Meet one of our 2025 Pilot Grant Recipients, Juan Manuel Fernandez, PhD! Dr. Juan Manuel Fernandez is an experienced researcher with over 15 years of expertise in myotonic dystrophy (DM) research. His extensive knowledge spans key areas, including molecular pathogenesis, biomedical model development, identification of novel therapeutic targets, and drug discovery for myotonic dystrophy. In recent years, his contributions have significantly advanced the understanding of the role of microRNAs (miRNAs) in DM1, muscle atrophy, and autophagy. Notably, he has spearheaded the development of therapeutic approaches based on miRNAs, culminating in the ArthemiR™ clinical trial, now in Phase I/IIa.

He has led the innovative development of functional 3D skeletal muscle models derived from patient cells for various muscular diseases. These models include the first-ever 3D bioengineered model for myotonic dystrophy, which accurately replicates patient-specific muscle weakness and myotonia. This model is currently used in his laboratory to test potential therapeutic candidates for the disease.

In the Biomimetic Muscle Models for in vitro functional analysis and drug assessment in DM2 (BMM-2) project, the applicant aims to leverage his extensive expertise in myotonic dystrophy and tissue engineering to develop a novel model for myotonic dystrophy type 2 (DM2). This effort aligns with his longstanding commitment to advancing research in this field and addresses the critical need for robust DM2 models to facilitate drug development.

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