Messenger RNA (mRNA) is a family of RNA molecules that transport genetic information from DNA to the ribosome, where it specifies the amino acid sequence that creates proteins. In theory, by coding your own mRNA, it should be possible to insert it into the cells and turn them into protein factories churning out whatever drug or molecule you program it to.
Although Moderna gets the lion’s share of attention in the mRNA market, it’s not the only company working to break away with this potentially disruptive technology.
Messenger RNA (mRNA) is a family of RNA molecules that transport genetic information from DNA to the ribosome, where it specifies the amino acid sequence that creates proteins. In theory, by coding your own mRNA, it should be possible to insert it into the cells and turn them into protein factories churning out whatever drug or molecule you program it to.
Moderna is the most prominent player in the field although it has yet to get a product to market. In December, the company went public, raising $604 million with its IPO. The company’s value is tagged at around $7.5 billion.
The company has a development pipeline of 21 programs. Ten are in the clinic and another three have open Investigational New Drug (INDs) submissions. Nine of those in the clinic are in Phase I and one is in Phase II, according to a July 2018 update.
But Moderna is not alone.
In August 2018, Pfizer entered a collaboration deal with Mainz, Germany-based BioNTech to develop mRNA-based vaccines to prevent influenza (flu). The two companies will jointly conduct research and development to advance the vaccines. Pfizer paid BioNTech $120 million upfront along with equity and near-term research payments. Another $305 million in milestones is up for grabs and if the vaccines make it to market, BioNTech will receive up to double-digit tiered royalties.
Earlier this month, Paris-based Sanofi extended a deal with BioNTech to co-develop and co-commercialize a cancer vaccine based on mRNA. And this deal follows a July deal Sanofi made with Lexington, Mass.-based Translate Bio to develop an mRNA vaccine for up to five infectious diseases. Sanofi Pasteur paid Translate Bio an upfront payment of $45 million. Translate Bio is eligible for up to $805 million in payments, including a $45 million upfront payment and various milestone payments and royalties.
On January 3, Translate Bio announced that its Phase I/II clinical trial of MRT5005, an mRNA treatment for cystic fibrosis, was ongoing. It also announced that its MRT5201, an mRNA candidate for patients with OTC deficiency, a common urea cycle disorder, was advancing, with the trial expected to begin in the first half of this year. However, on January 22, the company indicated the U.S. Food and Drug Administration (FDA) had requested more information about the IND submission for MRT5201.
Another company making waves in the mRNA market is CureVac, based in Tubingen, Germany and Boston. On October 23, 2018, the company announced it had initiated its Phase I dose-escalation trial of its mRNA-based rabies vaccine, CV7202. CV7202 is a “prophylactic mRNA-based vaccine encoding the rabies virus glycoprotein, RABV-G, formulated with the next generation LNP.”
LNP stands for Lipid Nanoparticle, which is a type of delivery system for mRNA.
On Jan. 8, 2019, the U.S. Patent and Trademark Office (USPTO) granted CureVac a fundamental patent for the use of MRNA for the Respiratory Syncytial Virus (RSV) F-protein to be used to vaccinate infants.
CureVac also has deals with Sanofi, Eli Lilly, and Boehringer Ingelheim.
In theory, mRNA could be used to treat any disease. However, it’s not all that simple. The major challenge is getting the mRNA to the right cells in order to express the protein. As a result, companies have tended to focus on what is often deemed “low-hanging fruit,” such as vaccines and cancer immunotherapies. That’s the case of CureVac, BioNTech and Moderna, but Translate Bio is focusing on a significantly more difficult challenge of cystic fibrosis.
