Orna Therapeutics Unveils Promising Preclinical Results for CAR Therapy Targeting Autoimmune Disorders

Orna Therapeutics Unveils New Insights into In Vivo CAR Therapy in Autoimmune Diseases

At the esteemed 28th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting held from May 13-17, 2025, in New Orleans, Louisiana, Orna Therapeutics presented groundbreaking preclinical data that supports its innovative in vivo CAR therapy for autoimmune diseases. This presentation marks a significant milestone in the landscape of biotechnological advancements aimed at treating complex autoimmune conditions.

CEO Joseph Bolen, Ph.D., emphasized the potential of their therapy, stating, "The preclinical data highlight our ability to fulfill the promise of in vivo CAR T therapy. Our focus on delivering effective solutions for autoimmune disorders underlines our commitment to improving patient outcomes."

Key Findings of the Presentation

During the oral session, Megan Hoban, Ph.D., the panCAR program lead at Orna Therapeutics, detailed the compelling results that stem from their proprietary circular RNA (oRNA®) technology paired with a state-of-the-art lipid nanoparticle (LNP) delivery system. Some pivotal findings include:

1. Effective Extra-Hepatic Delivery: They validated a highly effective delivery mechanism, ensuring disease-relevant immune cells, including T cells, received targeted treatment without the necessity of targeting ligands in both mice and non-human primates (NHPs).

2. High Delivery Rates: Their leading panCAR LNP achieved delivery rates exceeding 60% to T cells in peripheral blood and splenic regions of NHPs, demonstrating significant promise in therapeutic applications.

3. Low-Dose Efficacy: A noteworthy highlight was the observation that doses as low as 0.03mpk of CD19 panCAR led to substantial B cell depletion, enhanced by successive dosing that intensified the depletion effects in humanized mouse models.

4. Impact on Lupus Models: In humanized lupus mouse models, the CD19 panCAR exhibited not only strong B cell depletion but also a significant reduction in dsDNA titers when compared with the widely-used treatment agent rituximab.

5. Comprehensive B Cell Depletion: Observations in NHPs showed that the CD19 panCAR facilitated complete depletion of B cells across multiple anatomical compartments, including peripheral blood, spleen, lymph nodes, and bone marrow, with signs of reconstitution of peripheral B cells noticed after three weeks.

Looking Ahead

Orna Therapeutics is set to advance its CD19 panCAR program towards clinical trials by 2026, showing an unwavering commitment to translating scientific breakthroughs into tangible therapies that can impact patient care significantly. The potential for this technology to treat various immune-mediated diseases marks a pivot point in therapeutic options, aligning with current needs in precision medicine.

Orna’s focus remains steadfast on harnessing the transformative power of RNA-based treatments. By utilizing fully engineered circular RNA therapeutics, the company aims to unlock unprecedented therapeutic possibilities. The advantages of this method over traditional mRNA approaches remain a center of attention in the industry, especially regarding production efficiency and protein expression capabilities.

In conclusion, as Orna Therapeutics leads the charge in CAR therapy development, the focus on autoimmune diseases reveals not only a pathway to advanced treatments but also shines a spotlight on the positive trajectory of biotechnology aimed at comprehensive healthcare solutions.

For further information about Orna Therapeutics and their breakthroughs in the field, visit Orna Therapeutics' official site and keep updated through their social media channels on platforms like X and LinkedIn.