OUR SCIENCE

Programmable gene silencing to transform advanced therapies

A uniquely powerful approach to gene silencing


Harnessing the power of reprogramming the cell's own gene control systems to develop advanced therapies that respond to specific cellular conditions and/or disease states.



Our cutting edge bioinformatics platform identifies, from millions of options, unique programmable solutions based on re-purposing miRNA expressed under select conditions: context-specific silencing

Minimal gene editing then re-directs miRNA to a new target to give tunable levels of expression without loss of cell function

Targeting endogenous or exogenous genes 

Key advantages of our approach 

  • Programmable, tunable, stable and specific

    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.

  • Ability to target multiple pathways

    Able to target multiple pathways, thus addressing therapeutic opportunites limited by current approaches, and opening new ways of tackling hard to treat disease

  • Compatible with autologous and allogeneic (iPSC & donor derived) product development

    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.

  • Attractive regulatory profile

    In contrast to many other cell engineering approaches, gene modifications used are minimal and do not require stable incorporation of non-human sequences.

  • Universal platform that works with existing gene editing tools

    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.

Programmable

Utilising state of the art in-vitro and in-vivo models, we are able to profile miRNA expression patterns to identify miRNAs specifically expressed in a given cell type and state. These are prioritised for recoding, allowing us to introduce silencing activity only in the selected condition, as exemplified by monocyte/macrophage specific gene silencing.

Tunable

We have the ability to control gene expression with unprecedented precision. Our computational platform recodes endogenous miRNAs enabling a wide dynamic range of gene silencing activity.


Screening of engineered miRNAs through ectopic expression allows identification of preferred constructs prior to full implementation via gene editing. Minimal gene edits in both iPSC and Primary T-cells demonstrate high efficiency mono and bi-allelic silencing.

Stable

Our platform results in highly homogenous gene silencing activity when compared to alternative approaches, facilitating improved consistency in product profile and activity.

Specific

Minimal gene edits, redundancy in miRNA function, control of allelic editing copy number, and physiological expression results in no detectable gain or loss of function off-target effects in engineered iPSC lines.

From concept to product

Find out about how we are using our platform to develop a therapeutics pipeline.

Therapeutics