SCIENCE
Transforming T-cell Immunotherapy with AAV Gene Therapy
T-cell immunotherapies like CAR-T represent a powerful cancer-fighting approach that redirect T cells to recognize and kill cancer cells. While they’ve transformed the treatment of certain leukemias and lymphomas, T-cell immunotherapies have multiple drawbacks that have challenged their safety, efficacy, durability, practicality, and patient/indication reach.
With our TransJoin™ AAV Gene Therapy Platform, we aim to redefine the T-cell immunotherapy field and transform treatment outcomes for patients.
Transjoin’s Novel
Approach to Fight Cancer
We are leveraging our patented, novel TransJoin AAV Gene Therapy Platform, to create off-the-shelf, single-dose T-cell immuno-gene therapies to bring the power of T-cell-mediated tumor cell killing to more cancer patients.
Our proprietary TransJoin technology is designed to enable the expression of a secreted T cell engager that binds the tumor cell on one side (changeable depending on the product candidate indication target) and T cells on the other side.
Following a single, one-time intravenous infusion, the TransJoin technology instructs the liver to continuously secrete the bispecific protein into the bloodstream to redirect T cells to tumor cells. TransJoin provides a bridge that joins T cells and tumor cells, resulting in long-term, consistent serum levels of the therapy and, thus, long-term, consistent T-cell-mediated tumor cell killing. TransJoin’s extremely low-dose AAV delivery minimizes toxicity and adverse events.
The following table lays out how TransJoin’s potential stacks up against other available CD19-targeted immunotherapy classes, including CAR-T (chimeric antigen receptor T cell therapy).
| Differentiators of CD19-Targeted Therapies | CAR-T | TransJoin |
|---|---|---|
| Off-the-shelf | No | Yes |
| Single dose | Yes | Yes |
| Long-term efficacy (months to years) | Yes | Yes |
| Involves all T cells | No | Yes |
| Recruits freshly made T cells from bone marrow | No | Yes |
| Highly efficient T cell signaling (via the mature T cell receptor) | No | Yes |
| Risk of manufacturing failure | Yes | No |
| Requires chemotherapy conditioning | Yes | No |
| Risk of cytokine release syndrome | Yes (High) | No |
| Risk of neuro-toxicity | Yes (High) | No |
The Landmark Published Research Behind Vironexis
The foundational technology for our TransJoin platform was licensed from Nationwide Children’s Hospital.
In 2022, research led by Timothy Cripe, M.D., Ph.D., Chief of the Division of Pediatric Hematology/Oncology/Bone and Marrow Transplant, describing the Transjoin technology was published in Scientific Advances. Dr. Cripe is one of Vironexis’s co-founders.
Bringing TransJoin to Patients
We’re working as quickly as possible to translate the promise of AAV-delivered T-cell Immunotherapy for patients in need. We’ve built a pipeline of 10+ product candidates for blood-based cancers, solid tumor metastasis prevention, and cancer prevention through a vaccine.
Learn More About Our Pipeline