Societal need our project is addressing
Anemia is a condition characterized by insufficient healthy red blood cells (RBCs or erythrocytes), that affects around every fourth person worldwide. It constitutes a significant economic and societal issue as it is associated with increased morbidity and mortality, poor development in children, and by decreased work productivity. While most studies focus on iron-deficiency anemia, which is especially prevalent in low- and middle-income countries, around 40-50% of anemia is not due to iron deficiency. This anemia can be the main symptom of rare congenital diseases such as Diamond-Blackfan Anemia (DBA), β-thalassemia or Fanconi Anemia (FA). Congenital anemia constitutes a group of inherited diseases affecting the bone marrow leading to disturbed production of blood cells.Despite the high societal and economic impact of these rare diseases, there is no curative treatment available. Generally, treatments range from small molecules that boost RBC production to blood transfusions, depending on the severity of the anemia. These treatments are both limited in efficacy (duration) and/or associated with (severe) side-effects. Thus, there is a significant unmet medical need for effective intervention that reduces the need for blood transfusion in these patients and improve their quality of life.
Added value of NANEMIAR
In the past decades, gene therapy has shown promising results in the treatment of human diseases. Yet, the hurdles to clinical implementation are high. Therapy development is costly and time-consuming, and long-term safety and efficacy have yet to be established.With the recent market approval of the COVID-19 mRNA vaccines, we witnessed the emergence of a nanotechnology that can overcome these limitations and has the potential to revolutionize the field. NANEMIAR sets out to develop a ground-breaking bone marrow-targeted mRNA formulation applicable to most non-iron anemias and provide proof of concept in β-thalassemia.