From the fringes to the forefront — the mRNA era has taken hold of pharma

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The genetic technology mRNA has quickly come a long way in a short period of time.

Its story dates back more than 70 years when researchers began assessing its therapeutic potential. Over the past two decades, several mRNA-focused companies were established, but despite its promise and growing interest from large pharma, success was elusive.

All that changed in 2020 when Pfizer-BioNTech and Moderna received authorization for their mRNA-based COVID-19 vaccines. The breakthroughs during the pandemic have not only been integral in preventing more COVID-19 deaths, but have also brought extensive funding to mRNA-focused companies, enabling them to extend research into mRNA vaccines and therapies across the therapeutic spectrum.

In 2021 there were 49 deals signed involving mRNA companies or assets with a combined potential value of $5.37 billion, says Daniel Chancellor, director of thought leadership at Informa Pharma Intelligence. He adds that 2022 is off to the same pace.

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“The volume of deal-making for mRNA is the result of industry waking up to its potential for vaccinating against other infectious diseases, its use in cancer, and also, just more generally, mRNA as a way of producing proteins, adding to the range of different tools that we can use to produce proteins for therapeutic effects,” Chancellor says.

When Moderna received $1 billion in research funding from the US government, it also helped to de-risk mRNA and showed it was a platform worth investing in, according to Dr. Corey Casper, president and CEO at Access to Advanced Health Institute (AAHI).

While several companies — large and small — have been investing in mRNA research for years, the COVID-19 vaccine successes have spawned a swath of new mRNA startups, each seeking to improve upon or copy the platform. Now there are as many as 115 different companies with mRNA assets, Informa’s database shows.

“We have companies out of China, for example, that didn’t exist two years ago that are now well funded and performing such research,” Chancellor says. “We also have bigger companies looking at this space as well, because clearly the technology is validated and has broad potential. Sanofi, for example, entered the space through the acquisition of Translate Bio. CureVac, which has been in mRNA for many years, now has a number of partnerships with larger pharmaceutical companies.”

How is this flurry of mRNA-fueled action shaking out across the industry? Here’s a look at where the industry’s pipeline stands.

The mRNA pipeline

Data from Informa shows the company with the most mRNA drugs in development is Moderna, with 46, followed by BioNTech with 29, then CureVac with 19. Of the large pharma companies, Sanofi has 11 mRNA drugs in its pipeline, Pfizer 10, GSK five and AstraZeneca has four.

Moderna is advancing mRNA vaccines against additional respiratory viruses including COVID-19 booster vaccine candidates and a seasonal flu vaccine candidate (mRNA-1010). The company also recently announced that it is developing a single dose vaccine that combines a booster against COVID-19 with a booster against flu.

Chancellor says the eventual hope is that there will be a winter respiratory virus vaccine that will encompass a range of different flu strains. However, he adds that while mRNA has proven itself to be the superior technology for quickly producing and scaling up a vaccine during a pandemic, it’s not yet proven for diseases that are already endemic.

mRNA-focused companies are developing the technology in many different therapeutic areas. For example, a spokesperson for Moderna says the company’s mRNA medicines are focused on six medical areas — infectious diseases, immuno-oncology, personalized cancer vaccines, cardiovascular disease, rare diseases and autoimmune diseases.

The mRNA pipeline — By the numbers

211 = Drugs in preclinical development

Informa Pharma Intelligence

The company is developing vaccines for different latent viruses, which remain in the body after infection and can lead to life-long medical conditions, including a phase 3 clinical trial for cytomegalovirus (CMV).

BioNTech’s most advanced mRNA pipeline is in oncology, where the company has several phase 2 programs underway, including individualized cancer immunotherapies for melanoma and adjuvant colorectal cancer. The company has 28 vaccines or therapies in research, according to Informa data.

“BioNTech have been doing this for a long time, but they also now have huge financial power to speed up development timelines and run many more and more ambitious clinical trials simultaneously,” Chancellor says.

Among the other mRNA leaders, CureVac has been mRNA research for more than 20 years and, as such, is one of the earliest innovators in the field.

A more recent entrant to mRNA is Sanofi, which initially took a wait-and-see approach to mRNA technology, but has more recently invested heavily in the area, for example, accquiring clinical-stage mRNA therapeutics company Translate Bio in August 2021.

The next big mRNA breakthroughs

Speed ​​and versatility

According to Moderna, an advantage with mRNA technology is its potential to solve problems that are difficult for traditional drug development platforms.

“Speed ​​is one distinct differentiator,” the Moderna spokesperson says. “If we have the sequence of a virus, we can create a vaccine candidate to study in a lab in days rather than months or even years. We can also deliver vaccines against multiple viruses with a single shot. Also, with mRNA therapeutics, we can use mRNA to potentially restore the activity of missing enzymes responsible for various rare diseases with the goal of one day bringing treatment options to patients and their families.”

The benefit of the technology is its versatility and the fact that it can teach the body how to make its own medicine by instructing the body to make proteins.

“In some cases, these proteins can train the immune system to fight disease. In others, they may replace defective or missing proteins that are critical for survival,” the Moderna spokesperson says.

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