Sorry, you need to enable JavaScript to visit this website.

Unlocking the Power of
Our Body’s Protein Factory

By Sachi Fujimori | June 2022

The global pandemic showed the potential of mRNA technology to transform medicine in the coming years.

The versatility of messenger ribonucleic acid, or mRNA, can’t be overemphasized. The same technology that has helped protect people around the globe from COVID-19 may lead to potential treatments for rare diseases and help prevent other infectious diseases.

While Pfizer is currently focused on developing mRNA vaccines to help prevent the flu and shingles and to treat rare diseases, it continues to explore other opportunities where the technology could make a transformational impact on patients, and where its expertise has the potential to accelerate the pace of innovation.

If we look forward and really let our imaginations go and start marrying unmet medical needs to this new tool kit that we've developed over the past couple of years it’s going to be really exciting.

Learn more about how Pfizer is working to harness the power of mRNA to help prevent diseases other than COVID-19 and to potentially create new therapies.

Prevention

Well before the global pandemic, when mRNA was a lesser-known technology, Pfizer and BioNTech had already been collaborating to make a better flu shot. With traditional flu vaccines, scientists must work months in advance to predict which influenza strains will be dominant in the upcoming season. And depending on how well the flu shot matches the season’s prevalent strains, its effectiveness can vary year to year.

But because mRNA technology can be used to produce vaccines more quickly than conventional vaccine manufacturing technologies, scientists expect it can help create a shot that better matches the season’s flu strains and as a result, may offer better protection.

Another key benefit of mRNA technology is its flexibility, which may allow scientists to quickly “edit” vaccines. For viruses like the flu that are constantly changing and require vaccines that need to be updated each year, once a path is established with the FDA or other regulatory authorities, scientists may be able to swap out the mRNA instructions to match with the then-circulating variants.

In addition to the flu shot, Pfizer and BioNTech are collaborating on a project aimed at producing the first mRNA vaccine for shingles, a painful infection caused by the same virus as chicken pox. Nearly one in three adults will experience shingles at least once in their lifetime, with episodes lasting three to five weeks.

The new shingles vaccine development program will leverage learnings from the companies’ COVID-19 vaccine in addition to Pfizer’s research on antigens, substances that trigger an immune response.

Building upon their COVID-19 vaccine partnership, Pfizer and BioNTech are collaborating to develop the first mRNA shingles vaccine.

But for the mRNA flu vaccine, more things need to be considered, says Pirada Suphaphiphat, Vice President of Viral Vaccines at Pfizer. For one, the current flu vaccines are designed to protect against four different flu viruses, so any mRNA vaccine will likely need to also provide protection against those four viruses.

We’re really trying to find the best design that gives you the best immune response and that’s something that needs to be tested empirically.

Therapies

Looking beyond vaccines, mRNA technology may also help patients with rare diseases.

In collaboration with biotech company Beam Therapeutics, Pfizer scientists are developing mRNA technology as a new approach to gene editing, a form of gene therapy.

Gene editing works by delivering “tools” encased in lipid nanoparticles that have the potential to add, remove, or correct faulty genes in patients with rare diseases—providing a potential one-time treatment for these debilitating conditions.

Did_You_Know_Section_mRNA2There are some 7,000 known rare diseases, of which 80% are caused by genetic changes passed from one generation to the next. These include well-known conditions such as cystic fibrosis and hemophilia, and the lesser-known, such as transthyretin amyloid cardiomyopathy(ATTR-CM), a condition that leads to heart failure.

With this next-generation technology, mRNA encodes instructions on how to make the tools that could repair single-gene mutations. The instructions are accompanied by a guide RNA that directs the tools to the precise location that needs editing.

mRNA_Part02_Image01-final_0.jpg
mRNA instructions can be coded with gene-editing proteins, tools that could repair single-gene mutations.

Opening quote
mRNA technology is a good fit for gene editing. We want to make these editing proteins for just a short period of time to modify the genome. And producing the editing enzymes transiently helps to reduce the potential for off-target effects.
Closing quote

Seng Cheng, Vice President and Chief Scientific Officer of the Rare Disease Research Unit at Pfizer.

The success of mRNA vaccines has opened the floodgates for scientific innovation across a vast range of diseases and offers new hope for patients with limited treatment options.

“RNA is really giving us a more versatile toolbox to leverage for different diseases. It has the potential to give us the flexibility of using yet another technology to address diseases that are not amenable to traditional gene therapy approaches,” says Cheng.