Silence Therapeutics PLC CRISPR/Cas9 Gene Editing Data (1506P)
15 November 2016 - 6:00PM
UK Regulatory
TIDMSLN
RNS Number : 1506P
Silence Therapeutics PLC
15 November 2016
CRISPR/Cas9 Gene Editing Data
A single-particle RNA based approach to in vivo CRISPR/Cas9 gene
editing
London, 15 November 2016 - Silence Therapeutics plc, AIM:SLN
("Silence" or "the Company") a leader in the discovery, delivery
and development of novel RNA therapeutics for the treatment of
serious diseases with unmet medical need, is pleased to announce
significant in vivo CRISPR/Cas9 gene editing data. These data will
be presented at the EuroTIDES 2016 meeting in Berlin, Germany.
Using its proprietary liver targeted lipid nanoparticle (LNP) to
deliver the CRISPR/Cas9 components, Silence has confirmed the
potential of its delivery platform for in vivo gene editing:
-- Sustained down-regulation of two independent secreted liver
gene products in blood serum was observed for over 200 days
-- Serum levels of TTR protein were reduced by approximately 70%
-- In the cross-controlled experimental cohort, serum levels of
PCSK9 protein were reduced by approximately 55%
-- Target levels in the groups treated with a guide RNA (gRNA)
directed to a different gene were comparable to those of untreated
animals
Ali Mortazavi, Chief Executive Officer of Silence Therapeutics,
commented:
"As in RNA interference, efficient payload delivery is critical
in gene editing. We have leveraged our experience in RNA delivery
systems to demonstrate that our LNP can be used for CRISPR/Cas9
applications. The pre-clinical biological activity that we have
observed supports future in vivo clinical applications in the gene
editing space, which was initially thought to be limited to ex vivo
interventions. We have shown that our LNPs are suitable for the
delivery of large cargos such as RNA CRISPR/Cas9 components, and we
are discussing collaborations in this area with potential
partners."
Study details
The present experiment was designed as a preliminary
proof-of-concept for the use of our optimised LNPs to co-deliver
mRNA encoding Cas9 and gRNA targeting either the mouse TTR or PCSK9
genes. In vivo activity through gene-specific DNA disruption in
mouse liver was measured by quantification of corresponding
proteins in serum. Upon completion of the study, further data will
become available.
Notes to Editors
About Silence Therapeutics plc
Our RNA-based technologies harness the body's natural mechanisms
to create therapeutic effects within its cells. These technologies
can selectively silence or replace any gene in the genome,
modulating gene expression up as well as down in a variety of
organs and cell types, in vivo. We have developed proprietary
modifications to improve the robustness of RNA molecules, as well
as advanced chemistries to enhance the effective delivery of
therapeutic RNA molecules to target cells.
Enquiries:
Silence Therapeutics plc Tel: +44 (0)20
Ali Mortazavi, Chief Executive 3457 6900
Officer
David Ellam, Chief Financial Officer
Canaccord Genuity Limited (Nominated Tel: +44 (0)20
Adviser and Joint Broker) 7523 8350
Dr Julian Feneley/Henry Fitzgerald-O'Connor/Emma
Gabriel
Peel Hunt LLP (Joint Broker) Tel: +44 (0)20
James Steel/Oliver Jackson 7418 8900
Media Enquiries: Tel: +44 (0)
FTI Consulting 20 3727 1000
Simon Conway/Brett Pollard/Stephanie
Cuthbert
This information is provided by RNS
The company news service from the London Stock Exchange
END
MSCEAPFLFAFKFFF
(END) Dow Jones Newswires
November 15, 2016 02:00 ET (07:00 GMT)
Silence Therapeutics (LSE:SLN)
Historical Stock Chart
From Apr 2024 to May 2024
Silence Therapeutics (LSE:SLN)
Historical Stock Chart
From May 2023 to May 2024