Therorna to Showcase Clinical-Ready Circular RNA in Vivo CAR-T and CircRNA Pipeline at the 2026 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting

Key Takeaways

• TI-0032 entered first‑in‑human trial for autoimmune diseases
• Dual US/China IND filing keeps regulatory path on schedule
• Preclinical data shows complete B‑cell depletion at low dose
• CircMab platform enables in‑body CD19×CD3 engager production
• TI‑0093 HPV16 vaccine earned first circRNA oncology IND globally

Pulse Analysis

Circular RNA has emerged as a versatile vehicle for delivering therapeutic proteins without the integration risks associated with DNA or the instability of linear mRNA. Therorna, a Beijing‑Shanghai biotech founded in 2021, has built a splint‑free enzymatic circularization process coupled with a targeted lipid nanoparticle (tLNP) system to direct expression to T cells. By leveraging this platform, the company aims to overcome the manufacturing bottlenecks of conventional CAR‑T, which require patient‑specific cell processing, and to offer a scalable, off‑the‑shelf solution for high‑need indications.

The flagship candidate, TI‑0032, encodes a CD19 CAR on a circRNA payload and is delivered intravenously via tLNPs. Preclinical data presented at ASGCT showed durable CAR expression for at least two weeks in primary human T cells, >90 % CD8⁺ selectivity, and complete B‑cell depletion in humanized mice at a dose of 0.02 mg/kg. In non‑human primates, a single administration eliminated B cells across blood, spleen, bone marrow and lymph nodes, suggesting a potent, low‑dose regimen. The first‑in‑human investigator‑initiated trial now targets patients with refractory autoimmune disease, marking the first clinical test of an in‑vivo circRNA CAR‑T.

Therorna’s pipeline extends beyond CAR‑T with the CircMab platform, which programs the body to produce a CD19×CD3 bispecific engager, and the CircVac platform, exemplified by TI‑0093, the first circRNA‑based HPV16 therapeutic vaccine to receive an IND in oncology. A dual US‑China IND filing for TI‑0032 keeps regulatory timelines aligned across the two largest markets, potentially accelerating global rollout. If clinical efficacy mirrors preclinical potency, Therorna could reshape the immunotherapy landscape by delivering off‑the‑shelf, re‑dosable products that reduce cost, simplify logistics, and broaden patient access.