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Gene Silencing Technology Licensed in $3.7B Deal

RNA strand illustration

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

29 Oct. 2018. Dicerna Pharmaceuticals, developing treatments for disease that silence genes by interfering with their RNA instructions, is licensing its technology to drug maker Eli Lilly and Co. The deal with Eli Lilly and Co. in Indianapolis could bring Dicerna Pharmaceuticals Inc. in Cambridge, Massachusetts as much as $3.7 billion if all aspects of the agreement are fulfilled.

Dicerna Pharmaceuticals is a biotechnology enterprise designing therapies that block the actions of disease-causing genes using a process known as RNA interference. RNA is genetic material produced by genes with the instructions to cells coded from DNA to synthesize proteins. With RNA interference, those instructions are interrupted, preventing production of proteins from disease-causing genes, while limiting effects on other genes, RNA, and protein synthesis.

Dicerna’s calls its technology GalXC that silences disease-causing genes in the liver. The company’s process adds N-acetylgalactosamine, a sugar molecule, to Dicer enzymes that process double-stranded RNA. Dicerna says these engineered enzymes prevent messenger RNA, the part of RNA molecules with protein synthesis instructions, from reaching the target cells. The company says GalXC treatments are stable and can be delivered as injections under the skin, while targeting cells in the liver. Moreover, says Dicerna, the process is flexible enough to be applied to a range of target genes, addressing a number of disease types including viral infectious diseases, chronic liver disorders, cardiovascular conditions, and some rare diseases.

The agreement calls for Lilly and Dicerna to collaborate on development of RNA interference, or RNAi, therapies for more than 10 disease targets. Dicerna will work exclusively with Lilly on pain and neurodegenerative disorders, and on select, but undisclosed cardio-metabolic diseases.

Dicerna is receiving an initial payment of $100 million, as well as an equity investment from Lilly also valued at $100 million. In addition, Dicerna is eligible to receive up to $350 million for each disease target in the collaboration. Thus if the initial 10 disease targets are developed, the milestone payments alone could reach $3.5 billion. Dicerna is eligible as well for royalties on sales of products brought to market in the collaboration.

Daniel Skovronsky, Lilly’s senior vice-president and chief scientist, says in a joint statement that his company is eager use Dicerna’s “RNAi expertise to study targets that up until now have proven to be very technically challenging. RNAi has the potential to treat an array of diseases that are of strategic importance to Lilly. Together with Dicerna, we aim to employ this emerging modality for greater success in drug development.”

As reported about a year ago in Science & Enterprise, Lilly is expanding its portfolio of therapies that harness RNA. In October 2017, Lilly and the German biotech company CureVac AG agreed to develop a series of cancer immunotherapies based on CureVac’s messenger RNA technology. The deal, if completed in full, could bring CureVac as much as $1.8 billion.

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