Nanomedicine Approach to Normalize Erythrocyte Maturation in Congenital Anemia by messenger RNA

Messenger RNA (mRNA) has recently proven itself as a prophylactic modality that can be rapidly developed and employed with high efficacy and low toxicity.

Compared to DNA-based gene therapy, mRNA has significant advantages including higher intrinsic safety (non-integrating), lower cost of development, and less stringent regulatory requirements for clinical approval.

In the NANEMIAR project, we take advantage of these benefits to kill 2 birds with 1 stone: first, we develop a first-of-its-kind nanomedicine for congenital anemia, a group of rare, inherited blood disorders characterized by ineffective red blood cell production.

Our proposed bone marrow-targeted mRNA-based therapy contributes to the first Horizon Europe strategic plan (2021-2024) for new and advanced therapies for non-communicable diseases and is expected to be a vast improvement over current treatment options in terms of efficacy and safety.

Second, we provide critical knowledge on targeted non-viral polynucleotide delivery, with a platform that can be exploited for next-generation cell and gene therapy in a broader context.

To achieve this, our consortium combines the unique cell-targeting and mRNA knowledge from the Dutch Biotech Mercurna with two excellent research partners (from Spain and France) who have demonstrated expertise in the field of anemia.

By focusing on the well-recognized rare disease model, beta-thalassemia, and combining an established mouse model, patient material, and previous drug development experience we build a convincing case to treat congenital anemia.