Periodic Reporting for period 1 - T2Lead (Define a lead candidate for clinical development of a novel T cell therapy)
Reporting period: 2023-09-01 to 2025-02-28
Patients suffering from Acute myeloid leukemia (AML) have a dismal outcome when relapsing or progressing after initial therapy. Unlike other hematological malignancies, cellular therapies such as chimeric antigen receptor (CAR) engineered T cells could not demonstrate efficacy in this disease. A major cause of such lack of efficacy is the difficulty for CAR T cells so far to differentiate between healthy and cancer cells in AML patients, thus narrowing the therapeutic window of such therapies. In preliminary work, we could identify and demonstrate preclinical activity of novel AML-directed CAR T cells. The purpose of T2LEAD was now to identify, generate and test a CAR, which could then be further developped towards clinical testing. T2LEAD was successfull in identifying such promissing candidate, which we are currently continuing to develop and are seeking further funding thereto. Ultimately if successfull this concept could bring an innovative treatment to AML patients.
T2LEAD generated and tested new CAR against a proprietary target for AML. We successfully expressed these in primary human T cells and tested these in a variety of models. We could define a lead candidate for further development and testing, which is free of third party rights and would thus grant us freedom to operate. We have applied for further funding and defined a potential IP and business strategy for further development.
As outlined above, we could define a CAR lead candidate for further development. This is critical to be able to advance our strategy to further valorisation and clinical testing. Subsequent steps will include funding for regulatory development, safety requirements defined by regulators, pre-GMP-testing, process development, GMP upscale and funding for a clinical study. Depending whether this is managed in a spin out, a licence or directly from hospital, requirements will differ. Ultimately, our study may bring an innovative product to AML-patients in need.