The Benefits of Working with mRNA for Therapeutics

In this Biopharm International Peer Exchange roundtable discussion, participants explored the advantages of mRNA therapeutics. Fouad Atouf, PhD, commented on the rapid development and testing capabilities of mRNA, allowing quick responses to new targets. Constanze Blume, PhD, highlighted the broad range of indications mRNA technology can address, showing its versatility in treating various diseases. Kenneth Chien, MD, PhD, pointed out the precise dosing capabilities of mRNA, which allows for a controlled increase in protein expression and quantitative clinical efficacy. He also discussed the potential for mRNA to facilitate "one-and-done" treatments, such as CRISPR-mediated gene editing, where the transient expression of mRNA is beneficial.

Peter Marks, MD, PhD, added that the familiarity and predictability of the mRNA platform, gained through extensive use during the COVID-19 pandemic, are significant advantages. He also noted the potential for mRNA technology to advance genome editing and the importance of continued learning as mRNA is applied in new therapeutic domains.

Discussion Topics

• 0:00:08 - Introductions and background of speakers
• 0:02:42 - Benefits of working with mRNA for therapeutics
• 0:03:35 - Rapid development and reduced risk of genomic integration with mRNA
• 0:04:08 - Dosability and one-and-done potential of mRNA therapies
• 0:05:13 - Familiarity with mRNA platform and versatility for genome editing