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AstraZeneca, Biotech Partner on Messenger RNA Therapies

RNA illustration

RNA illustration (University College London, Flickr)

21 August 2017. Global drug maker AstraZeneca is acquiring an option to license the rights to a technology based on genetic material transcribed from DNA, for new respiratory disease treatments. The deal with MedImmune, AstraZeneca’s biologics subsidiary is expected to bring Ethris GmbH, a biotechnology company in Munich, an immediate payment of €25 million ($US 29.3 million).

Ethris develops therapies for respiratory and metabolic disorders based on engineered messenger ribonucleic acid or mRNA, a single strand of nucleic acid related to DNA with the instructions used by cells to produce the amino acids in proteins for carrying out functions in the body. Messenger RNA, however, is highly unstable and can induce an immune reaction, which makes it difficult to use directly in therapies. In addition, mRNA needs a mechanism for crossing cell membranes to take effect.

To overcome these obstacles, Ethris developed its Stabilized Non-Immunogenic mRNA, or SNIM-RNA technology. The company says SNIM-RNA modifies the chemical building blocks in mRNA to remove components invoking immune reactions, as well as stabilizing the mRNA so it can be taken repeatedly as a medication. Ethris says these modifications make it possible to design treatments with mRNA for hereditary diseases and in regenerative medicine that replace missing or boost non-functioning proteins. The company also devised a carrier for mRNA to cross cell membranes.

In Ethris’s pipeline are treatments for inherited respiratory diseases including cystic fibrosis and ciliopathies, genetic disorders that result in abnormal formation or function of cilia, tiny fragments on cells, including in the lungs and respiratory system. The agreement calls for Ethris and MedImmune in Gaithersberg, Maryland to use SNIM-RNA technology for identifying new respiratory disease targets including asthma, chronic obstructive pulmonary disease or COPD, and idiopathic pulmonary fibrosis.

In the 5-year deal, AstraZeneca, based in London, and MedImmune have an option to acquire a worldwide exclusive license for each target once research plans are completed. Ethris is receiving an initial fee of €25 million and unspecified research funding during the collaboration. Ethris will also be eligible for future R&D milestones and royalties on sales for therapies that reach commercialization stage.

The collaboration with Ethris adds to AstraZeneca’s profile with mRNA therapies. As reported in Science & Enterprise, AstraZeneca in 2013 licensed mRNA technology from Moderna Therapeutics in Cambridge, Massachusetts to develop treatments for heart disease, metabolic disorders, and cancer. That deal, valued as high as $420 million for Moderna, released its first product candidate for clinical trials in July 2016, a treatment for cardiometabolic diseases encoding the protein vascular endothelial growth factor-A. In August 2016, AstraZeneca announced it would invest another $140 million in Moderna.

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