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Shire divests mRNA platform to RaNa

RNA activation specialist RaNa Therapeutics has acquired Shire's messenger RNA drug discovery platform, along with the scientists who have worked to develop it over the last eight years. Dublin-based Shire receives an equity stake in RaNa and is eligible for future milestones and royalties on products developed with the technology. Shire developed the mRNA Replacement Therapy (MRT) technology for both monogenic disease target validation and drug development. MRT enables the in vivo production of both intracellular and secreted proteins; the platform allows scalable manufacturing and can be applied to cell-free production and to the development of delivery vehicles. Cambridge, Massachusetts–based RaNa has been working on mRNA therapeutics since its founding in 2011, based on technology licensed from Massachusetts General Hospital around the discovery of long noncoding regions of RNA that interact with polycomb repressor complex 2 (PRC2). Blocking the interaction of this so-called 'junk RNA' with PRC2 activates the expression of single genes. RaNa has yet to advance a compound into the clinic using the technology; the Shire deal includes preclinical drug programs in cystic fibrosis and urea cycle disorders to augment RaNa's pipeline. In 2015, RaNa raised $55 million in venture capital to continue development of epigenetic gene upregulation and mRNA stabilization technologies and support the company's programs in spinal muscular dystrophy and Friedreich's ataxia. RaNa is also advancing a preclinical candidate to treat ornithine transcarbamylase deficiency, a rare X-linked genetic disorder.

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Shire divests mRNA platform to RaNa. Nat Biotechnol 35, 103 (2017).

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