This website uses cookies. By continuing to browse the site you are agreeing to our use of cookies. Further information

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.

NANEMIAR contribution to EU priorities

Our approach and vision are completely aligned with the priorities of the Horizon Europe Strategic Plan with expected impacts in Cluster 1 Health, by delivering a technological breakthrough with future potential for destination 3 tackling diseases and reducing disease burden. Through the development of innovative therapeutic approaches, the project will specifically contribute to more effective, cost-efficient and affordable treatment for patients with a rare disease.

Expected impact of our project

Scientific Impact

New breakthrough scientific knowledge on mRNA therapies
Strengthtening R&I on congenial anemia
Promoting knowledge transfer and Open Science

Societal Impact

Addressing needs for new therapeutic options that ultimately increase patients' ability to work/participate in society
Strengthtening awarenss and uptake of mRNA appraches in society

Economic Impact

Promoting innovation-based growth in academia and industry
Leveraging R&I investment with potential for reduced development and healthcare costs

Research Centres

Research Group Spanish Node
Research Group French Node
Research Group Dutch Node