TIDMETX
RNS Number : 4488N
e-Therapeutics plc
01 February 2021
e-therapeutics plc
("e-therapeutics" or the "Company")
RNA Therapeutics Platform
Oxford, UK, 01 February 2021 - e-therapeutics plc (AIM: ETX)
today announces the addition of its RNAi platform as a therapeutic
drug modality.
-- Experiments underway to characterise GalNAc-siRNA platform
and corresponding patent application has been filed, with
additional applications to be filed shortly
-- New team established to identify novel hepatocyte-expressed
therapeutic targets, using generated hepatocyte-specific protein
interactome
-- The Company's proprietary GAINs functional genomics
capability will provide genetic validation of new targets
-- Informatics platform highly synergistic and will provide
mechanistic insights into relevant biologic networks
The Company has recently filed a patent application related to
the chemistry of novel GalNAc-conjugated siRNAs (short interfering
RNA), an important element of its RNAi platform that enables
specific hepatocyte targeting for liver gene silencing. In
addition, a number of siRNA constructs have been designed with
potentially beneficial safety and potency profiles and additional
patent applications are expected to be filed shortly. We believe
that the new patent applications, combined with our know-how, will
provide a significant competitive advantage in RNAi. The new
constructs will include features to minimise potential microRNA
off-target effects, thermally destabilising regions of the siRNA
for reduced toxicity and important position-specific stabilisation
chemistries to improve potency. Multiple in vitro and in vivo
experiments are underway to test these constructs with contract
research organisations in Germany and China.
We expect to offer our proprietary platform to potential
business development partners in 2021-2022 and anticipate that
these constructs will demonstrate at least equivalence to
competitors' platforms. In line with our stated strategy of the
capital raise of July 2020, we intend to advance high confidence
candidates to in vivo studies to realise higher value through our
partnerships.
Liver Target Group and Functional Genomics
Alongside our RNAi technology platform and significant in-house
know-how in the field, our ability to investigate novel biology and
drug targets using our computational platform will be a key
differentiator from competitors in the RNAi space. Efforts in this
area include proprietary data resources in hepatocyte biology using
our hepatocyte-specific interactome, which is an important and
recently completed component. By leveraging our cutting-edge
informatics platform alongside our novel RNAi technology platform,
we believe that we can harness the many opportunities that the
liver offers which have not yet been pursued by competitors.
A highly skilled team of scientists and physicians has been
formed to identify novel hepatocyte-expressed targets for complex
indications such as cardiovascular and metabolic diseases. This
team will conduct validation of those targets in appropriate
phenotypic assay systems and animal models and assess genetic
support for the therapeutic targets using our functional genomics
capability. To this end, headcount has increased over the past six
months, from 15 to 25 FTEs at the end of 2020 with recruitment in
all key areas of the Company.
Genome-Associated Interaction Networks (GAINs) for Genetic
Validation
GAINs, a proprietary network biology-based functional genomics
approach that places genes identified by GWAS (Genome Wide
Association Studies) into their network context will play an
important role in our RNAi platform. This approach permits
identification of the processes that are affected by background
variations in DNA and hence impact the risk of developing a
particular disease. It also reveals mechanistic insights that are
not resolvable at the individual SNP (Single Nucleotide
Polymorphism) or gene level and not discoverable from the lists of
genes typically generated by GWAS. GAINs technology enables
actionable insights to inform target- or network-driven drug
discovery to be derived from GWAS data. Typically, GWAS data yields
tens or even hundreds of genetic variants identified as jointly
contributing to disease risk, being of limited value for novel drug
discovery and development.
Chairman & CEO Ali Mortazavi said: "In 2020, the speed and
specificity of RNA-based therapies have been further demonstrated
with RNA-based vaccines against SARS-CoV-2. GalNAc-conjugated siRNA
is amongst the most powerful and validated technologies in the RNA
field. This class of therapeutics has a safety and performance
profile which is now well established with approved drugs and a
large human safety and efficacy precedent.
Our platform has the significant and added advantage of being
able to leverage our expertise in network biology. In an area where
many monogenic and previously validated targets are being
prosecuted by multiple companies, we are confident that we can
harness the potent performance of the GalNAc-siRNA platform to
derive novel biology and therapeutic targets using our
computational approaches, particularly in complex polygenic
disease. Genetic validation is a major de-risking checkpoint in
drug discovery for many biopharmaceutical companies and we believe
our informatic technologies such as GAINs will give us an edge over
competitors in the space.
We believe that e-therapeutics offers a unique partnering
proposition in hepatocyte-associated indications, as well as in
other areas of biology given the disease-agnostic computational
toolkit we have developed over many years. I would like to
congratulate the team on the speed of execution since our July 2020
capital raise and we look forward to the future with great
confidence. In addition, we remain in business development
discussions with potential partners using our informatics
platform."
For further information, please contact:
e-therapeutics plc
Ali Mortazavi, CEO Tel: +44 (0)1993 883 125
www.etherapeutics.co.uk
Numis Securities Limited
Freddie Barnfield/Duncan Monteith (Nominated Adviser)
James Black (Corporate Broking)
Tel: +44 (0) 207 260 1000
www.numis.com
About e-therapeutics plc
e-therapeutics plc is an Oxford, UK-based company with a
powerful computer-based approach to drug discovery, founded on its
industry-leading expertise in network biology to fully capture
disease complexity. The Company combines network science, machine
learning, artificial intelligence, statistics and big data with
expertise in drug discovery and development to transform the search
for new medicines and intervention strategies.
e-therapeutics has developed an in silico laboratory that
enables the rapid screening of millions of compounds and the
identification of small sub-sets that are enriched for highly
active hits. Its proprietary platform also has novel applications
in functional genomics, being able to analyse complex genetic
datasets, provide a deep understanding of pathological mechanisms
and distil actionable insights for the discovery of novel drugs,
biomarkers and diagnostics.
e-therapeutics has deployed and validated its disease-agnostic
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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