Rare Disease Day: OneChain Immunotherapeutics receives €1.3m grant for CAR-T leukemia treatment

According to the company, this therapy could treat up to 80% of patients with T-cell acute lymphoblastic leukemia (T-ALL), a rare condition with few therapeutic options.

The treatment, based on CAR-T technology​, simultaneously targets two new therapeutic markers with minimal presence in healthy tissues, the company added.

The grant builds on the success of a previous therapy developed by OCI, which is currently being evaluated in a phase 1 clinical trial.

However, this molecule is present in cortical T-ALL (coT-ALL), a subset of T-ALL, representing only 30-40% of T-ALL patients.

"The first CAR T therapy we developed, targeting the CD1a antigen, is already in clinical trials, demonstrating its safety and efficacy for a specific group of patients," said Dr. Victor Díaz, research director at OneChain Immunotherapeutics.

"By incorporating a second target into our strategy, we not only increase the percentage of patients we could treat—up to 80%—but also enhance the effectiveness of the therapy."

The treatment of acute lymphoblastic leukemia type T, one of the most aggressive forms of leukemia, has historically relied on intensive chemotherapy.

Despite improvements in survival rates, this therapy results in devastating effects for patients, with a significant percentage not responding to treatment.

"For patients who have experienced a relapse, finding effective treatments is particularly challenging. Our aim is to bring hope to these patients and develop this therapy over the three years of the project to initiate a first clinical trial in humans,” added Díaz.

“We are in a unique position to make a real difference in the lives of patients, expanding our therapies to include those who previously had few options.”

The project, set to last three years, will involve collaboration with the Josep Carreras Institute and the Blood and Tissue Bank.