Less than two weeks after filing for an IPO, Translate Bio Inc. landed an $805 million research and development deal focused on messenger RNA (mRNA) vaccines for five infectious pathogens.

The Lexington, Mass.-based company signed a three-year collaboration with Paris-based Sanofi SA's vaccines unit Sanofi Pasteur, which gains exclusive worldwide commercialization rights, is covering all research costs and is paying $45 million up front for the vaccines. The agreement includes development, regulatory and sales milestones and option fees if Sanofi chooses additional pathogens. Translate Bio also is eligible for tiered royalty payments and will be responsible for manufacturing under a separate supply deal not yet established.

In addition to validating its platform, the Sanofi deal enables Translate Bio to "apply our mRNA technology beyond the current therapeutic applications that we are pursuing in cystic fibrosis and ornithine transcarbamylase (OTC) deficiency," said CEO Ronald Renaud.

The company has historically targeted diseases caused by protein or gene dysfunction using the mRNA therapeutic (MRT) platform. Delivered to a target cell that recognizes and translates it, the mRNA restores or augments protein function. Potential advantages with mRNA vaccines are its ability to restore gene expression without entering the cell nucleus and its ability to treat undruggable diseases by using the body's cells to produce natural proteins.

"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," said Sanofi's senior vice president of R&D, John Shiver.

Translate Bio's focus on restoring expression of intracellular and transmembrane proteins in patients with genetic diseases led to two clinical-stage candidates targeting the lung and the liver. MRT-5005 entered a phase I/II trial in May for cystic fibrosis (CF), and MRT-5201 is set to begin a phase I/II trial in the first half of 2019 for metabolic liver enzyme disorder OTC deficiency.

MRT-5005 is being developed for CF regardless of the underlying genetic mutation. It is designed to be inhaled via a handheld nebulizer and administered once weekly. The company intends to start the multiple ascending-dose part of the phase I/II trial by the end of this year. In November 2015, MRT-5005 received orphan drug designation from the FDA for CF, which affects 30,000 patients in the U.S.

MRT-5201 is intravenously delivered to the liver to enable hepatocytes to produce normal OTC enzyme, potentially reducing or eliminating the need for low-protein diets or ammonia scavengers. The FDA granted orphan drug designation in March for OTC deficiency, which occurs in one of 56,500 live U.S. births.

Preclinically, Translate Bio researchers have observed targeted mRNA delivery to the eye, central nervous system, lymphatic system and circulatory system. The platform is expected to be flexible and scalable as the mRNA sequence can be varied to fit different needs. The mRNA is manufactured by a cell-free enzyme catalyzed process using structural components identical to natural mRNA. It is then packaged in lipid-based nanoparticle delivery vehicles.

The deal between Translate Bio and Sanofi Pasteur is the fourth largest this year that involves a private biotherapeutic firm, according to Cortellis Deals Intelligence data tracked by BioWorld. It falls behind a $2.8 billion Allogene Therapeutics Inc. cancer immunotherapy pact with Pfizer Inc. in April, a $969 million Lodo Therapeutics Corp. genome mining agreement with Roche Holding AG's Genentech unit in May, and an $830 million Arvinas Inc. small-molecule deal with Pfizer Inc. in January. (See BioWorld, April 4, 2018, May 10, 2018, and Jan. 4, 2018.)

Translate Bio emerged in early 2017 as a relaunch of Rana Therapeutics Inc., of Cambridge, Mass., which raised $20.7 million in a series A round in 2012, and then $55 million in a series B in 2015. Rana had just acquired the MRT platform from Shire Human Genetic Therapies Inc., a subsidiary of Shire plc. (See BioWorld Today, July 23, 2015, and Jan. 5, 2017.)

Earlier this month, Translate Bio filed for a $115 million IPO and plans to list on Nasdaq under the symbol TBIO. The company declined comment on the Sanofi deal due to the pending IPO.

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