RNAi Platform Characterisation Results
- RNAi platform benchmarking studies successfully completed
- Equivalent performance to leading competitor platforms
- Excellent and comparable safety profile seen in lead designs
- Consistent translation from in vitro to rodent and non-human primates (NHP)
- Additional six new patent applications filed to protect innovation bringing to a total of eleven
On 1 February 2021, the Company announced the commencement of multiple in vitro and in vivo studies to test newly designed small interfering RNA ("siRNA") delivery moieties (GalNAc conjugates) and novel RNA stabilisation chemistries. Three gene targets expressed in hepatocytes (liver cells) were selected for benchmarking and eight different GalNAc-siRNA construct designs were tested per target.
The Company is now pleased to announce that it has successfully established two different GalNAc-siRNA construct designs that have shown at least equivalent performance in terms of potency, target gene silencing and duration of action (up to three months) against the best competitor data in the same targets. Benchmarking studies included one approved RNAi drug and one currently in registration.
To this end, on 11 October 2021 the Company announced that five new patent applications had been filed to protect these novel construct designs and a further six new patent applications have now also been filed. The relevant summary dataset will be presented at the Company's R&D Day in 2022 and headline non-human primate data can be found in the interim results presentation at http://www.rns-pdf.londonstockexchange.com/rns/2014Q_2-2021-10-25.pdf
These results are a crucial step forward for the Company. We set a very high hurdle to benchmark our platform against leading peer platforms and we are now confident that our proprietary GalNAc-siRNA technology is competitive, having shown equivalent performance. In the space of nine months, e-therapeutics has achieved what has taken the industry many years to achieve. The Company is now in a position to execute on its ambition of developing an in-house pipeline combining novel target ideas generated using its computational biology platform, and RNAi as a modality.
Chief Executive Officer