RNA Therapies Developer Spins-Off Rare Disease Venture

Stéphane Bancel (Brenda Bancel, Wikimedia Commons)

12 May 2015. Moderna Therapeutics, a developer of RNA-based treatments, is starting a new company to design therapies with its technology to address rare diseases. Financial details of the spin-off company Elpidera LLC, the third such venture launched by Moderna, were not disclosed.

Moderna, a biotechnology enterprise in Cambridge, Massachusetts, develops medications that use genetic material to produce therapeutic proteins in the body, with a technology is based on research licensed from Harvard University and MIT. That technology harnesses messenger RNA, a nucleic acid related to DNA delivering genetic code used by cells to produce the amino acids in proteins for carrying out bodily functions. Moderna designs what it calls modified messenger RNA to produce proteins that act like drugs as treatments for diseases, creating antibodies, and in regenerative medicine, with the potential to cut the time and expense for creating therapeutic proteins over current recombinant methods.

Elpidera LLC plans to apply Moderna’s messenger RNA technology to rare diseases, which Moderna says is a vastly underserved field in pharmaceuticals, with some 7,000 rare diseases identified and few of those adequately addressed by current therapies. In January 2014, Moderna  agreed to provide Alexion Pharmaceuticals with 10 product options for treating rare diseases, for which Moderna received $100 million as well as a $25 million equity stake from Alexion. Moderna says the activities of Elpidera LLC will be separate from and not conflict with the work being undertaken for Alexion.

Elpidera is the third spin-off company from Moderna, itself founded in 2010, and the second spin-off in 2015. Moderna says it creates new ventures to allow the enterprises to concentrate on a single clinical area. In January 2015, Moderna started Valera LLC to apply messenger RNA technology to vaccines and infectious diseases. In January 2014, Moderna created a spin-off subsidiary Onkaido Therapeutics that applies messenger RNA technology to cancer treatments, including an early-stage drug discovery partnership with Karolinska Institutet in Sweden.

“The creation of new venture teams is critical to advancing our decentralized drug development business strategy at Moderna,” says CEO Stéphane Bancel in a company statement. “Ventures are the other leg of our strategy to add to the efforts of our pharma and biotech partners. In total, we are driving more than 50 preclinical mRNA programs across cardiovascular, infectious diseases, oncology, and rare diseases.”

Since its founding, Moderna attracted some $600 million in two venture funding rounds, as well as executing license agreements with AstraZeneca, Alexion Pharmaceuticals, and most recently with Merck that will collaborate with Valera LLC on vaccines and infectious diseases. In January 2015, Fortune magazine estimated Moderna has $800 million in cash and is valued at about $3 billion, with none of its therapies yet in human clinical trials.

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