRNA) genes, avoiding exogenous siRNA/shRNA transgenes, and harnessing the native regulatory machinery. Silencing activity is redirected to new targets by editing context-specific miRNAs, exploiting widespread redundancy in microRNA networks.
Bringing programmable functionality to achieve superior gene modulation with minimal intervention - programmable, tunable, stable, specific and multiplexable.
We identify miRNAs uniquely expressed in desired cell types, states, or environments using our proprietary miRNA profiling pipeline.
We design recoded microRNAs, utilising miRNAs with desired expression patterns, to target any mRNA, including through allele or mutation specificity. Our platform uses minimal sequence changes to preserve physiological functions and ensure processing fidelity.
We rapidly identify silencing RNAs with the preferred gene silencing activity using our pooled and arrayed screening platforms.
We edit shortlisted sRNAs into the target cell genome using our high efficiency, single step gene editing protocols. Our genotyping technologies ensure we can select the exact cell pools or clones we need for functional characterisation.
Once edited cells have been generated they enter a phenotypic profiling workflow to identify the optimal designs based on the intended application.
Our computational platforms allow us to design bespoke, engineered RNAs, allowing programmable gene silencing, unlocking new RNAi targets and enabling novel applications such as gene activation.
Highly potent new therapies which are programmed to be active only when and where needed: maximising effectiveness while minimising toxicity and off-target impacts on cell fitness.
Able to target multiple pathways, thus addressing therapeutic opportunites limited by current approaches, and opening new ways of tackling hard to treat disease
Our technology has been demonstrated in both iPSC-derived and primary cell contexts, enabling implemention for autologous and allogeneic product development.
For iPSC derived products, editing of non-coding genes is carried out in iPSCs prior to differentiation, allowing efficient gene editing workflows and assessment of off-target profiles, while avoiding limitations associated with autologous products such as cost-of-goods and scalability.
In contrast to many other cell engineering approaches, gene modifications used are minimal and do not require stable incorporation of non-human sequences.
Our technology can be deployed using a broad range of different nucleases, providing freedom to choose which gene editing tools to utilise based on the specific project, application and targeting requirements.
