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Business & Regulation Trends & Forecasts, Small Molecules

mRNA Trend Setters

What we asked: “Looking ahead to the next 5–10 years, what will be the key disruptors and/or what can be improved upon in the pharma industry?”

Response from: Mahesh Karande, President & CEO, Omega Therapeutics

“One of the enduring rate-limiting factors in R&D is the identification of new biological targets and novel modalities to effectively drug them. In recent years, mRNA-based medicines have risen to the forefront of the industry, offering the ability to prospectively design and engineer therapeutics that work within the natural cellular processes to control physiology with exquisite precision. In parallel, we’ve developed a deeper understanding of how gene expression is regulated by epigenetic marks. Combining an mRNA-based approach with the science of epigenetics yields a tremendous opportunity to create the next leading therapeutic class and transform drug discovery efforts and the pharma industry as we know it.

“Programmable epigenomic mRNA-based therapeutics enable specific, tunable, and durable modulation of gene expression without altering DNA sequences. This unprecedented level of precision allows for more effective and targeted treatments, reducing potential side effects and increasing therapeutic efficacy. Moreover, these therapeutics are adaptable to various delivery methods, enhancing their versatility and applicability across different therapeutic areas.

“A notable advantage of this technology is its potential to regulate the expression of nearly any gene, including those previously deemed undruggable by other modalities. This capability vastly expands the list of available targets and opens exciting avenues for treating a wide range of genetic disorders, cancers, and chronic diseases that have long eluded effective treatment.”

Response from: Alexander Zehnder, CEO, CureVac

“A key disruptor driving the industry over the past ten years has been mRNA technology, which was validated and accelerated by the COVID-19 pandemic. This groundbreaking technology enabled the rapid development and deployment of effective vaccines at the height of a global crisis, highlighting its immense potential in infectious diseases and beyond.

“In the next decade, mRNA technology is poised to revolutionize many areas of medicine. Its flexibility and efficiency in addressing multiple biological targets at the same time opens doors to novel treatments for a wide range of conditions, including cancer, genetic disorders, and rare diseases. The ability to personalize therapies by quickly designing and producing mRNA sequences tailored to specific medical needs could drive personalized medicine, offering patients more targeted and effective therapies.

Moreover, advancements in delivery systems and mRNA stability are expected to further enhance the efficacy and applicability of mRNA-based treatments. As research continues, we anticipate a surge in innovative applications, transforming not just the pharmaceutical industry but also the broader healthcare landscape.

“In summary, the validation of mRNA technology during the COVID-19 pandemic marks just the beginning of its transformative potential, setting the stage for significant advancements and new frontiers in medical science over the next decade.”

Read over 100 other views on the future of the pharma industry on our special web page.

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