Key Takeaways

• Orna Therapeutics presented promising preclinical data on its ORN-252 anti-CD19 panCAR program and its anti-BCMA panCAR program at the ASH Annual Meeting.
• ORN-252 demonstrated robust B cell depletion in a humanized lupus model and is set to enter clinical trials by early 2026.
• The anti-BCMA panCAR showed effective tumor control in multiple myeloma models, indicating significant potential for treating plasma cell-related diseases.

Orna Therapeutics, based in Watertown, Massachusetts, made headlines at the 67th American Society of Hematology (ASH) Annual Meeting by presenting groundbreaking preclinical data supporting its in vivo CAR programs. These novel therapies aim to target B cell-mediated autoimmune conditions and plasma cell-related diseases, such as multiple myeloma.

Joseph Bolen, Ph.D., CEO of Orna, emphasized the advantages of their panCAR platform, highlighting its effectiveness in B cell autoimmunity and BCMA-related diseases without the need for prior lymphodepletion. The data showcased also indicated successful specific depletion of B cells and plasma cells in non-human primate (NHP) models. Orna plans on submitting its first Clinical Trial Application for the ORN-252 anti-CD19 panCAR program by the end of 2025 and hopes to kick off human trials in early 2026.

Focus on Autoimmune Diseases

The company presented preclinical findings about ORN-252’s efficiency in treating lupus. Highlights from the studies included:

– ORN-252 achieved significant B cell depletion in humanized mice with minimal dosing, as low as 0.03 mg/kg.
– In a lupus model, the therapy substantially reduced anti-dsDNA titers when compared to rituximab, a standard treatment.
– ORN-252 resulted in total B cell depletion in NHPs at doses starting from 0.1 mg/kg, demonstrating strong effectiveness.

The CAR+ T cells activated cytotoxicity markers prominently after the first dose, and the treatment sustained durable B cell depletion, enhancing naïve B cell appearance over time.

Advancements in Multiple Myeloma Treatment

Additionally, Orna showcased its anti-BCMA panCAR program aimed at multiple myeloma and related conditions. Key findings from this research revealed:

– The anti-BCMA panCAR exhibited significant expression on immune cells in a dose-dependent manner and retained effectiveness for at least 72 hours in vitro.
– In a humanized mouse model with BCMA-expressing tumor cells, this therapy led to superior tumor control, successfully eliminating tumors for over 30 days, outperforming existing anti-BCMA treatments.
– The anti-BCMA panCAR led to targeted plasma cell depletion in NHPs while sparing the overall B cell population.

About Orna Therapeutics

Orna Therapeutics focuses on engineering new forms of circular RNA (oRNA®) therapeutics, aiming to enhance RNA medicine’s capabilities in treating various diseases. The company’s oRNA transcripts offer benefits over traditional mRNA approaches, including more straightforward production and improved delivery efficacy. With strong potential to innovate RNA medicines, Orna Therapeutics positions itself at the forefront of transforming patient care.

The content above is a summary. For more details, see the source article.