The next chapter in RNA-based medicine

In the race to combat COVID-19, mRNA vaccines were designed, tested and rolled out in record time to save millions of lives around the world. This was a landmark event that accelerated the development of RNA-based therapeutics, setting them up to revolutionise the way we treat health and disease. 

To take advantage of this momentum and improve the health of more people, we need to improve funding for innovative research and find the right collaborators to scale up production and commercialisation. CPI can help navigate the unique challenges that come with manufacturing RNA-based therapeutics, as well as providing industry-leading training in this area.

Harnessing the potential of RNA in medicine

RNA therapeutics hold immense potential due to several attributes that offer significant advantages over conventional therapies, including:

• Greater specificity: RNA therapeutics can target specific genes or molecules with high precision, minimising side effects.

• Versatility: The ability to address a broad range of diseases, including previously untreatable conditions and those unable to be targeted by conventional drug therapies. Various types of RNA can be used for different purposes, from silencing genes to triggering immune responses. 

• Rapid development: RNA-based therapies can be designed, manufactured and tested relatively quickly compared to traditional drugs.

The various properties of different RNA technologies are leading to a slew of possible new therapies. For example, mRNA vaccines are being designed and tested to fight infections, cancer and immune-based diseases. Additionally, a range of RNA-based therapeutics are being researched to combat rare genetic diseases. This has brought hope to some patients who thought treatments would never be viable. 

While the potential of RNA-based medicine is clear, there are still challenges to overcome before RNA therapy becomes commonplace. These include: 

• Stability: RNA is naturally fragile and needs to be protected from degradation both during the production process and in delivery before it can reach its target. 

• Delivery: Safely and effectively delivering RNA molecules into the target cells remains a complex task. The RNA must be protected from enzymes or the immune system that would destroy it, and toxicity caused by the release of the molecules into the cell must also be avoided. 

• Cost: The development, production and administration of RNA therapeutics can be expensive, making it difficult not to pass costs on to healthcare systems or patients, especially for the treatment of rare diseases.

• Regulatory landscape: As a relatively new field, the regulatory framework for RNA therapeutics is still evolving.

However, these challenges are not insurmountable, and CPI has the facilities and expertise to help overcome them. For example, our end-to-end process workflows streamline manufacturing and reduce costs. Advancements in the chemical modification of RNA and delivery systems such as lipid nanoparticles (LNPs) are enabling safe and effective delivery, while the right compliance certification ensures that we are aligned with regulation. 

Bridging the gap between research and commercialisation

At CPI there is a full suite of flexible and bespoke methods for the development and scaling of RNA-based vaccines and therapeutics from synthesis, purification and encapsulation to downstream processing and analysis. 

These facilities are housed in our RNA Centre of Excellence, which was opened to build on the expertise CPI accumulated as a member of the UK Government’s Vaccine Taskforce during the COVID-19 pandemic. The research and development work is IS9001 certified, whilst GMP-certified manufacturing minimises pharmaceutical manufacturing risks and ensures that products, medicines, and vaccines are safe, of consistent high quality and ready for early-stage clinical trials. 

Our facility is one of the largest RNA vaccine and therapeutics manufacturing capabilities in the UK and the only open-access facility in the UK that can manufacture lipid-nanoparticle encapsulated RNA. The mRNA-LNP platform offers a ​‘plug and play’ solution for companies to accelerate vaccine and therapeutic scale-up. 

To further support the research and development of new LNP formulations, we have announced our Intracellular Drug Delivery Centre. In partnership with the Medicines Discovery Catapult, the University of Strathclyde, the University of Liverpool and Imperial College London, the Centre provides innovators with access to cutting-edge tools, getting drugs into cells that will help unlock the potential of RNA-based medicines. 

As with the partnership involved in this Centre, CPI fosters collaboration with industry, academia, entrepreneurs and SMEs. An important part of that is ensuring workforce development keeps pace with research and technological advances. To that end, the RNA Training Academy was established to help build biomanufacturing skills through RNA and lipid nanoparticle training courses that cover all parts of the processes involved.

The future is here

We are at a critical moment where RNA-based therapeutics are poised to transform medicine. Towards the end of 2023, the first CRISPR-based gene therapy was approved for use, first by the UK and then the US Food and Drug Administration (FDA). In 2024, trial patients at Hammersmith hospital in London were given an experimental mRNA-based cancer therapy.

As research continues to gather pace and more drug candidates are identified, CPI is ready to support their development. From design to delivery, we can help your RNA-based therapeutic reach the patients who need them, improving health and saving lives.