Orna Therapeutics to Present Progress of Lead Program ORN-101 at PEGS and ASGCT

Orna’s circular RNA (oRNA) and LNP delivery technologies, integrated and enhanced by our FoRCE™ optimization technology, have created a new modality with potentially disruptive application in immuno-oncology

ORN-101, currently in development for B cell malignancies, demonstrates over 20x greater potency after optimizing IRES expression

CAMBRIDGE, Mass., May 4, 2023 Orna Therapeutics, a biotechnology company pioneering a new investigational class of engineered circular RNA (oRNA) therapies, today announced two presentations at the Protein Engineering Summit (PEGS) taking place May 15-19 in Boston as well as a poster presentation at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in Los Angeles May 16-20. Orna is presenting preclinical results for ORN-101, an in situ CAR program in development, as well as further details about its platform.

“We are paving the way for advancements in RNA therapeutics that reach beyond infectious disease to treat cancer and genetic disorders,” said Robert Mabry, PhD, Chief Scientific Officer at Orna. “Our ORN-101 program demonstrates the benefits of our platform’s ability to create a paradigm-changing treatment with the characteristics we believe matter most to patients, including a rapid timeline from diagnosis to treatment, strong expression and tumor-killing potency at low dose, and a regimen without the harsh chemotherapeutic lymphodepletion protocols typically required for standard engineered cell therapies.”

Details for the presentations are as follows:

PEGS presentations

Title: Synthetic Circular RNA as a New Therapeutic Modality

Speaker & Chair: Nelson Chau, PhD, Senior Vice President, Platform

Session: Innovating RNA Therapeutics

Time: Tuesday May 16, 4:30pm ET

Title: In situ CAR Therapy Using oRNA

Speaker: Amy Becker, PhD, Director of Immunology

Session: Cellular Reprogramming, Increasing Specificity

Time: Thursday, May 18, 1:50pm ET

ASGCT poster

Title: In situ CAR Therapy Using oRNA Lipid Nanoparticles Regress Tumors in vivo

Speaker: Akinola Emmanuel, PhD, Principal Scientist

Board: 1119

Time: Thursday, May 18, 12-2pm PT

About ORN-101:

ORN-101, Orna’s lead program, is an in situ CAR therapy designed to modify a patient’s immune cells inside their body. Comprising an oRNA molecule packaged inside a proprietary lipid nanoparticls (LNP) formulation, this easily redosable format could avoid patient lymphodepletion and allow for reliable dose control, overcoming barriers of existing ex vivo CAR-T therapies without sacrificing efficacy. Preclinical data demonstrates tumor suppression and eradication in an animal model, suggesting the possibility that oRNA-LNP based cancer therapies could eventually overtake cell therapies.

About Orna Therapeutics:

Orna Therapeutics is reshaping RNA therapeutics to open a new world of treatment possibilities. Orna’s circular RNA (oRNA) is created in one step by self-circularization of an engineered linear RNA. In this form, oRNA enjoys unmatched advantages over traditional linear mRNA therapies including simplified production, increased protein expression, easier encapsulation into lipid nanoparticles (LNPs), and immunoquiescence. Orna’s proprietary LNPs deliver the oRNA to specific areas of the body, such as immune cells. Founded in 2019 based on groundbreaking research from MIT, Orna was built by MPM Capital and its affiliate BioImpact Capital. To learn more visit: and follow us on Twitter and LinkedIn.

Media Contact

Peg Rusconi, Verge Scientific Communications