In Vivo CAR-T Platform Achieves Proof-of-Concept, Reprograms 40–60% of Circulating T Cells

▎Edited by the WuXi AppTec content team

Umoja Biopharma, Inc. announced that the companyVivoVec Technology Platform for In Vivo Generation of CAR-T Cell Therapy Effectively and Durably Generates CAR-T Cells with Good Tolerability in a Non-Human Primate (NHP) Study.

CAR-T cells have demonstrated astonishing efficacy in treating various blood cancers, bringing hope of a cure to patients. However, the current manufacturing method for CAR-T cell therapy is complex, requiring the extraction of T cells from the patient, genetic engineering modifications outside the body, and then reinfusion into the patient. The entire process is time-consuming and labor-intensive, being one of the significant reasons limiting the accessibility of CAR-T therapy.

Umoja BiopharmaVivoVec Technology Platform Generates CAR-T Cells Targeting Tumors Directly in Patients Through Third-Generation Lentiviral Vector Technology Called VivoVec Particles. This "Off-the-Shelf" Technology Eliminates the Complex Processes Associated with Ex Vivo Manufacture of CAR-T Cell Therapy, Potentially Improving the Accessibility of CAR-T Cell Therapy.

The data released this time include the results of the first four NHPs treated in the ongoing study, which showWithout pre-conditioning lymphodepleting chemotherapy, CD20-targeted CAR-T cells were rapidly and effectively generated in vivo following a single infusion of Umoja's VivoVec particles. Moreover, the data indicate that the CAR-T cells produced in vivo exhibit targeted activity and persistent T-cell memory.

Key results include:

After a single administration of VivoVec, CAR-T cells targeting CD20 were generated in vivo, with the peak level of CAR-T cell expansion occurring between days 7 and 10.

CAR-T cells targeting CD20 exhibit targeted activity, leading to persistent B-cell aplastic anemia.。

In three animals that received the planned clinical dose and did not receive pre-treatment chemotherapy,The peak of CD20 CAR-positive cells accounts for approximately 40-60% of the total circulating T cells., including the confirmed central memory T-cell response.

In the first animal, additional independent CAR-T cell expansion was observed after Day 30.

No toxicity related to VivoVec administration was observed.