Orna Therapeutics to Present Novel, First-in-Class Circular RNA Data at Upcoming ASGCT 2022 Annual Meeting

First in vivo studies to demonstrate the potential of Orna’s circular RNA (oRNATM) platform in cancer, genetic disorders, and infectious diseases

Cambridge, Mass., May 2, 2022 – Orna Therapeutics, a biotechnology company dedicated to designing and delivering a new class of fully engineered circular RNA therapeutics, today announced multiple data presentations at the American Society of Gene and Cell Therapy (ASGCT) 25th Annual Meeting taking place in Washington, D.C., or virtually, from May 16 – 19, 2022. Oral presentations will describe Orna’s pipeline for the first time, revealing key data on its in situ CAR (isCAR™) program, amongst others, and detail the development of a powerful, new screening platform (FoRCE™). Poster presentations will provide additional information on the development of delivery solutions for the isCAR platform and on the development of our muscle genetic disease program.

Details for the presentations are shared below.

Oral Presentations:

In-situ CAR Therapy Using oRNA Lipid Nanoparticles Regresses Tumors in Mice

Presenter: Tom Barnes, Ph.D., CEO

Date/Time/Location: Monday, May 16, 2022 from 9:10 – 9:45 a.m. ET in Room 207

Session: Scientific Symposium: Function and Therapeutics Applications of Circular RNAs (circRNAs)

Summary: Orna will introduce its pipeline along with key data from its isCARTM program. Data from iterative animal studies will demonstrate that oRNA lipid nanoparticles (oRNA-LNPs) can be designed to overcome current challenges of engineered cell therapies. Orna will also present progress on oRNA-LNP (non-viral) delivery of long forms of dystrophin and advances with vaccine therapies.

Discovery of Translation Initiation Elements Enabled by a Parallel Arrayed Screen of Full-length Viral UTRs in Synthetic Circular RNA

Presenter: Alexander Wesselhoeft, Ph.D., Director, Molecular Biology

Date/Time/Location: Monday, May 16, 2022 from 11:30 – 11:45 a.m. ET in Salon H
Session: Oral Abstract Session: Oligonucleotide Therapeutics

Summary: Newly discovered internal ribosome entry sites (IRES) show greater activity (vs EMCV, a common IRES) and some produce different expression levels based on cell type, granting more options for improved expression and control of oRNA.

Poster Presentations:

Improved Immune Cell Expression with Circular RNA (oRNA) in vivo

Presenter: Kevin Kauffman, Ph.D., Principal Scientist

Date/Time/Location: Monday, May 16, 2022 at 5:30 p.m. ET in Hall D

Session: Poster Session: Oligonucleotide Therapeutics I

Summary: oRNA lipid nanoparticles (oRNA-LNPs) show higher splenic T cell expression and biodistribution to the spleen in vivo with improved formulation characteristics compared to their linear mRNA-LNP counterparts.

Systemic Delivery of Circular RNA Encoding Partial Dystrophins and Expression in Skeletal Muscle

Presenter: Tatiana Fontelonga, Ph.D., Scientist

Date/Time/Location: Tuesday, May 17, 2022 at 5:30 p.m. ET in Hall D

Session: Poster Session: Oligonucleotide Therapeutics II

Summary: Micro and mini versions of the dystrophin gene can be encoded in a high capacity oRNA, delivered via LNP, and properly expressed in primary human cells and the mdx mouse model of Duchenne muscular dystrophy.

About Orna Therapeutics
Orna Therapeutics is a biotechnology company dedicated to designing and delivering a new class of fully engineered circular RNA (oRNATM) therapeutics with the potential to change the way we treat disease. Orna’s proprietary platform combines novel technology to design circular RNA transcripts that drive protein expression with validated and unique delivery solutions. oRNATM has many advantages over traditional mRNA approaches, including simplified production, improved formulatability, and superior protein expression – making it a highly disruptive, new class of RNA therapeutics with vast potential to change patient’s lives. To learn more visit: and follow Orna Therapeutics on Twitter and LinkedIn.

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