Sanofi’s acquisition of its partner Translate Bio is a clear sign that pharmaceutical companies are interested in messenger RNA technologies beyond Covid-19.
Based in Boston, US, Translate Bio develops mRNA therapies for a range of conditions, including respiratory, liver, and infectious diseases. The company’s €2.7B ($3.2B) buyout, announced by Sanofi earlier this month, seems a tacit acknowledgment of the potential of messenger RNA (mRNA).
To respond to the Covid-19 pandemic, Sanofi had partnered with GlaxoSmithKline to develop a vaccine using more traditional recombinant protein technologies, but they were hamstrung late last year by clinical data showing insufficient immune response in older adults. The companies reformulated their jab and launched a phase III trial after a delay, and are now under rolling review by the European Medicines Agency. But even a quick regulatory approval would leave it nearly a year behind rivals like Moderna and Pfizer/BioNTech that used newer platforms based on mRNA.
“It’s a good move, definitely,” for Sanofi given their struggles, says Mathieu Ghadanfar, Business Development Officer and Chief Medical Officer of Belgian firm Ziphius Vaccines. “It’s just confirming the great interest of large pharmas in mRNA technology.” Ziphius raised a €29.3M Series A in May to build out its self-amplifying mRNA pipeline in infectious diseases.
According to Ghadanfar, observers of the space had wondered to what degree pharmaceutical companies were willing to invest in mRNA technologies. In the case of the mRNA Covid-19 vaccines, Pfizer partnered with BioNTech rather than developing an internal pipeline or acquiring a company while Moderna developed its vaccine without a pharmaceutical partner at all.
The Translate Bio deal confirms a broader interest. However, pharma has not traditionally shown big interest in new vaccine technologies. Johnson & Johnson and AstraZeneca developed their Covid-19 vaccines with adenoviral vector technologies. Merck had been a long-term investor in Moderna but sold its shares in December, suggesting a lack of faith in the technology.
“I think when you see the number of biotech companies that are moving into the mRNA vaccine space, the question one asks is, will there be sufficient capacity in the pharma world to bring all of those programs into development?” asks Ghadanfar.
Sanofi partnered with Translate Bio in 2018 to co-develop mRNA vaccines for infectious diseases and expanded the deal last year as the partners worked on mRNA Covid-19 vaccines. Through the acquisition, “we can now integrate their technology and capabilities into our broader R&D organization,” says Nicolas Kressmann, a Sanofi spokesperson. “We can meaningfully accelerate our existing mRNA vaccine programs, by fully focusing all of their resources on strategic programs and streamlining decision making.” Sanofi expects readouts from the phase I/II trial for their lead Covid-19 vaccine candidate this quarter, and from a phase I seasonal influenza mRNA vaccine by the end of the year.
Kressman noted the possibilities beyond infectious disease. Translate Bio’s pipeline includes inhaled therapeutics that deliver mRNA to encode proteins to treat rare lung diseases. Its lead program is in phase I/II testing in patients with cystic fibrosis. And while the company has not previously disclosed an oncology program, the platform also holds potential in areas like cancer vaccines, Kressman told me.
“A number of companies are deploying mRNA technology in rare disease opportunities, or specific disease niches,” says Steven Powell, CEO of another Belgian mRNA company, eTheRNA. “The utility is broad. It will start to emerge as not just a promising tech but a go-to tool in a number of areas.”
A big move into the cancer space is inevitable, Powell added. “Many companies already have franchises in oncology.” Although it also has an infectious disease portfolio, eTherRNA’s lead asset is an intravenous mRNA vaccine that is expected to enter human testing later this year for head and neck cancer. Similarly, BioNTech has five clinical cancer vaccine programs plus three mRNA-based cancer therapies in human testing; Moderna has two cancer vaccines in the clinic as well.
Other European RNA companies are following suit. German biotech CureVac has an mRNA vaccine for Covid-19 with Bayer in phase III testing and two RNA cancer immunotherapies as well.
The technology still faces some hurdles for broad implementation. For one, mRNA vaccines require extremely cold storage, and while the urgency of the pandemic pushed the healthcare sector to adapt for Covid-19 it’s still a limiting factor. Powell said more innovation will be needed in terms of formulation, including lipid nanoparticle delivery systems.
Ghadanfar says that the pandemic created a unique environment that helped validate mRNA technologies. Heavy investment, especially from the US, and regulatory flexibility around Covid-19 approaches helped de-risk the technology, which might change the landscape in Europe. “We’ve started seeing interest in the mRNA platforms, and this is good news,” he says. “The question is whether big companies will invest, and when.”