Orna Therapeutics Presents New Preclinical Data Supporting its in vivo CAR Therapy Approach in Autoimmune Diseases at the American Society of Gene and Cell Therapy Annual Meeting

WATERTOWN, Mass., May 15, 2025 /PRNewswire/ -- Orna Therapeutics, a leading biotechnology company developing a proprietary pipeline of in vivo therapies across a broad range of autoimmune and oncology indications, today announced the presentation of new preclinical data supporting its in vivo CAR therapy approach in autoimmune diseases during an oral session at the 28^th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting being held May 13-17, 2025, in New Orleans, Louisiana.

"The preclinical data presented today at ASGCT highlight our potential to deliver on the promise of in vivo CAR T therapy," said Joseph Bolen, Ph.D., Chief Executive Officer of Orna Therapeutics. "Our CD19 panCAR program has demonstrated not only successful delivery of our lead panCAR LNP to disease-relevant immune cell types, but also robust and sustained B cell depletion at low doses in both peripheral blood and lymphoid tissues in non-human primates (NHPs). These compelling results continue to reinforce our commitment to translating our promising science into meaningful therapies for patients and we look forward to advancing our CD19 panCAR program towards the clinic in 2026."

Presentation Details:

Title: In Vivo panCAR™ Therapy Using Circular RNA for the Treatment of Autoimmune Disease
Speaker: Megan Hoban, Ph.D., panCAR Program Lead, Orna Therapeutics
Date/Time: Thursday, May 15, 2025, 8:00 AM – 9:45 AM CDT
Session Name: Cellular and Gene Therapies for Autoimmune Disease
Location: Room 388-390

In today's presentation, Orna will showcase preclinical data demonstrating the potential of its in vivo panCAR therapy, enabled by its proprietary circular (oRNA®) technology and best-in-class lipid nanoparticle (LNP) delivery system to achieve robust and sustained B cell depletion in both humanized mouse models and non-human primates across multiple doses.

Key findings from the study include:

• Validated extra-hepatic delivery to disease-relevant immune cell types, including T cells, in mice and NHPs without requiring targeting ligands.
• Lead panCAR LNP achieved over 60% delivery to peripheral blood and splenic T cells in NHPs.
• CD19 panCAR doses as low as 0.03mpk led robust B cell depletion, with multi-dosing achieving increased B cell depletion in humanized mice.
• In a humanized lupus mouse model, CD19 panCAR showed strong B cell depletion and a meaningful and differentiated reduction in dsDNA titers compared to rituximab.
• CD19 panCAR induced full depletion of B cells across peripheral blood, spleen, lymph nodes, and bone marrow in NHPs, with peripheral B cells beginning to reconstitute after three weeks.

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 oRNA 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, comprehensive editing programs and validating large pharma partnerships 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.

Orna Therapeutics Investor Contact:
Alex Lobo
Precision AQ
[email protected] 

SOURCE Orna Therapeutics