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$850M Deal Kicks Off RNA Vaccine Research

RNA strand illustration

RNA strand illustration (Vossman, Wikimedia Commons, via Flickr)

12 June 2018. Drug maker Sanofi is partnering with biotechnology company Translate Bio to develop vaccines against infectious diseases from messenger RNA, material transcribed from genetic codes in DNA. The deal with Sanofi Pasteur, the vaccines division of Sanofi based in Paris, could bring Translate Bio, in Lexington, Massachusetts, as much $850 million if all aspects of the agreement are exercised.

Translate Bio is a 2 year-old spin-off enterprise from drug maker Shire that develops therapies based on messenger RNA, nucleic acids derived from the genetic codes in DNA, and used by cells to produce the amino acids in proteins for cellular functions. Its technology designs messenger RNA as therapies to correct missing or malfunctioning proteins, which the company says restores functioning gene expression without entering the cell nucleus or changing a recipient’s genome.  Translate Bio says its treatments harness the cells’ own mechanisms to produce working proteins, thus making these therapies able to address targets previously considered undruggable.

The company is currently developing treatments for cystic fibrosis, a genetic disorder causing a build-up of thick mucus in the lungs and other organs, and ornithine transcarbamylase deficiency, a rare inherited disorder that causes toxic ammonia to accumulate in the blood. But Translate Bio says its technology platform can be adapted to address a range of diseases where producing a functioning protein can have the desired effects.

In the case of Sanofi Pasteur, the targets are 5 infectious diseases, although the specific disorders are not disclosed. The companies plan to develop preventive or therapeutic vaccines against the 5 targets, focusing on nucleic acid sequences that produce proteins either preventing pathogens from infecting cells or treating an infection after it begins. Translate Bio says its messenger RNA molecules can be designed to express specific vaccine proteins without making large-scale adjustments in its production process.

The deal calls for Sanofi Pasteur and Translate Bio to collaborate over 3 years on research to develop the 5 messenger RNA, or mRNA, vaccines. Translate Bio is receiving an initial payment of $45 million, and will be eligible for up to $805 million in development and commercialization milestones if Sanofi chooses to license all of the candidates, and exercises options for further vaccines. Sanofi is paying all of the research costs under the agreement, and will receive exclusive rights to commercialize the vaccines. Translate Bio will manufacture vaccines for clinical development, and will be eligible for further payments for those supplies.

A Translate Bio statement quotes John Shiver, vice president for R&D at Sanofi Pasteur, that “We believe mRNA technology has significant potential for rapid and versatile manufacturing, reduced industrialization costs for multiple vaccines, and the improved breadth of immune response for infectious disease vaccines. The Translate Bio platform may allow us to further address medical needs worldwide, including those not readily accessible using conventional vaccine strategies.”

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