Closing the European gap towards a large scale ex vivo platelet production built upon a silk-based scaffold bioreactor

Introduction

The generation of ex vivo functional megakaryocytes (MK) and platelets is questioning current transfusion medicine's exclusive reliance on blood donors to produce platelets. The demand for platelets is, in fact, in continuous rise, driven by an aging population, advances in medical procedures, and even more aggressive cancer therapies, although the supply of blood donors continues to remain stagnant.

Ex Vivo Platelet Production Systems

Ex vivo platelet production systems have emerged as a feasible supply that can secure universal compatibility to avoid platelet refractoriness. If during the FET Open application we have proved that the platelet production ex vivo can be precision-tailored with the correct physical and biochemical environment, now with SilkPlatelet, our consortium is looking forward to demonstrating the feasibility of an upscaled ex vivo production system.

Goals and Objectives

To bring our technology from TRL4 to TRL6, we will:

1. Increase the ex vivo platelet production capacity with media and stem cells aligned to GMP requirements for large-scale clinical transfusion.
2. Produce universal platelets particularly for patients with immune reactions to previous transfusions with HLA null platelets and patients with acute hemorrhage.

Technology and Methodology

At the core of our "from lab-to-patients" roadmap, we will utilize:

• UNICAM proprietary technology to differentiate stem cells into MK precursors (natural source of platelets).
• UNIPV disruptive bioreactor made of a silk scaffold to mimic the bone marrow conditions.

These will be complemented by:

• IGR's long-term experience in iPSC and MK biology.
• ISENET's cell quality control to contribute to the standardization of the platelet production pipeline and its validation and verification.

Conclusion

Under the guidance of our experienced tech transfer officers, by the end of the project, we expect to complete the transition into a commercial innovation exploited by a next university spin-out.