It is in the Genes

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It is in the Genes
N4 Pharma and Nuvec Particles
Why access to our facilities, intellect and expertise was instrumental in developing a new drug delivery system.

Whether you are responding to a pandemic, or fighting an endemic disease, having multiple versions or types of vaccines that can respond quickly to natural mutations is key.

That is why complex medicines, such as mRNA vaccines, have played such a huge role in the fight against COVID-19.

mRNA vaccines rely heavily on Lipid Nanoparticles (LNPs)
It is a nanotechnology that took decades to refine, and acts as the crucial delivery system that enables mRNA molecules to access target cells. LNPs are currently the most advanced drug delivery vehicle in the clinic. But they also have drawbacks such as possible toxicity and sub-optimal cellular penetration. Or viral vectors, which can cause side effects.
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N4 Pharma
N4 Pharma is a speciality pharmaceutical company that is developing a patented silica nanoparticle, Nuvec®.
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Nuvec is a unique, non-viral adjuvant delivery system for vaccines and cancer treatments.
It is an alternative approach to traditional vaccine delivery that has the potential to revolutionise vaccines and cancer treatment.
Why? Because it promises high-efficacy with low-cost manufacturing – and has the capacity for rapid development and safe administration. There is just one problem...
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As a virtual organisation, N4 Pharma has no laboratory facilities of its own.
So they have no access to the scarce technologies needed to gain an understanding of Nuvec’s mechanism of action at the cellular level. Which is why we offered them our expertise.
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The type of cargo modules that could be loaded onto the nanoparticles (DNA, RNA, siRNA, small molecules)

2

The essential properties of the particles (size, charge, loading capacity)

3

The cell mode of action after uptake

4

The optimum particle/drug ratio for maximum efficacy

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The efficacy of in vivo administration for vaccine applications

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The biodistribution in vivo

Medicines Discovery Catapult conducted pre-clinical imaging studies using Nuvec particles labelled with a fluorescent dye.

We found that the Nuvec particle enters the cell by a known endocytotic pathway and releases the payload plasmid, resulting in effective transfection.

Using this labelled article, it was also possible to estimate the number of particles that enter the cell.

An important statistic that now paves the way for estimates of the number of plasmids required to effect transfection.

Investigation of Nuvec Particles for Effective Gene Delivery
Thanks to our facilities and advanced technology, we were also able to estimate the depth of penetration of nanoparticles within the cell. So N4 Pharma now have a complete understanding of Nuvec’s mechanism of action

With the support of Medicines Discovery Catapult, N4 Pharma has the data and guidance it needs to accelerate the rational design of Nuvec for efficient gene delivery applications.

And looking to the future, silica nanoparticles like Nuvec could deliver even more benefits, including:

Vaccines that are easy to store, transport, and use

Scalable production that can respond to pandemics in an efficient and cost-effective way

Delivery systems that optimise the safe release of nucleic acids into target cells

So the potential for fighting pandemics and endemic diseases looks brighter than ever.

Dr David Templeton

Technical Director

N4 Pharma

“Working with Medicines Discovery Catapult over the past year has allowed N4Pharma access to technology that has provided an understanding, at the cellular level, of the mechanism of action of Nuvec, which has been instrumental in progressing its development. Importantly, as well as providing access to high-end technology, working with MDC has also provided expertise and intellectual input. It has also enabled access to a wider network of companies providing specialist services.”

Visit the Website
To find out more, about Medicines Discovery Catapult and the impact we could have on your next drug discovery project, explore our website.
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