Logic Gated Precision Molecular Adaptor System for Juvenile Myelomonocytic Leukemia Treatment

About This Grant

Juvenile myelomonocytic leukemia (JMML) is a rare and high-mortality hematologic malignancy affecting young children. Currently, the primary treatment for JMML is allogeneic hematopoietic stem cell transplantation (HSCT); however, relapse remains a significant risk following transplant. In the Concept phase of our research, Gencyte established the synNotch-controlled PMA CAR platform and generated preliminary in vitro proof-of-concept data for JMML treatment. In Phase I, the company will conduct in vivo assays using immunocompromised mice to evaluate synNotch-controlled PMA CAR-T cells. The company also plans to optimize leads to reduce HAMA (human anti-mouse antibody) responses, improving safety for human application. Then in vitro and in vivo functional assays will be conducted on the optimized leads. Preclinical development will follow, focusing on producing GLP/GMP-grade materials and completing IND-enabling studies to support an IND application.