The challenges of using RNA as a therapeutic or a gene editing tool

RNA technologies, including mRNA therapeutics, have emerged as a promising drug modality due to their essential biological role in protein expression and their potential versatility in manufacturing. As a result, RNA is in the global spotlight and is being prioritized due to the Covid-19 pandemic.

However, to maintain the current momentum, experts in this field need to come together to identify and address challenges unique to the RNA modality.

BioPhorum has started this by bringing together industry leaders to identify RNA-focused manufacturing challenges and is working towards solutions for improving current processes.

This paper introduces this critical area and highlights the unique RNA challenges, not only limited to the mRNA therapeutics already identified by the cross-industry team, but also to include how these challenges could potentially negatively impact companies and the RNA industry. It sets out what the team hopes to achieve via this collaboration, both in terms of outputs and benefits to the industry.

The team has identified 15 topic areas – ranging from RNA pharmacodynamics and pharmacokinetics to scale-up challenges for RNA DS and DP manufacturing – to highlight how these will negatively impact companies using RNA technologies.

The team is now prioritizing these topics, benchmarking their members’ experience and knowledge, and solving the associated challenges to establish and align best practices. This will enable the promise of RNA to accelerate into a mature and well-established manufacturing and therapeutic platform.

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The challenges of using RNA as a therapeutic or a gene editing tool December 2021.pdf
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