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Cancer Program


First Indication: Multiple myeloma (MM)


Multiple myeloma (MM) is the second most common hematologic malignancy worldwide, representing approximately 1% of all malignancies, with an incidence estimated to be 6 cases per 100,000 persons per year. With the 34,000 patients diagnosed with MM in the US in 2022, more than 12,000 would succumb to this blood disorder   . Despite many new drugs approved in the last decade, the overall 5-year survival rate for people with multiple myeloma in the United States is 55%      .


RORA first-in-class Bispecific anti-BCMA-CS1 CAR-Tscm cell therapy for RRMM


In recent years, BCMA (B-cell maturation antigen) CAR-T cell immunotherapies have shown unprecedented success in the treatment of some refractory MM   . However, a substantial fraction of patients encountered cancer relapse after treatment with BCMA CAR-T cells.  One of the unmet needs is to provide durable cancer remission in the treatment of relapsed and refractory MM who have failed multiple lines of treatment (RRMM).  


CS1 (also known as CD319, CRACC and SLAMF7) is overexpressed in multiple myeloma. The RORA anti-BCMA-CS1 CAR-Tscm differs significantly from the conventional monospecific anti-BCMA CAR-T design. While the monospecific anti-BCMA CAR only recognizes the BCMA molecules on MM cells, the bi-specific CAR recognizes two different cancer biomarkers on MM cells, namely BCMA and CS1. RORA Tscm cells are first collected from patient’s blood and expanded with our proprietary manufacturing method. The anti- BCMA-CS1 CAR is engineered onto the cell surface using a viral vector under GMP manufacturing conditions. The resulting anti-BCMA-CS1 CAR-T’s are infused back to the patient. Once inside the body, these modified Tscm cells expand into effector T cells that are capable of targeting BCMA and CS1 molecules on the myeloma cells and eventually kill the cancer cells.


Advantages of the RORA Bispecific anti-BCMA-CS1 CAR-Tscm


There are major limitations to the conventional monospecific anti-BCMA CAR-T therapy that still must be addressed including non-durable cancer efficacy, tumor escape, life-threatening CAR-T cell-associated toxicities, and high manufacturing costs. The RORA anti-BCMA-CS1 CAR-Tscm is designed to resolve many of these limitations and are expected to provide: 1) more durable cancer efficacy, 2) better safeguards against tumor escape, 3) lower toxicities associated with many other CAR-T therapies and 4) lower production costs.


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