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Orna Therapeutics Unveils Groundbreaking Preclinical Data at ASH 2025

Orna Therapeutics has emerged as a pioneering force in biotechnology, particularly in the field of immunotherapy. In their latest announcement, the Massachusetts-based company provided new data showcasing their in vivo CAR programs targeted at autoimmune diseases and oncology, which was presented at the 67th American Society of Hematology (ASH) Annual Meeting held in Orlando, Florida, from December 6-9, 2025.

In a detailed address, Orna's Chief Executive Officer, Joseph Bolen, Ph.D., discussed the promising findings from preclinical studies. These studies demonstrate the powerful potential of Orna's innovative technology, which employs non-viral, transient, and tunable strategies to develop CAR therapies without the need for preconditioning lymphodepletion. This approach, according to Bolen, not only showcases robust activity in addressing B cell autoimmunity but also holds promise for treating diseases linked to BCMA.

Innovative Approaches in In Vivo CAR Technology

Orna's panCAR platform has shown significant potential in both B cell autoimmunity and BCMA-related diseases. The preclinical research revealed notable findings for both ORN-252, their anti-CD19 panCAR, and their anti-BCMA approaches. In the humanized lupus model, ORN-252 achieved remarkable B cell depletion with a simultaneous reduction in anti-dsDNA titers, a crucial indicator in lupus treatment.

Furthermore, Orna's studies highlighted that the treatment led to complete depletion of B cells in non-human primates (NHPs) at notably low doses, indicating a high efficacy and promising translational potential for human patients. The results also showed that CAR+ T cells gained significant cytotoxic markers immediately after the initial dose, enhancing their capability to target and kill diseased cells effectively.

In Vivo panCAR™ Therapy: A Closer Look

A presentation titled “In Vivo panCAR™ Therapy Utilizing Circular RNA for Treatment of Autoimmune Diseases” focused on the revolutionary technology that Orna is utilizing. Their proprietary circular RNA (oRNA®) technology, combined with a best-in-class lipid nanoparticle (LNP) delivery system, has positioned Orna at the forefront of in vivo therapy innovation. Highlights from this presentation included:

• - Significant B Cell Depletion: ORN-252 demonstrated robust B cell depletion in humanized mice at doses as low as 0.03 mg/kg.
• - Enhanced Efficacy: Compared to rituximab, ORN-252 displayed superior results in reducing dsDNA levels in lupus models.
• - Complete B Cell Depletion in NHPs: The therapy achieved a total B cell depletion at doses of 0.1 mg/kg, arguing for its potential application in humans.

Moreover, the anti-BCMA panCAR therapy results showcased its promising ability to eliminate tumors in humanized models for multiple myeloma, demonstrating superior tumor control and specific plasma cell depletion without affecting the broader B cell repertoire in NHPs.

Looking Ahead: Clinical Trials on the Horizon

As Orna Therapeutics continues to gather compelling data, the company is poised to advance its clinical aspirations. Bolen emphasized a timeline wherein they expect to submit their first Clinical Trial Application for ORN-252 by the end of 2025 and kick off their first in-human study in early 2026. This expected leap into clinical trials signifies an important milestone not only for Orna but also for numerous patients those therapies stand to benefit.

About Orna Therapeutics

Orna Therapeutics is at the helm of innovative medical solutions, focusing on circular RNA to revolutionize treatment options for various diseases. Their commitment to developing advanced in vivo therapies is set to redefine standards in RNA medicine. Through their transformative approaches, Orna’s ability to develop robust RNA-based therapeutics is drawing attention in the medical community, with the potential to substantially improve patient outcomes. Stay updated on their latest advancements by visiting Orna Therapeutics' website or following them on social media channels.