RaNA Therapeutics buys out Shire’s mRNA platform

RaNA Therapeutics has acquired Shire’s mRNA therapy platform, originally developed by its MRT employees, who have also now joined RaNA as part of the deal.

This subsidiary will now become part of the upstart, which will continue to push on with work on this platform, specifically on its lead programs in cystic fibrosis (CF) and urea cycle disorders (UCD), which can lead to excess ammonia in the blood, and is also of interest to biotech PhaseRx.

Monetary details were thin on the ground, but for its side, Shire gets an undisclosed equity stake in RaNA and is in line for future bio bucks on any meds made with the tech. 

This comes about 18 months after RaNA saw a $55 million B round as it put together the cash it needed to move a pair of novel treatments toward phase 1 and build out its internal product engine.

The biotech has been at work on drugs designed to elevate the production of certain proteins within the body’s cells by interfering with epigenetic signaling. RaNA has developed targeted therapies that home in on RNA and up-regulate beneficial proteins, a tech the company believes is ideal for diseases tied to protein deficiencies.

With its 2015 fundraise, RaNA focused on two preclinical treatments for the rare genetic disorders spinal muscular atrophy and Friedreich’s ataxia. Each condition is caused by the suppression or breakdown of a key protein, and RaNA sees its in-house technology as being key to reversing the course of disease. It had slated 2017 as the year it would start human testing.

“Shire and the MRT employees joining RaNA have done a tremendous job of building a leading mRNA therapy platform, and we are prepared to accelerate the translation of this powerful platform with the goal of developing breakthrough therapies for people living with life-limiting diseases,” said Ron Renaud, CEO of RaNA.

“This acquisition results in the most comprehensive RNA-based therapeutic approach in the industry and significantly expands RaNA’s ability to correct a wide range of disease genotypes regardless of mutation and access new targets not currently addressable by existing modalities. We are thrilled to welcome our new colleagues and introduce two new programs to our pipeline.”

A few years back, Shire struck up a deal with the Cystic Fibrosis Foundation's nonprofit drug development arm in hopes of developing an mRNA solution for the disease.

The charity committed up to $15 million to support Shire’s work in mRNA, through which it hoped to craft a treatment that can correct the errant CFTR gene at the heart of the disease.

“Shire recognizes the potential of the MRT platform,” added Philip Vickers, head of research and development at Shire.

“We are committed to a smooth transition to RaNA and look forward to the development of therapies with this platform to ultimately treat patients living with debilitating diseases.”

A number of biopharmas are working on this area, including most notably Moderna and one of its partners, Merck (who work together on cancer vaccines), as well as Vertex (which is working with Moderna on an mRNA CF candidate) and CureVac, backed by The Bill & Melinda Gates Foundation and rival to Moderna.