Dr. Christophe Lachaud, research Director at CNRS who is a beneficiary of the NANEMIAR Project, explains to us how hir team is contributing to NANEMIAR.

Hello, Dr. Lachaud. Thank you for joining us. Could you explain the role that CNRS and your team specifically play in the NANEMIAR project ?

"At CNRS, our role in the NANEMIAR project is centered on the in vivo studies that are crucial to understanding how the developed nanoparticles behave in a living organism. Specifically, our team focuses on assessing both the safety and effectiveness of these particles, evaluating their tolerance and toxicity levels in murine models. By conducting these in vivo tests, we aim to provide robust data that support further stages of development and ensure the nanoparticles meet essential safety standards before advancing to clinical applications."

What expertise does your team bring to the project, particularly in the study of anemia and hematopoiesis in murine models ?

"Our team brings a specialized background in hematology, particularly in studying normal and abnormal hematopoiesis, or blood formation, within murine models. This expertise allows us to carefully assess how nanoparticles affect red blood cell production and to model anemia disorders realistically. We also have extensive experience in working with ethical frameworks and procedures for animal studies, enabling us to perform the necessary in vivo validations in a responsible and impactful manner."

What are the main challenges in evaluating the tolerance and toxicity of nanoparticles in in vivo models, and how do you address these challenges ?

"One of the main challenges is ensuring that the nanoparticles are not only effective, but also safe when administered in a complex organism. Nanoparticles can interact with various tissues and organs in unexpected ways, so it’s critical to monitor for any adverse effects closely. We address these challenges by conducting detailed analyses of the biological responses in our murine models, measuring not only efficacy, but also signs of toxicity at multiple levels. Our lab is equipped with the latest tools for tracking these responses, allowing us to gather comprehensive data on how the body tolerates the nanoparticles over time."

How does working with complex murine models enhance the validation process for new therapeutic nanoparticles compared to cellular models ?

"Murine models provide a far more realistic environment than cellular cultures, allowing us to observe how nanoparticles interact within a living system, including how they are distributed, absorbed, and processed by different organs. This level of complexity is essential for validating a therapy’s safety and effectiveness in ways that cellular models simply cannot replicate. By using murine models, we’re able to simulate the therapeutic process much more accurately, gathering data that is crucial for translating findings into human applications down the line."

What are your hopes for the potential impact of NANEMIAR’s findings on the future of treatments for congenital anemia ?

"Our hope is that the findings from the NANEMIAR project will pave the way for groundbreaking therapies for congenital anemia, a condition with limited treatment options today. By successfully demonstrating a safe and targeted delivery method for mRNA-based treatments, we aim to improve the quality of life for patients who suffer from this challenging disorder. Furthermore, our work in NANEMIAR could open doors to similar therapies for other genetic conditions, extending the benefits of these innovations beyond congenital anemia."

Source of information: NANEMIAR project