Shire Sells RNA Therapy Program to RaNA Therapeutics for Stake in Company

Shire Sells RNA Therapy Program to RaNA Therapeutics for Stake in Company January 4, 2017
By Alex Keown, BioSpace.com Breaking News Staff

CAMBRIDGE, Mass. – RaNA Therapeutics bolstered its mRNA research after acquiring Shire ’s MRT program, which includes 12 employees and two mRNA therapy platform.

RaNA will use the new platform to advance research into treatments for cystic fibrosis and urea cycle disorders. The company hopes to advance the mRNA research on these two areas into the clinic by 2018. The deal will shift about 12 Shire employees to RaNA, along with the two mRNA programs. The new employees will send RaNA’s headcount to about 60 employees, according to reports.

The deal gives Shire PLC an equity stake in RaNA, as well as future milestone and royalty payments on products developed with the technology, the company said in an announcement. How much equity Shire is gaining in RaNA was not revealed.

Ron Renaud, RaNA’s chief executive officer, said the company is prepared to “accelerate the translation of this powerful platform” with the goal of developing breakthrough therapies.

“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,” Renaud said in a statement.

Renaud told the Boston Business Journal that his company has been pursuing Shire’s MRT platform for some time.

In addition to cystic fibrosis and urea cycle disorders, Renaud told the Boston Business Journal that the company will focus the mRNA research on “as many genetic diseases as possible.”

RaNA is currently investigating RNA therapy for the treatment of spinal muscular atrophy (SMA). The company’s technology selectively activates gene expression within the body’s own cells to treat SMA.

On its website RaNA said the company takes a multi-dimensional approach to developing RNA-based medicines. “Using our deep expertise in genetics, RNA biology and biochemistry, and cellular and molecular biology, we assess disease indications from every angle and select the approach within our diverse platform best suited to selectively target and treat their underlying causes at the genetic (DNA), RNA or protein level,” the company said.

In 2015, RaNA received $55 million in a funding round backed by pharma powerhouse Merck . This was not the first time Merck invested in mRNA technology. In January 2015, the company forged a deal with another Cambridge, Mass. mRNA company, Moderna Therapeutics for the discovery and development of vaccines and passive immunity treatments. Moderna has 11 drugs in its pipeline, including one developed with AstraZeneca that recently filed an application to begin a Phase I trial in Europe. The two companies are developing AZD8601, an investigational mRNA-based therapy for the treatment of cardiometabolic disease.

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