Orna Therapeutics Presents New Data Supporting its in vivo CAR Programs for Autoimmune Diseases and Oncology at the 67th American Society of Hematology Annual Meeting
Anti-CD19 panCARTM program (ORN-252) demonstrated robust B cell depletion with significant reduction in anti-dsDNA titers in humanized lupus model
Anti-BCMA panCARTM program showed robust and durable plasma cell depletion in preclinical models
Orna expects to submit its first Clinical Trial Application for ORN-252 by the end of 2025 and anticipates initiating a first-in-human study in early 2026
WATERTOWN, Mass., Dec. 7, 2025 /PRNewswire/ -- Orna Therapeutics (Orna), a biotechnology company dedicated to engineering immune cells in vivo to treat autoimmune and oncology diseases, today announced new preclinical data supporting the Company's in vivo CAR programs to target and treat a broad range of B cell-mediated autoimmune diseases and plasma cell or BCMA-related diseases at the 67th American Society of Hematology (ASH) Annual Meeting taking place in Orlando, Florida from December 6-9, 2025.
"The preclinical data presented at this year's ASH Annual Meeting further highlights our potent, non-viral, transient, tunable, and scalable approach to in vivo CAR therapies in both autoimmune diseases and oncology," said Joseph Bolen, Ph.D., Chief Executive Officer of Orna. "Our panCAR platform has demonstrated robust activity in B cell autoimmunity and BCMA-related diseases without the need for preconditioning lymphodepletion. Additionally, the non-human primate (NHP) data presented across both programs demonstrated specific and targeted depletion of B cells or plasma cells. As we look ahead, we anticipate submitting our first Clinical Trial Application for ORN-252, our anti-CD19 panCAR program this year and entering the clinic in early 2026."
Oral Presentation details:
In Vivo panCAR™ Therapy Utilizing Circular RNA for Treatment of Autoimmune Diseases
In this presentation, Orna highlighted preclinical data demonstrating the potential of its proprietary circular (oRNA®) technology paired with its best-in-class lipid nanoparticle (LNP) delivery platform to enable the next generation of in vivo therapies and treat a variety of B cell-mediated autoimmune diseases.
Key findings in the study include:
- ORN-252, Orna's anti-CD19 panCAR mediated robust B cell depletion in humanized mice in vivo at doses as low as 0.03 mg/kg.
- In a humanized mouse lupus model, ORN-252 showed robust B cell depletion with concurrent reduction in dsDNA titers in contrast to rituximab.
- Treatment with ORN-252 led to complete peripheral and splenic B cell depletion in NHP at doses as low as 0.1 mg/kg.
- CAR+ T cells upregulated cytotoxicity markers after the first dose when the majority of cell killing occurs.
- ORN-252 demonstrated durable B cell depletion with a reduction in switched memory and an increase in naïve B cell phenotype upon repopulation.
- ORN-252 showed superior binding affinity, expression, and killing of CD19 cells in human vs. NHP cells, potentially providing increased potency upon translation to humans.
Orna anticipates filing a Clinical Trial Application for this program by the end of 2025 and expects to initiate a first-in-human study in early 2026.
Poster Presentation details:
In Vivo panCARTM Therapy Utilizing Circular RNA for Treatment of Multiple Myeloma
In today's poster presentation, Orna will showcase preclinical data demonstrating the potential of its in vivo panCARTM therapy to treat a range of plasma cell or BCMA-related diseases including multiple myeloma.
Key findings in the study include:
- High surface expression of anti-BCMA panCAR was observed in a dose-dependent manner on human and cynomolgus immune cells and was maintained at least 72 hours in vitro.
- In a humanized mouse model engrafted with BCMA-expressing tumor cells, anti-BCMA panCAR demonstrated superior tumor control, eliminating tumors for at least 30 days, functionally outperforming a clinically validated anti-BCMA binder.
- In NHPs, treatment with BCMA panCAR resulted in specific and targeted plasma cell depletion.
- Anti-BCMA panCAR is highly selective, demonstrating no significant impact on the overall B cell repertoire in NHPs.
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 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 on X and LinkedIn.
Orna Therapeutics Investor Contact:
Alex Lobo
Precision AQ
SOURCE Orna Therapeutics
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