Collaboration at the core

Drawing on our diverse backgrounds, we collaborate as a team with one goal in mind: to help people.

RaNA’s technology has broad therapeutic potential to treat a wide range of diseases, including rare genetic disorders. RaNA’s goal is to generate new, highly selective therapeutics, with lead programs in cystic fibrosis and ornithine transcarbamylase (OTC) deficiency, a urea cycle disorder.

Our Genetic Code

Based in Cambridge, Massachusetts, RaNA was co-founded in 2011 by Dr. Jeannie T. Lee, M.D., Ph.D., of Massachusetts General Hospital, who pioneered the discovery of the relationship between lncRNA and chromatic modifiers, along with Jean-Francois Formela, M.D., partner at Atlas Venture, and Art Krieg, M.D., co-inventor on 44 issued U.S. patents covering oligonucleotide technologies. In 2015, RaNA successfully completed a $55 million oversubscribed Series B financing, co-led by MRL Ventures, the early-stage therapeutics fund of Merck & Co., and The Baupost Group, LLC, with additional participation from new investors Rock Springs Capital Management, Brookside Capital, Leerink Partners LLC and an undisclosed blue chip public investment fund. Existing investors Atlas Venture, Monsanto, MS Ventures, Omega Funds, Partners Innovation Fund, Pfizer Venture Investments and SR One also participated in the round. One also participated in the round. In December 2016, RaNA acquired the MRT platform, a mRNA therapy platform, developed by Shire plc.


We are actively seeking partnerships with industry leaders in areas of high unmet need including immuno-oncology, inflammation, metabolic, ophthalmologic and neurodegenerative diseases. We are currently looking for partners with dedication and expertise in these areas to develop next generation RNA-based precision medicines that can improve the lives of people suffering from serious life-altering conditions.

Please contact us at for more information.

IP Stats


issued U.S. patents

> 70

target genes, many of which are aligned with highest priority therapeutic programs


pending patent applications

If you are a fearless collaborator eager to discuss how to make the lives of patients better, we’d love to hear from you.