#United States #San Diego #Orna Therapeutics #panCAR™ #RNA Delivery

Innovations in RNA Therapy: Orna Therapeutics at ASH 2024

At the 66th Annual Meeting of the American Society of Hematology (ASH) held in San Diego, Orna Therapeutics presented groundbreaking findings that could transform treatment for B cell-mediated diseases. Their innovative panCAR™ approach promises to offer a scalable and transient immune cell therapy without the necessity for lymphodepletion, a repetitive and often harsh procedure traditionally associated with CAR therapies.

Orna's new circular RNA medicines, delivered through leading-edge lipid nanoparticle (LNP) technology, aim to tackle various oncological and autoimmune challenges. Data showcased revealed that a single administration of their anti-CD20 panCAR™ markedly reduced B cell levels in non-human primates (NHPs) by an impressive 95%, with sustained effects lasting a week after dosing. This data signals a critical advancement in how we might manage B cell-dependent conditions, according to company officials.

The effectiveness of Orna's LNP candidates was notably impressive; they achieved the delivery of RNA to 60% of splenic T cells and 84% of T cells in peripheral blood during preclinical trials. Furthermore, the combination of LNPs with oRNA cargo demonstrated approximately 70% success in transferring an anti-CD20 CAR to T cells from both NHPs and humans, maintaining expression for over 72 hours. Such efficacy could potentially revolutionize patient treatment regimens, removing prolonged and uncomfortable preparation processes commonly seen today.

Dr. Joe Bolen, Chief Scientific Officer at Orna Therapeutics, expressed optimism about the findings, stating, “Our panCAR™ strategy, which integrates our synthetic circular RNA platform with proprietary LNP technology, positions us to redefine the landscape of CAR therapies. The data presented at ASH reinforce our hypothesis regarding LNP efficacy and point towards promising in vivo applications for addressing complex diseases.”

The findings didn't end there; additional assays demonstrated that both anti-CD20 and anti-CD19 CAR constructs exhibit significant cytotoxicity against human B cell lines, marking 75-80% reductions across peripheral blood, spleen, and bone marrow samples within just 24 hours of treatment. Impressively, this depletion persisted even after 7 days, showcasing the potential longevity of the therapeutic impact.

In summary, Orna Therapeutics’ presentations at ASH 2024 represent an exciting leap forward in the field of RNA-based therapies, particularly in treating diseases influenced by B cells. As the company continues to advance its unique therapeutic strategies, it opens new avenues for clinical applications, potentially bringing relief to countless patients in the future.

As a pioneer in the design and delivery of circular RNA therapeutic solutions, Orna is swiftly gaining a reputation for its groundbreaking LNP technologies and editing methods, which are hailed as necessary innovations within the industry. With grand visions of transforming patient care through RNA medicine, the future looks brighter than ever for Orna Therapeutics.

About Orna Therapeutics

Orna Therapeutics focuses on engineering circular RNA (oRNA®) therapeutics, aimed at unlocking the full potential of RNA medicine across various disease modalities. Their unique approach addresses many manufacturing challenges associated with traditional mRNA therapies, thus advancing novel treatment possibilities. For more details, follow Orna on social media platforms or visit their official website.