Moderna Therapeutics emerged from stealth mode with a $40 million financing led by Flagship Ventures and a consortium of private investors. Moderna is developing a platform technology for delivery of messenger RNA (mRNA) to stimulate production of therapeutic proteins in the cells.

It has preclinical programs in several areas: oncology supportive care, inherited genetic disorders, hemophilia and diabetes. Proof-of-concept studies in nonhuman primates have demonstrated in vivo production of target proteins.

According to Moderna CEO Stephane Bancel, Moderna's founders had good reason to fly under the radar since the company was founded in July 2011. "Because of how profound the impact of the technology is, as a brand new class of drugs . . . we can only start talking about it when we are sure it's working in a robust way," Bancel said.

In addition, discretion was necessary while the Moderna legal team worked to build a bulletproof intellectual property estate around the technology.

Moderna's mRNA therapeutics are designed to trigger production of therapeutic proteins in the cells without setting off the innate immune response. The company said it believes the technology is potentially revolutionary, as a completely new way of making a protein therapeutic, avoiding the need to manufacture the protein in a factory.

Bancel said the technology can produce proteins that are secreted into the bloodstream, and because the technology works inside the cell, it could potentially be used to address diseases that are considered currently undruggable.

According to Moderna, it also reduces the timeline for preclinical development and testing of therapeutics to just a few weeks.

The technology is now the subject of more than 80 patent applications, with more than 4,000 claims directed to the company's technology platform and discovery and development programs.

"As biotech and pharma companies realize the power of this technology, they will come work with us. We'll have so many patents, it will be impossible to develop an mRNA therapeutic without access to Moderna technology," Bancel said.

Moderna was founded by Noubar Afeyan, a managing partner and CEO of Flagship Ventures, with Harvard University's Derrick Rossi and Ken Chein, and with Massachusetts Institute of Technology scientist Robert Langer.

Rossi's work on creating induced pluripotent stem (iPS) cells at Harvard provided the basis for Moderna's technology. IPS cells have embryonic stem cell-like properties, but are created by reprogramming adult cells. Rossi used synthetic, modified RNA to generate the iPS cells, without irreversibly altering the cellular DNA.

Previous attempts at creating iPS cells used DNA or viruses, and mRNA was a significant advance. However, some engineering was required to avoid triggering the cell's immunity. The resultant chemically modified RNAs were able to sneak into the cell and drive expression of the desired proteins.

That work was published in 2010 in Cell Stem Cell. The success of the synthetic mRNAs for creating iPS cells hinted at wider use, with potential for just about any disease caused by an absent or defective protein. Moderna was formed to exploit that potential.

Flagship Ventures has been involved in funding a number of other biotech start-ups. It recently supported Visterra Inc. in a $26 million Series A financing. Visterra is developing a broad-spectrum antibody for influenza. (See BioWorld Today, Sept. 12, 2012.)

It also lent a hand to combination drug start-up Blend Therapeutics Inc. early in 2012. (See BioWorld Today, July 9, 2012.)

In other financings news:

• AcelRx Pharmaceuticals Inc., of Redwood City, Calif., proposed an underwritten public offering of 10 million shares of its common stock, with a 30-day option for underwriters to acquire up to an additional 1.5 million shares for overallotments. The company did not disclose pricing or use of proceeds. Jefferies & Co. Inc. and Cowen and Co. LLC are acting as joint book-running managers, with Canaccord Genuity Inc. acting as co-lead manager. On Wednesday, the company's shares (NASDAQ:ACRX) lost 29 cents, closing at $3.65.

• Rexahn Pharmaceuticals Inc., of Rockville, Md., closed its underwritten public offering of 19.1 million units and a partial exercise of the underwriter's overallotment option for a total offering of 20 million units, priced at 33 cents each. Including the overallotment, gross proceeds were $6.6 million. Maxim Group LLC and Burrill LLC acted as joint book-running managers.

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