Global coalition backs Imperial College London’s RNA vaccine platform to fight ‘Disease X’

CEPI partners with Imperial College London to develop a self-amplifying RNA vaccine platform. (Getty Images)

One of the first missions that the Coalition for Epidemic Preparedness Innovations (CEPI) set when it came into being in 2017 was to facilitate development of vaccine platforms that can be quickly adopted against unknown pathogens. Now, it is shelling out the first batch of money for that purpose.

In a new deal, the outbreak-focused group is putting down $8.4 million for Imperial College London to work on a self-amplifying RNA (saRNA) vaccine platform that can be used to rapidly develop vaccines against pathogens—even unknown ones. And the British institution will partner with BioNTech in the manufacturing of such vaccines to assess their potential.

Imperial will first develop the platform, called RapidVac, to produce vaccines against H1N1, rabies, and Marburg as proof of concept for the project, a CEPI spokesman told FiercePharma. Once the platform is validated, CEPI hopes it can be used to develop vaccines rapidly against new and unknown pathogens, known as “Disease X.”

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“We cannot predict where or when Disease X will strike, but by developing these kinds of innovative vaccine technologies we can be ready for it,” CEPI CEO Richard Hatchett said in a statement.

Given CEPI’s focus on vaccines against emerging infectious diseases, the organization will only retain rights to the Marburg candidate and other vaccine candidates against WHO R&D Blueprint priority pathogens that are built using the platform, the CEPI spokesman said in an emailed response.

Unlike traditional vaccines that inject the whole antigen in the body to induce an immune response, RNA vaccines incorporate synthetic molecules encoding the antigen genes, which instruct the body to make antigenic protein itself.

RELATED: Pfizer pays BioNTech $120M upfront to form mRNA flu vaccine pact

Because the RNA approach forgoes the cumbersome and time-consuming virus purification, inactivation and culture process of manufacturing traditional vaccines, and DNA sequencing has made much advancement, RNA-based vaccines can be produced much faster—a key advantage in the face of emerging epidemics. In fact, if successful, CEPI hopes Imperial’s RapidVac can produce clinical trial-ready vaccines against Disease X within 16 weeks of identification of an antigen.

Sound familiar? Companies such as the Bill & Melinda Gates Foundation-funded CureVac and high-profile Moderna—which is in the process of the largest-ever biotech IPO—are developing vaccines based on similar vaccine platforms. However, in a potential advantage over other mRNA platforms, Imperial’s saRNA platform also keeps the part of the RNA that allows it to multiply.

Though there’s no conclusion as to which platform is optimal, in a recent study published in the journal Molecular Therapy, an Imperial-BioNTech team argues that compared with synthetic mRNA vaccines, the saRNA technology requires a much lower dose of the vaccine to achieve the same level of protection, potentially saving more time in production. In mice challenged with H1N1 infection, 80 μg mRNA vaccine was needed to offer full survival, while only 0.05 μg saRNA was needed to achieve that, the team reported.

“We believe that synthetic self-amplifying RNA based vaccines offer the best opportunity for a ‘just in time’ response to infectious outbreaks, providing the needed technological shift to aggressively redefine the timelines for vaccine production,” Imperial’s saRNA project leader, Prof. Robin Shattock, said in a statement.

RELATED: Outbreak alliance hands Johnson & Johnson, Oxford $18.7M for MERS work and more

The $8.4 million marks the first investment CEPI has made in vaccine platform technologies that enable rapid vaccine development. Before that, the public-private alliance shelled out millions of dollars to entities such as Johnson & Johnson and the University of Oxford, IDT Biologika, Profectus and Emergent BioSolutions, the International AIDS Vaccine Initiative, Inovio, and Themis to develop vaccines against Lassa fever, MERS and Nipah, three of its priority pathogens chosen from the Who R&D Blueprint.

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