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Silence Therapeutics hails early success of lipid nanoparticle technology in gene editing

Last updated: 12:44 15 Nov 2016 GMT, First published: 08:15 15 Nov 2016 GMT

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The work has shown some early promise.
Silence Therapeutics PLC (LON:SLN) has shown in lab tests that its targeted lipid nanoparticle technology (LNP) can be used in gene editing.
 
It gained expertise in LNP when developing cancer drug candidates that required a special delivery mechanism.
 
It used a liver-targeted lipid in harness with CRISPR/Cas9, the world standard genome editing tool. It said the in vivo study delivered “significant data”.
 
The results, which will be unveiled at the EuroTIDES conference in Berlin, are as follows:
  • 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
Chief executive Ali Mortazavi said: “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."

What the broker says...

The shares were changing hands unchanged at 114.5p, valuing the business at £82mln. The broker Canaccord Genuity reckons the stock is worth 249p.

Analyst Howard Miller said: “This makes Silence one of the few companies to have demonstrated effective CRISPR-/Cas9 gene editing through in vivo delivery, compared with the traditional ex vivo approach.

“The LNPs have been shown to be suitable for effective delivery of large cargos such as RNA CRISPR-Cas9 components, opening up partnering discussions in this field.” 

 

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