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Decades of mRNA research culminated in vaccines that are taming Covid-19. Now scientists and clinicians are exploring the opportunity mRNA presents in therapeutics.

Treating the big ‘C’

The holy grail in health care is curing cancer. Researchers have made remarkable progress; testicular cancer survival rates have increased from 69% to 98% and bowel cancer patients can now survive 10 years1.

But there’s still  work to do, and scientists think mRNA may be one answer. Look at the recent data from a trial to treat notoriously deadly pancreatic cancer2: half the participants remained cancer-free 18 months after treatment.3

“This example shows the potential to cure diseases we haven’t been able to cure before, which can include not only cancer but also autoimmune, neurological, and rare diseases,” explains Linda Mathiasson, Strategic Customer Leader, Nucleic Acid Therapeutics at Cytiva. “It’s an absolutely thrilling time to be in the space, but we have a lot of things to learn and overcome to get there.”

1) Getting the message

Scientists will need to ensure mRNA technology gets to the right place in the body. mRNA therapeutics will require biological engineering — to create an effective mRNA molecule and make sure the targeting and delivery are right.

2) Smaller and smaller

Biopharma has been moving towards smaller manufacturing batches since we started branching out from blockbusters. Manufacturing workflows have gone from producing 2000+ liters for monoclonal antibodies to 25-50 liters of mRNA vaccines for pandemic response. We’ll move to 1 or 2 liters, or less when we’re producing therapies with higher efficacy and/or for smaller patient populations, even for truly personalized medicines. Think downsized manufacturing with many more production lines.

3)  Going cell-free

While cells are standard, all eyes are on a cell-free, synthetic future.

“DNA template creation — the first step in the mRNA manufacturing process — today is mostly done in a bacterial fermentation process, requiring biopharma manufacturers to grow cells,” explains Mathiasson. “We are seeing companies moving toward enzymatic or synthetic DNA, which is quicker and avoids problems with, for example, endotoxins. But it also requires a change to the current manufacturing workflow.”

Being cell-free has benefits: more control, fewer contaminants, and a faster process. And when you consider industry initiatives like the CEPI’s 100 Days Mission4 to make safe and effective vaccines within 100 days, cell-free mRNA processes could be a gamechanger.

4)  Looking beyond the ‘m’ in RNA

Traditional mRNA holds promise and has delivered with vaccines, but other RNA variants have great potential for therapies – circular RNA’s (which are more stable and have potentially longer-lasting impacts) and self-amplifying RNA’s (which are larger but could be distributed in a lower dose). Both could unlock future breakthroughs.

The science of future-proofing

Covid was a masterclass in being prepared to move quickly for future demand. In our next article, we’ll explore how the industry is investing in this new frontier — tackling the business and science hurdles to move therapeutics forward; to realize the pipe dream of personalized medicine, and to transform human health — one big step at a time.

Read more about the potential for mRNA here.