Orna Therapeutics Presents Data Highlighting Novel panCAR™ Approach to Unlock LNP-Mediated RNA Delivery for B Cell Mediated Diseases at the 66th ASH Annual Meeting

--A single dose of Orna's anti-CD20 panCAR™ resulted in a significant reduction in non-human primate (NHP) B cells, with sustained depletion observed 7 days after dosing--

--Approach offers a transient and scalable immune cell therapy without the need for lymphodepletion--

WATERTOWN, Mass., Dec. 9, 2024 /PRNewswire/ -- Orna Therapeutics, a biotechnology company dedicated to designing and delivering a new class of circular RNA medicines and unprecedented lipid nanoparticle (LNP) delivery solutions for oncology and autoimmune diseases, today presented a poster highlighting new preclinical data that demonstrates the potential of its in vivo panCAR™ platform for the treatment of B cell mediated diseases at the 66th American Society of Hematology (ASH) Annual Meeting taking place in San Diego from December 7-10, 2024.

Orna's lead LNP candidate, demonstrated effective delivery of oRNA cargo to 60% of splenic T cells and 84% of peripheral blood T cells in non-human primates (NHPs). When paired with an oRNA cargo, the Company's LNP demonstrated up to ~70% delivery of an anti-CD20 CAR to NHP T cells and human T cells in vitro and maintained expression over 72 hours. A single dose of the anti-CD20 panCAR™ resulted in a 95% reduction in NHP B cells, with sustained depletion (82%) observed 7 days after dosing.

"Our panCAR™ approach combining a synthetic, circular coding RNA platform oRNA^® and proprietary immunotropic LNP holds the potential to deliver an entirely novel class of in vivo CAR therapies that eliminate lengthy patient cell processing and lymphodepletion regimens," said Joe Bolen, Ph.D., Chief Scientific Officer for Orna. "The data presented today at ASH further validate our hypothesis, demonstrating our ability to effectively deliver oRNA cargo and achieve robust and sustained B cell depletion in NHPs, highlighting the potential of our work in advancing our in vivo CAR therapies across both cancer and autoimmune diseases towards the clinic."

Additional key findings are as follows:

• In cytotoxicity assays, both the anti-CD20 CAR and an anti-CD19 CAR construct killed human B lymphoblast cell lines in vitro.
• In vivo, both the anti-CD19CAR and anti-CD20 CAR oRNAs showed significant B cell depletion that manifested as a 75-80% reduction in B cells in peripheral blood, spleen, and bone marrow at 24 hours. This effect was sustained for 7 days after dosing in peripheral blood and bone marrow.

About Orna Therapeutics
Orna Therapeutics is dedicated to designing and delivering a new class of fully engineered circular RNA (oRNA^®) therapeutics to unlock the potential of RNA medicine to treat diseases anywhere in the body. Orna's circular RNA transcripts have advantages over traditional mRNA approaches, including simplified production, improved formulation into lipid nanoparticles, and superior protein expression. Its industry-leading LNP-based delivery systems and comprehensive editing programs position Orna to advance novel RNA medicines with vast potential to transform patient care. To learn more, visit: www.ornatx.com and follow Orna Therapeutics on X and LinkedIn.

Investor Contact:
Precision AQ
Alex Lobo
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SOURCE Orna Therapeutics