CRISPR meets RNAi: a whole new Targeted Therapy

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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, Intellia, and CRISPR Therapeutics.

Silence Therapeutics Lipoplex technology
Lipid nanoparticles are used to deliver CRISPR to the desired tissue

With the promise of curing any genetic disease, CRISPR technology is causing a furor while the huge investment fuels the hype. However, some major drawbacks seem to have started affecting the finances of leading CRISPR companies.

First of all, some feel that focusing on CRISPR has become rather a gamble than an investment since no clinical results are available to this date. In addition, there’s a fierce patent battle, although this hasn’t stopped Bayer or the Swiss Evolva from acquiring licenses.

Silence Therapeutics still hasn’t chosen a partner to develop the new platform, but the company will undoubtedly have plenty of offers. Its expertise in RNA delivery technology, which already proved effective and safe in Phase I for pancreatic cancer, might give the company a significant advantage over the already established competitors.

Images via Sergey Nivens/; Strumberg D. et al., Int J Clin Pharmacol Ther. 2012 Jan;50(1):76-8.

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