Orna Therapeutics Brings to Market New Therapy to Treat Cancer and Other Diseases

Orna Therapeutics, a Cambridge-based biotech company, announced on February 24th that they have managed to close their $80 million Series A financing round. The start-up originating out of Massachusetts Institute of Technology (MIT) focuses on designing and delivering a new class of mRNA therapeutics, called circular RNA (oRNA), to unleash the full potential of mRNA in treating a variety of diseases.

The financing round was led by MPM Capital, Taiho Ventures, and F2 Ventures, with participation from other investors including Kite, Bristol Myers Squibb, Astellas Venture Management, Novartis Institutes for Biomedical Research, and the PAGS Group.

“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,” said Dr. Thomas M. Barnes, Ph.D., Chief Executive Officer. “Furthermore, 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.”

Circular RNA

Although 98% of the genome in humans is not translated to proteins, most of it is transcribed into different types of RNAs. These transcribed RNAs were considered junk for a long time, until recent studies have attributed various physiological functions to them, including protein decay, stability, transcriptional regulator of mRNA, and others.

Circular RNA is one such type of non-coding RNA, which is a covalently closed single-stranded RNA. They can act as a microRNA sponge, thereby acting as a decoy for mRNA translation. Orna Therapeutics took advantage of these non-coding RNAs and developed fully synthetic circular RNA for therapeutic purposes (oRNA).

Applications of oRNA in the Medical Field

The groundbreaking research originating out of MIT discovered how to make oRNAs express higher levels of proteins than a linear stranded RNA. This would mean that patients could be given a lower dose to get the desired treatment and open up new alleys for diseases requiring high protein expression levels.

Over the past few years, Orna Therapeutics researched and developed delivery vehicles for their oRNA, ultimately narrowing it down to liver cells and immune cells. The company is determined to develop a method that would resemble cell therapy, except instead of removing and engineering a patient’s cells, they would target the immune cells using their proprietary toolbox.

“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. Furthermore, 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.” Dr. Barnes added.

While multiple companies aim to curb the limitations of CAR-T therapy and bring new technologies to the market, the holistic method used by Orna Therapeutics is yet to be tested. If the company manages to tick the right boxes with their proposed research using the cash from their financing round, we might be looking at better treatment options for several deadly diseases, including cancer.

References

1. https://www.prnewswire.com/news-releases/orna-therapeutics-launches-with-over-100m-raised-to-develop-a-new-class-of-fully-engineered-circular-rna-therapies-301234119.html
2. https://www.cell.com/molecular-therapy-family/molecular-therapy/fulltext/S1525-0016(19)30310-7

Author

Tulip Chakraborty