What's the difference between a heart attack and cardiac arrest?
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Silence Therapeutics, a stunning example of cutting-edge British science, has announced positive results from a single dose of its siRNA treatment, known as SLN360. Small interfering RNA (siRNA) is a class of nucleic acid-based drugs able to block gene expression by interaction with a single-stranded molecule of RNA. They successfully targeted high levels of lipoprotein in the liver and significantly lowered it by 98 percent, with 81 percent persisting at 150 days.
Mark Rothera, President and Chief Executive Officer at Silence, said: “These very encouraging results for our wholly-owned SLN360 program marking the second positive study from our proprietary mRNAi GOLD platform, further underscoring its potential to safely and substantially reduce levels of disease-related proteins in liver cells.
“There is currently no specific treatment option approved for high Lp(a), a genetically determined cardiovascular risk factor affecting 20 percent of the world’s population.
“Today’s announcement brings us one step closer to addressing a major unmet need in cardiovascular disease.
“We are engaged in global partnership discussions to ensure we are well positioned to scale up SLN360 development and future commercialization.”
There are currently no approved medicines that selectively lower this genetic risk factor, which is why Silence Therapeutics has spent more than half a decade researching and readying their treatment.
Silence’s proprietary mRNAi GOLD platform can be used to create siRNAs that precisely target and neutralise the disease-associated gene in the liver.
This treatment works by injecting siRNA into the body, which then latches on to the disease-associated gene and destroys it.
The aim is for the injection to be taken between two and four times a year.
The trial also found the treatment was safe and well-tolerated amongst participants.
Silence Therapeutics’ breakthrough represents a substantial opportunity not only to treat heart disease following a heart attack, but also to prevent it in people who are high risk.
Heart disease affects around 20 percent of the global population – that is more than 13 million Britons.
Across the Atlantic, in the US, heart disease claims the lives of more than 600,000 people every year, making it the leading cause of death.
There are some measures that can be taken to reduce your risk of the disease, such as quitting smoking, lowering cholesterol, controlling high blood pressure, maintaining a healthy weight and exercising.
However, there is also a genetic risk factor that affects one in five people worldwide, making it a major public health issue.
Mr Rothera told Express.co.uk he hopes the infrastructure will soon be put in place to screen Britons for high levels of lipoprotein which would allow effective preventative treatment.
Giles Campion, M.D., EVP, Head of R&D and Chief Medical Officer at Silence, said: “These first-in-human data for SLN360, which align with our pre-clinical findings, reinforce our confidence in its potential to substantially lower Lp(a) levels with long-lasting action and address a major unmet need in cardiovascular disease.
“More broadly, siRNA is proving to be a powerful modality for treating genetic conditions, both common and rare, by precisely engaging targets that have previously been considered ‘undruggable’.”
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