Source - LSE Regulatory
RNS Number : 2014Q
e-Therapeutics plc
26 October 2021

e-therapeutics plc

("e-therapeutics" or the "Company")


RNAi Platform Characterisation Results



Oxford, UK, 26 October 2021 - e-therapeutics plc (AIM: ETX; OTCQX: ETXPF), a specialist in computational drug discovery with a focus on developing RNA interference ("RNAi") therapeutics, today announces the results of the Company's RNAi platform characterisation studies.




-     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


Ali Mortazavi, Chief Executive Officer of e-therapeutics, commented: "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."


The information contained within this announcement is deemed by the Company to constitute inside information as stipulated under the Market Abuse Regulations (EU) No. 596/2014 ('MAR') which has been incorporated into UK law by the European Union (Withdrawal) Act 2018.  Upon the publication of this announcement via Regulatory Information Service ('RIS'), this inside information is now considered to be in the public domain.




e-therapeutics plc

Tel: +44 (0)1993 883 125

Ali Mortazavi, CEO

Karl Keegan, CFO





SP Angel Corporate Finance LLP


Tel: +44(0)20 3470 0470

Nominated Adviser and Broker


Matthew Johnson/Caroline Rowe (Corporate Finance)


Vadim Alexandre/Rob Rees (Corporate Broking)




About e-therapeutics plc

e-therapeutics plc is an Oxford, UK-based company integrating computational power and biology to accelerate the discovery of life-transforming medicines. The Company has developed and validated a powerful, disease and modality agnostic computational approach to drug discovery, leveraging its industry-leading expertise in network biology to fully capture and interrogate human disease complexity. 


The Company's multi-disciplinary team builds computational models of biological functions to transform the search for new medicines, interventions, mechanisms and genetic support. Its biology-led in silico laboratory enables rapid hypothesis generation and phenotypic screening of millions of compounds leading to 100-1000x higher hit rates in the wet lab and successful mode of action elucidation. Novel targets can also be identified, prioritised and assessed. Harnessing internal target gene discoveries, e-therapeutics is currently building an in-house pipeline of RNAi based medicines, using its proprietary GalNAc-siRNA technology. 


e-therapeutics has deployed and validated its disease-agnostic computational drug discovery platform both in house and with partners, including Novo Nordisk, Galapagos NV and a US-based, top 5 pharmaceutical company.


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