Imagine your bone marrow is a factory that forgot how to make healthy red blood cells. This project creates a tiny delivery vehicle that carries a set of instructions to the factory to fix the production line. Instead of changing your DNA forever, it uses a temporary message to get the cells working correctly again.
Current treatments for congenital anemias like beta-thalassemia are often inefficient, carry heavy side effects, and place a significant financial and physical burden on healthcare systems.
A bone marrow-targeted nanomedicine using lipid nanoparticles to deliver therapeutic mRNA to hematopoietic stem and progenitor cells.
If you are a drug developer dealing with the high cost and risk of DNA-based gene therapies — this project developed a non-integrating mRNA delivery system that offers higher intrinsic safety and lower development costs.
If you are a biotech company dealing with off-target effects in systemic drug delivery — this project developed bone marrow-targeted lipid nanoparticles (LNP) that ensure the medicine reaches the specific site of blood cell production.
If you are a clinic dealing with the burden of lifelong blood transfusions for beta-thalassemia patients — this project developed a therapeutic formulation aimed at restoring normal blood cell production to improve patient quality of life.
Based on available project data, mRNA-based therapies offer lower development costs and less stringent regulatory requirements for clinical approval compared to DNA-based gene therapies.
Based on available project data, the project includes a biotech partner, Mercurna, specializing in mRNA and cell-targeting, though specific industrial scale-up metrics are not provided.
The project develops a first-of-its-kind nanomedicine and a platform for targeted non-viral polynucleotide delivery that can be exploited for next-generation cell and gene therapies.
Based on available project data, mRNA therapies are expected to have less stringent regulatory requirements for clinical approval than DNA-based alternatives.
The project runs from 2023-10-01 to 2027-02-28, with a 3-year goal to deliver proof of concept in human and animal models.
The consortium is a lean 4-partner group across 3 countries (ES, FR, NL). It features a balanced mix of 2 research institutions and 1 SME (Mercurna), resulting in a 25% industry ratio. This structure combines academic expertise in anemia with specialized biotech capabilities in mRNA and lipid nanoparticle delivery.
Contact the Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia
Contact us to explore licensing opportunities for the bone marrow-targeted LNP platform.
