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Synthetic RNA Start-Up Gains $80M in Early Funds

RNA strand illustration

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

24 Feb. 2021. A start-up company, spun off from a biomedical engineering lab at MIT, is raising $80 million for therapies made from synthetic circular RNA. The two year-old Orna Therapeutics, in Cambridge, Massachusetts, is based on research by company founder R. Alexander Wesselhoeft while a doctoral candidate in the chemical and biomedical engineering lab of Daniel Anderson at Massachusetts Institute of Technology.

Orna Therapeutics is designing a technology with RNA produced in circular form, for therapies that the company says improves on messenger RNA, as used in new vaccines for preventing Covid-19 disease. According to its developers, circular RNA, or oRNA as its called by the company, is easier to produce than messenger RNA, more compact thus easier to package in lipid nanoparticles, and more durable to provide higher levels of protein expression.

The company expects to apply its technology to regenerative medicine, as well as treatments for cancer, infectious diseases, and autoimmune disorders. An application of oRNAs being explored by Orna Therapeutics is delivery of chimeric antigen receptors directly to patients’ T-cells, rather than producing engineered T-cells, either from the patient’s own T-cells or cultured from healthy donors.

Started by life science venture investor

Wesselhoeft’s dissertation research in Anderson’s lab at MIT produced and demonstrated single-stranded RNA in circular form. His process creates a self-splicing intron, a section of genes that does not code for amino acids, which produces circular RNA in the lab. In papers published in the journals Nature Communications and Molecular Cell, Wesselhoeft described translating circular RNAs first in lab-culture cells, and then in lab mice. In the tests with mice, the circular RNA in purified form did not produce an immune reaction, but when linear RNA is added, can produce a strong immune response.

Orna Therapeutics was formed by life science venture investor MPM Capital in Cambridge, Mass. that provided seed capital and incubation. Along with Wesselhoeft and Anderson, the company is also founded by Ansbert Gadicke, managing director of MPM Capital, and Raffaella Squilloni, former entrepreneur In residence at Harvard University. Wesselhoeft is Orna Therapeutics’ director of molecular biology, while Squilloni is the company’s director of business development. Anderson is on the company’s board of directors.

“Our elegant solution to the circular RNA engineering problem has allowed us to reveal that oRNA is simply the better format for long coding RNA, says Thomas Barnes, Orna Therapeutics’ new CEO in a company statement. Barnes adds, “our initial combination of oRNAs with technology to deliver them to immune cells has paved the way to create groundbreaking new therapies to fundamentally change the way we treat cancer and autoimmune diseases.”

Orna Therapeutics is raising $80 million in its first venture funding round. The financing is led by MPM Capital, Taiho Ventures, and F2 Ventures. Taking part in the round are the PAGS Group and venture investing or research arms of drug and biologic makers Gilead Sciences, Bristol Myers Squibb, Astellas, and Novartis. The company expects to use the proceeds to advance its technology, as well as for new lab and office space.

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