21 October 2024

Development of a Targeted Ex-Vivo Gene Therapy Using Allogeneic Effector Lymphocytes from Cord Blood for the Treatment of Hematopoietic Malignancies in the Polish Population.

Project Title: Development of a Targeted Ex-Vivo Gene Therapy Using Allogeneic Effector Lymphocytes from Cord Blood for the Treatment of Hematopoietic Malignancies in the Polish Population.

Project: Funded by the Medical Research Agency with state budget funds.
Call: Development of Targeted or Personalized Medicine Based on Nucleic Acid or Small Molecule Therapies (Call Number: ABM/2022/6).

 

Project Objective:
"Gene therapies that modify patient effector lymphocytes using CAR receptors targeting the CD19 antigen have been a breakthrough in the treatment of B-cell malignancies of the hematopoietic and lymphatic systems. Autologous CAR products are characterized by high clinical response efficacy and long-term disease remission. CAR therapies are established and reimbursed treatment standards, particularly for patients with diseases resistant to traditional chemotherapy or radiotherapy and prone to relapse. However, the cost of therapy and the time required to produce an individualized
product for each patient, combined with the severe condition of patients after two lines of treatment, are barriers to the widespread use of these methods for all patients in need.
To address these challenges, allogeneic solutions have been proposed. These can be developed at scale in advance, based on a universal donor model. However, due to multiple genetic modifications, intensive expansion, and the necessary differentiation processes, these therapies raise justified safety concerns among regulatory agencies. In response to this unmet medical need, the proposed project presents an ex vivo gene therapy using a CAR construct expressed in a cord blood cell population with desired characteristics of allogeneic products (availability, scalability, and rapid treatment initiation) while ensuring safety. The CAR
product bank created in this way will provide 'off-the-shelf' CAR therapy access for almost the entire Polish population. Patients with B-cell malignancies are eligible for CAR therapy. Ultimately, the scope of allogeneic CAR products produced will encompass all potential
patients."

 

Grant Amount: PLN 20,802,247.86

Total Project Value: PLN 44,541,120.72