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Expression Therapeutics Receives NCI SBIR Grant for ET501

Expression Therapeutics, Inc., a company focused on clinical-stage gene and cell therapies, announced a significant advancement in cancer treatment for children. The National Cancer Institute (NCI) has granted the company a Phase II award worth $2,058,885 to further the development of ET501, an investigational γδ T-cell therapy specifically targeting PTK7 for pediatric patients diagnosed with high-risk neuroblastoma. This funding will facilitate activities leading to an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA).

Neuroblastoma is a serious pediatric cancer affecting the sympathetic nervous system, with over 650 cases diagnosed annually in the United States at a median age of 17 months. Current five-year event-free survival rates hover around 51.2%, reflecting the dire need for more effective treatments for high-risk patients. The award from NCI aims to bridge the gap between ET501's current stage and the eventual submission of an IND.

ET501 utilizes the innovative ADEPT-STAR platform by Expression Therapeutics, which employs the LENTeT lentiviral delivery system to engineer γδ T-cells. These engineered cells are designed to secrete a T-cell engager directly at the tumor site, enhancing the immune response against cancer cells effectively. In particular, ET501 targets PTK7, a protein present on neuroblastoma cells, alongside interacting with CD3 on T-cells, thereby rallying both infused and pre-existing immune cells to fight the cancer.

David Townson, Ph.D., CEO of Expression Therapeutics, emphasized the critical need for improved treatment options for these vulnerable children, stating, "Children with high-risk neuroblastoma need treatment options beyond today's regimens. This award funds the work that stands between ET501 and an IND application."

Additionally, Brian Petrich, Ph.D., Vice President of Research and Development and the contact principal investigator on this grant, discussed the innovative aspects of ET501: "This project is crafted to enable γδ T cells to produce a PTK7-directed engager inside the tumor. This permits the immune system to utilize existing immune cells effectively, maximizing the therapeutic response."

Although ET501 is in the preclinical stage and has not yet been tested in humans, the company's eventual goal is to receive regulatory approval to remedy the current treatment landscape for pediatric neuroblastoma patients.

Expression Therapeutics will conduct studies funded by this grant to validate the efficacy of ET501 and whether it can lead to the submission of an IND. The company is also conducting advanced studies on another program within its γδ T-cell platform, aimed at treating adults afflicted by neuroendocrine tumors.

About Expression Therapeutics

Founded in Tucker, Georgia, Expression Therapeutics is committed to developing cutting-edge gene and cell therapies. The company’s lead program involves ET3, an autologous hematopoietic stem cell gene therapy for hemophilia A, which has already completed Phase 1 trials. Expression is also at the forefront of γδ T-cell therapies for both pediatric neuroblastoma (ET501) and adult neuroendocrine tumors (ET401), showcasing their diverse technological innovations such as ADEPT-STAR and LENTeT. They operate a state-of-the-art cGMP cell and viral vector manufacturing facility in Cincinnati, Ohio, propelling their vision for breakthrough treatments forward.

Research under this grant is acknowledged as being supported by NCI under Award Number R44CA281633, with outcomes reflecting the views of the authors, not necessarily mirroring those of the National Institutes of Health.

In conclusion, the recent funding signifies a pivotal step toward transforming treatment options for children suffering from high-risk neuroblastoma. Should the studies yield positive results, ET501 could provide a groundbreaking therapeutic option that enhances survival rates and overall outcomes for these young patients.