CRISPR meets RNAi: a whole new Targeted Therapy

15/11/2016 - 2 minutes

Silence Therapeutics just announced preclinical data for a novel CRISPR therapy targeted to the liver using its RNAi delivery expertise. How does it plan to find a space in this challenging and competitive field?

Based on the UK’s golden triangle, Silence Therapeutics develops RNAi therapies to treat cancer. Now, it will be applying its targeted RNA delivery platform to CRISPR gene editing, the ‘scientific discovery of the century’. The company has just announced positive in vivo preclinical data for this novel system.

In particular, Silence Therapeutics has successfully applied CRISPR to reduce serum levels of two liver proteins: TTR, associated with neurodegenerative diseases, and PCSK9, involved in cholesterol metabolism. The company is now discussing partnerships to develop the new technology.

The novelty of the approach is a targeted delivery system using lipid carriers. In particular, the preclinical data has shown that CRISPR can be directed to the liver. This could result in improved safety and provide an advantage over the three established CRISPR companies Editas,

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