Creating the Right Environment for mRNA To Thrive

The development of mRNA vaccines is undoubtedly one of the most significant scientific breakthroughs that contributed to bringing the world out of the pandemic.

Now, researchers are capitalizing on the increased investment since the pandemic, to continue their research to develop mRNA therapeutics for cancers and other diseases.

However, advancing the next generation of therapeutics isn’t based on scientific advancement alone. Fully realizing the potential of mRNA therapeutics will require the standardization of processes, manufacturing agility and collaboration between industry, academia and government bodies, all working together to create regulatory pathways for drug developers.

Benefits of mRNA

The mRNA landscape is evolving quickly, with nearly 900 drugs in clinical development across 7 application areas.¹ The majority are in pre-clinical stages, demonstrating the recent increased investment from biopharmaceutical manufacturers.

There is a growing indication and therapy diversity, including vaccine, oncology, protein replacement and ex vivo/in vivo cell applications. Oncology applications include both personalized and medium batch size indications with potential approvals from 2026 onwards.

mRNA vaccines can also be manufactured faster. Compared with traditional vaccine platforms, like live-attenuated or viral-vectored vaccines, mRNA vaccines offer the potential to be completed in as little as five weeks.² Viral vector therapeutics can take from 6 to 36 months.³

The increased speed from discovery to delivery for mRNA vaccines can help reduce costs in process development – another benefit of mRNA.

If you standardize it, will they come….

While mRNA therapeutics have so much potential to transform the way diseases are treated, there are still many considerations and challenges. One of the biggest challenges is adapting processes. Solutions are often optimized for other molecules and distributed processes can lead to bottlenecks.

The new areas of manufacturing and development have not yet been standardized, leading to challenges with operations, personnel, process, quality control, contamination and others.

With mRNA therapeutics being manufactured at smaller scales than cell-based manufacturing, they provide both benefits and challenges to address. Manufacturing at smaller scales can provide considerable space and cost savings, but manufacturers may need to think differently about their facility setup, especially if they are looking at multi-product manufacturing.

However, changing parameters within mRNA manufacturing presents less contamination risks compared to cell-based manufacturing.

Flexibility is key to success

Flexible and resilient solutions that can scale to GMP manufacturing will be one of the key drivers to delivering on the promise of mRNA technology. Building resilient and flexible solutions will allow researchers the ability to scale rapidly if needed.

Currently, scaling is a challenging bottleneck in mRNA manufacturing. Planning for the potential uptake of the therapy, assessing material suitability, sourcing materials early, and considering the fill-finish process during early-stage development will also help build resiliency and flexibility into the process.

Government policy creates a shift

While industry and researchers can develop the tools and solutions needed to advance mRNA vaccines, government policies are critical to ensuring the proper pathways exist to make sure these medicines reach those who need them the most.

Japan is a great example of a country that is adopting policies that help advance and accelerate the development of novel therapeutics.

Realizing that they must prepare for future pandemics, Japan’s government adopted a national vaccine development and production strategy in 2021.⁴ This shift in policy is creating a clear path for mRNA drug developers. Daiichi-Sankyo, a global pharmaceutical company headquartered in Tokyo, opened Japan’s first mRNA vaccine production facility in Kitamoto, which has the potential to produce 20 million doses per year by 2024.⁵

The Japanese government also awarded a 115 million USD grant to Arcalis, a joint venture between Arcturus and Axecelead, to build a mRNA contract development manufacturing organization (CDMO).⁶

Collaboration between governments, industry and academic researchers is critical to advancing next-generation therapeutics such as mRNA vaccines. Cytiva’s Biopharma Resilience Index, a survey of more than 1,250 biopharma executives in 22 countries found that only 46% of respondents believe government tax or trade policies actively encourage domestic/biopharma manufacturing.

Japan is proving that strategic public policy and investment is critical to their ability to respond to future pandemics and meet the demand for next-generation therapeutics.

Japan’s shift in government policy and regulation made it possible for it to be the first country to approve the first self-amplifying mRNA vaccine approved for COVID-19 in adults. This provides valuable learnings for other governments, especially when, as an industry, we look towards our response to future pandemics.

While science is advancing quickly, government policies and regulations must also keep pace with innovation.

About the author:

Scott Ripley is the general manager of the Nucleic Acid Therapeutics business and Precision Nanosystems for Cytiva, leading the global commercial go-to-market strategy. Scott has over twenty years of experience across research and bioprocess businesses including a recent focus on advancing and accelerating the development of mRNA therapeutics.