INSERM: A key partner in the DREAMS project
The DREAMS project (Drug Repurposing with Artificial Intelligence for Muscular disorders) aims to discover and validate shared therapies for multiple rare neuromuscular disorders (NMDs), for which there are currently no effective treatments. Among the nine partners from six countries, the French National Institute of Health and Medical Research (INSERM) plays a central role in advancing the project’s scientific and translational goals.
With a strong legacy in biomedical research and a mission to improve human health, INSERM brings decades of expertise to DREAMS. Its contribution is particularly significant in the identification of shared molecular mechanisms across genetically distinct NMDs, supporting the project’s cross-disease strategy for drug repurposing.
INSERM: Scientific leadership in health research
Founded in 1964, INSERM is a public scientific and technological institute under the joint authority of the French Ministries of Health and Research. Its activities span from laboratory research to patient care, with a focus on translating scientific discoveries into tangible health benefits. INSERM collaborates with leading institutions worldwide and covers a wide range of disciplines, including genetics, immunology, neuroscience, and epidemiology.
In DREAMS, INSERM contributes to the development and application of innovative tools and methodologies aimed at identifying and characterizing shared molecular and pathophysiological features of selected neuromuscular disorders. This includes a focus on autophagy dysfunction and desmin-related cytoskeletal abnormalities—two recurrent features across the targeted diseases.
Dr. Antoine Muchir: 25 years of expertise in muscle biology
INSERM’s involvement in DREAMS is led by Dr. Antoine Muchir, who has dedicated the past 25 years to studying the nuclear lamina and nuclear envelope, particularly in the context of genetic cardiomyopathies. His research began in 1999 in the laboratory of Dr. Ketty Schwartz, who first established the link between LMNA gene mutations and muscular dystrophies. He later joined the laboratory of Professor Howard Worman at Columbia University, who originally cloned the LMNA gene.
Dr. Muchir has developed deep expertise in muscle biology, with a focus on preclinical models of Emery-Dreifuss muscular dystrophy. His work has significantly contributed to the field, including the initiation of the first clinical trial in patients with this condition. He currently leads the research team “Signaling Pathways and Striated Muscle” within the Center of Myology, focusing on Emery-Dreifuss and Duchenne muscular dystrophies.
His academic record includes 45 peer-reviewed publications, two patents, and an H-index of 35. He has led numerous national and international research initiatives supported by the French National Research Agency (ANR), Horizon Europe, and Fondation Leducq.
INSERM’s role in DREAMS
Within DREAMS, INSERM is responsible for coordinating and executing specific work packages (WPs) that align with its expertise in muscle biology and translational research. The project’s approach targets NMDs that are genetically distinct but share common disease mechanisms. By focusing on these shared features, DREAMS aims to accelerate therapeutic discovery and drug repurposing.
INSERM’s contribution supports the identification of drug candidates that modulate shared molecular pathways. This is achieved through AI-driven data analysis, high-throughput compound screening, and systems biology. The goal is to develop broad-spectrum therapeutic strategies that can be applied across multiple NMDs.
A collaborative and translational approach
DREAMS is committed to engaging a wide range of stakeholders—researchers, clinicians, patients, advocacy groups, biotech and pharmaceutical companies, and regulatory agencies. INSERM’s leadership in translational medicine and collaborative research ensures that the project remains aligned with patient needs and scientific rigor.
The knowledge and tools generated by DREAMS, with INSERM’s contribution, aim to shift the current paradigm in rare disease drug development. Instead of focusing on single-disease models, the project proposes a mechanism-centered framework that can be applied across conditions with similar biological underpinnings.
Conclusion
INSERM’s role in DREAMS exemplifies the power of scientific excellence and collaboration in addressing unmet medical needs. By contributing its expertise in drug repurposing for neuromuscular diseases, INSERM is helping to pave the way for more efficient, inclusive, and impactful therapeutic development for rare disorders.
