Define a lead candidate for clinical development of a novel T cell therapy

Introduction

Acute myeloid leukemia (AML) is a disease with a poor prognosis, especially for patients who either fail initial treatment or relapse thereafter. In AML, specific immune cells such as T cells have been proven to bear curative potential in the context of stem cell transplantation, but toxicity and therapy failure are frequent.

CAR T Cell Development

The use of T cells modified for specificity with chimeric antigen receptors (CAR) has changed the landscape of several blood-borne cancers of the B cell lineage. In AML, however, ongoing development of CAR T cells is associated with severe toxicities, limiting dosing and efficacy due to the poor specificity of the targets used for AML blasts.

Research Findings

In my ERC funded research, I have identified unique AML targets that are not shared with healthy hematopoiesis or other relevant healthy organs. By developing CAR against these targets, I could demonstrate the potential of my strategy.

Project Goals

To enable clinical development and exploitation, T2LEAD will:

1. Identify and develop lead CAR candidates for further translation.
2. Explore the medical and commercial potential of lead CAR.
3. Route exploitation towards licensing, spin-out, or both.

Support and Impact

T2LEAD will be supported by a strong network of advisors from academia, industry, and regulatory frameworks. Ultimately, T2LEAD will help advance the treatment landscape in AML to the benefit of AML patients.