Moderna and BioNTech – who are they?

Moderna and BioNTech, two rising stars in biotech, were in the limelight this year with their pioneering mRNA vaccines against COVID-19. Just who are these companies and who is behind them, asks Richard Staines.

A year ago, the names Moderna and BioNTech were known mainly to those who followed biotech and pharma dealings.

But the tragic events of 2020 have meant these companies have become household names as their trailblazing mRNA vaccines became the first to be approved by regulators against the COVID-19 scourge.

Compared with many of the established names in pharma these companies are young upstarts but they have managed to achieve what other big names in the industry have failed to do and harness the power of mRNA to make medicine.

Their technology is based on synthetic messenger RNA – short for ribose nucleic acid – which is a short transcript of a longer DNA code.

As it’s just a messenger molecule, it does not affect the body’s own genetic code when it is injected as a vaccine – but what it does do is instruct cells to code for copies of a certain protein.

In this case that is the “spike” protein seen on the surface of the coronavirus that it uses to invade host cells.

The body produces antibodies against the protein, which neutralise the virus in the event of an infection.

It’s a revolutionary approach that allowed the vaccine to be made within a few days of the SARS-CoV-2 genetic code becoming available – what’s taken months are the rigorous clinical trials that proved the vaccines were safe and effective.

There has been considerable anxiety over whether using such an untried technology would be the best approach against COVID-19.

What the companies have now proved is that the technology can be used to make safe and effective vaccines in record time.

The companies are based on opposite sides of the Atlantic - Moderna was founded in Massachusetts in 2010 under the name ModeRNA Therapeutics by a team of investors two years after BioNTech began operating from a small lab in Mainz, Germany.

But both drew on the mRNA technology developed by Katalin Kariko from the University of Pennsylvania and her collaborator Drew Weissman, an immunologist from Boston University.

Together the pair had managed to find a way to get the human body to accept strands of mRNA without an immune reaction, by tinkering with the chemical make-up of the molecule.

It was a landmark discovery that meant a technology once derided as being a pipe dream could have applications in the real world as a medicinal therapy.

Moderna’s founders were led by Derrick Rossi, a post doctorate fellow from Stanford University who championed the technology to a team from Flagship Ventures, a Massachusetts firm founded and run by Noubar Afeyan.

CEO Stephane Bancel was hired the following year from France’s bioMerieux and Moderna went on to raise more than $2.6 billion in a series of private funding rounds, while fiercely guarding any details about the technology it was developing.

This changed in 2018 when Moderna went public with what was at that time the largest IPO in biotech history.

The launch raised $500m from investors using what is arguably the slickest stock market ticker out there – MRNA.

Its portfolio of products was broad – including a drug being developed with AstraZeneca for cardiovascular diseases, a cancer immunotherapy and a potential Zika vaccine.

While Moderna blazed its investment trail and tried to find ways to make sure its mRNA vaccines did not produce dangerous immune reactions, a small biotech called BioNTech had licensed in the technology from Kariko and Weissman.

Run by husband-and-wife team Ugur Sahin and his wife Ozlem Tureci, BioNTech wanted to use the technology to create individualised cancer therapies.

Matthias Kromayer, a general partner at the German investment fund MIG that helped found BioNTech in 2008, said he first met the pair 15 years ago and was immediately intrigued.

He told pharmaphorum: “They are very good doctors, scientists and entrepreneurs. Whatever they were beginning they did it with the end in mind.”

The company began to strike several deals with big pharma companies to develop cancer drugs and in 2013 hired Kariko, who had spent years working on mRNA at Penn, to oversee the mRNA work as senior vice president.

Fast-forward to the beginning of 2020 and the companies realised that they would have to quickly change their research priorities to vaccine development as the coronavirus pandemic began to emerge in China.

Kromayer said the risks and opportunities to take on the coronavirus vaccine challenge were huge for BioNTech, which only went public on the Nasdaq at the end of 2019.

He said: “It was a risk - if (the vaccine) had failed it would have shed negative light on mRNA tech. But it was a once in a lifetime opportunity.”

But according to Kromayer the BioNTech team were confident that mRNA was the solution to the coronavirus problem, as mRNA sequences can be generated in such a short amount of time.

“You can manufacture mRNA vaccine overnight and still it is stable,” said Kromayer.

The rest is history – Pfizer stepped in and licensed the technology, choosing the most promising of four potential mRNA vaccines from BioNTech.

Both Moderna and Pfizer’s shots aced the quickly convened clinical trials and became the first vaccines to make it to market less than a year after the pandemic began.

Thanks to the innovation there are hopes that peoples’ lives may return to normal as 2021 progresses but according to Kromayer this is just the tip of the iceberg for mRNA therapeutics.

The founders of both Moderna and BioNTech realised early on that this is far more than just a vaccine and could be used to tackle diseases that have proved impossible to tackle with the previous generations of medical technology.

Rossi, who left Moderna in 2014 had initially thought it could be used to reprogramme cells to act like stem cells but realised its potential after a bruising encounter with Robert Langer, the legendary MIT scientist and entrepreneur.

According to Kromayer potential uses include pre-emptive vaccines for diseases such as Parkinson’s as well as to create personalised cancer therapies.

Kromayer concluded: “The message is that mRNA based medicines are not just here to serve as vaccines it is much, much, more. This will revolutionise medicine over the next 10-15 years in areas we do not even imagine.”