Our focus is on indications with well understood biology, predictive biomarkers for early proof-of-concept, efficient clinical development pathways, and high unmet medical need.
We are pioneering in vivo CAR-T therapies by delivering RNA-based treatments that reprogram immune cells (e.g., T cells) within the body to express chimeric antigen receptors (CARs) and other immunomodulatory proteins. Leveraging our RNA platform, we aim to enhance antigen recognition and modulate immune responses with precision. Our approach is designed to enable targeted delivery of RNA to immune cells, offering a potentially more effective and scalable solution for treating autoimmune diseases.
We are advancing our lead RNA immunotherapy candidate, OTX-201, a potential best-in-class in vivo CAR-T therapy for B cell-driven autoimmune diseases, where the therapeutic goal is to deplete autoreactive B cells and reset the immune system. B cell-driven autoimmune diseases span more than 40 disease indications across multiple therapeutic areas, including rheumatology, neurology, and dermatology.
OTX-201 comprises an optimized circular RNA encoding a CD19-targeted CAR delivered via targeted lipid nanoparticles (LNPs) with in vivo administration. This in vivo approach, in which the patient’s own body serves as the manufacturer of CAR-T cells, has the potential to offer a reduced treatment burden and improved accessibility compared to ex vivo CAR-T therapies, which require patient cell collection and complex manufacturing processes followed by intensive conditioning regimens prior to infusion.
Orbital is advancing OTX-201 through IND-enabling studies and plans to begin clinical development in the first half of 2026.
We are in a position to leverage in vivo
We have the ability to develop next-generation RNA vaccines that improve immune responses to protect and treat diseases. These capabilities include heterologous prime-boost vaccines to simplify dosing strategies, combination vaccines to recognize and neutralize multiple antigens, and polyfunctional vaccines combining antigens with adjuvants and/or other immune enhancers to promote greater efficacy.
We envision a future where protein therapeutics such as antibodies, Fc fusion proteins, enzymes, peptides, cytokines, and chemokines can be delivered through RNA medicines. This approach holds promise for treating genetic protein deficiencies, metabolic diseases, and other diseases requiring complex biologics. Our platform is designed to overcome key challenges in the field, including expression durability, redosing capability, protein size and complexity, and targeted delivery beyond the liver. Through advanced payload engineering and delivery innovation, we are unlocking the full potential of RNA as a modality for protein-based therapeutics.
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