Orna Therapeutics: Tapping into the Power of oRNA

RNA has recently ascended as a therapeutic class with vast potential. Existing mRNA technologies, however, face numerous and ongoing challenges to production, delivery, and performance. Circular RNA, by its very nature, has the potential to address these challenges, but the technology to create and engineer these molecules efficiently in a laboratory setting remained undeveloped. Founded in 2019, Orna Therapeutics has this technology – building on nature’s ingenuity with its sophisticated molecular innovations to advance a system for protein expression, RNA production, and superior delivery compared to conventional long RNA approaches.

The Cambridge, Massachusetts-based company has married these capabilities with exciting delivery solutions to create a simple, powerful platform: the next breakthrough in RNA therapeutics. Built on groundbreaking research from Prof. Daniel Anderson’s lab at MIT, the company's oRNA^TM technology is the next revolution in RNA therapeutics: synthetic circular RNA efficiently generated by an elegant and proprietary molecular platform. oRNA^TM circular RNAs lack caps and tails and are always full-length, and are made by combining nature-driven insights with rational design. They are easily made, well-tolerated, and are engineered to express therapeutic proteins through original and proprietary mechanisms.

RNA has recently ascended as an exciting therapeutic class. Current mRNA technologies, however, face numerous and ongoing challenges to production, delivery, and performance that have prevented the field from reaching its full potential. Lacking caps or tails and having greater cellular persistence, circular RNA had the potential to address these challenges. However, the technology to create and engineer them at will in a manner that was also scalable was absent – until now.

"The company's oRNATM molecules are easy to make, are always full length, and are well-tolerated inside cells creating a simple, powerful platform."

Inspired by RNA enzymes and RNA viruses, Orna now possesses this technology – harnessing sophisticated molecular innovations to advance a system for production, delivery, and performance that is superior to conventional long RNA approaches. The company's oRNA^TM molecules are easy to make, are always full length, and are well-tolerated inside cells creating a simple, powerful platform. From the design of the oRNA^TM molecules to their delivery, Orna carefully optimizes each step for superior product performance in the body.

The potential applications of Orna’s technology are far reaching, including cancer, autoimmune diseases, regenerative medicine, and genetic disorders. Orna is currently evaluating where its medicines that can make the biggest difference, with an initial focus on treating challenging diseases where other modalities have fallen short. As an initial focus, Orna believes it can apply its oRNA^TM technology to potentially address the limitations of current immunotherapies by delivering chimeric antigen receptors (CARs) directly to patient’s immune cells within the body (isCAR^TM therapy).

In March 2022, Orna Therapeutics, a biotechnology company dedicated to designing and delivering a new class of fully engineered circular RNA therapeutics (oRNAᵀᴹ), announced the appointment of Thomas Ebeling to the Board of Directors and William Shen, Ph.D., as Chief Business Officer. Both appointments provide the opportunity to advance Orna’s strategy and expand relationships to create an entirely new class of RNA medicines poised to change the way they treat disease.

Earlier in his career, Mr. Ebeling served as Chief Executive Officer of Novartis Pharmaceuticals. Under his leadership, he was responsible for more than doubling the company’s revenues, after which he became Chief Executive Officer of their Consumer Health Division. Most recently, he served as Chief Executive Officer of ProSiebenSat.1 Media SE. Mr. Ebeling also served on the Boards of Bayer and Lonza and now serves as an Advisor to MPM Capital. Dr. Shen joins Orna from his former role as Chief Operating Officer of Virtuoso Therapeutics with more than 19 years of experience in R&D, venture capital, and business development. Prior to Virtuoso, Dr. Shen served as Vice President, Head of Business Development at Biogen. Under his leadership, Biogen completed more than 15 transactions in 2020, including partnerships with Denali Therapeutics and Sage Therapeutics, two of the largest neuroscience deals that year. Dr. Shen also served as the M&A Lead at Bristol Myers Squibb (BMS), where he led the acquisition of IFM Therapeutics and Cardioxyl Pharmaceuticals, as well as the option agreement to acquire Promedior.

"Built on groundbreaking research from Prof. Daniel Anderson’s lab at MIT, the company's oRNA^TM technology is the next revolution in RNA therapeutics: synthetic circular RNA efficiently generated by an elegant and proprietary molecular platform."

About the Leader

Thomas Barnes, PhD, Chief Executive Officer

Tom is a veteran entrepreneur who has turned creative scientific visions into successful businesses for both startups and established organizations for over 20 years and has co-founded or helped launch several companies, including Intellia Therapeutics and Eleven Biotherapeutics.

While at Intellia Therapeutics, a CRISPR/Cas9 company which Tom joined as Chief Scientific Officer, he helped build the research team, raise funds (including an IPO), and provide a vision for technology extension. He played a significant role as the corporate spokesperson to lay and industry press, and to governmental and non-governmental organizations on the rapidly evolving and intensely followed CRISPR landscape. Tom has wide-ranging knowledge of biological systems through his work across diverse platforms including gene editing, genomics and gene discovery, small molecule drug repositioning, and protein engineering. In addition to Tom’s many accomplishments he has also survived a shark tank!

He received his PhD from the University of Cambridge and completed research fellowships at Harvard Medical School and McGill University. He is currently also an Executive Partner at MPM.