Presented on June 9th, 2021.
Each month biotechnology, pharmaceutical and academic partners, large and small, working on treatments and a cure for myotonic dystrophy will sit down with our community to share their progress and answer your questions. Click here to learn more about ARTHEx biotech.
About the Presenters
Dr. Beatriz Llamusi
Msc and PhD in Biochemistry, CEO of Arthex Biotech S.L
Dr.Llamusi focused her scientific career on studying the molecular basis of pathogenesis and potential treatments by generating animal models of human diseases. As a postdoc she moved her focus from neuro-regeneration in spinal cord, to rare diseases, specifically Myotonic Dystrophy disease (DM), working under the supervision of Prof. Dr. Artero, in the laboratory of Translational Genomics affiliated to the University of Valencia and the INCLIVA health research institute. She contributed more than 20 scientific articles in indexed journals, including original research and reviews on the state of the art in Myotonic Dystrophy. Her main research interest is to understand myotonic dystrophy neurodegeneration, heart dysfunction, and muscle atrophy, to investigate their molecular basis and to find drugs that suppress these phenotypes. Her motivation is the translation of her research to benefit society and, as such, she discovered five anti-DM1 compounds or repurposing drugs, which are protected by patents and licensed to biotech companies.
Currently, she founded the biotech company ARTHEX Biotech S.L, in September 2019, dedicated to the development of an antimiR-based drug against Myotonic dystrophy disease. The company raised seed funds from Invivo Capital and ADVENT France VCs and is actually working on the preclinical development of these molecules, planning to enter FIH on 2022.
Dr. Rubén Artero
Msc and PhD in Biochemistry, full professor of Genetics at the University of Valencia.
Leader of the translational genomics lab in the UV. His laboratory is focused on the molecular basis of muscle atrophy in myotonic dystrophy and in identifying appropriate therapeutic targets and candidate drugs using cell, animal and bioinformatic approaches. During his doctoral degree, he discovered the muscleblind gene, the founding member of the MBNL family of proteins. He received six years of postdoctoral training at the Memorial Sloan-Kettering Cancer Center in New York studying Drosophila myogenesis and is leading the Translational genomics lab since 2005. He contributed more than 50 research articles related to Myotonic Dystrophy disease mechanisms, and pioneeres the research on MBNL function and regulation. He produced more than 10 patents with his research in the field and acted as advisor for different companies. Currently, he co-founded Arthex Biotech S.L and is a member of its scientific advisory board.
Dr. Nicholas E. Johnson
MD, MSCI, FAAN at Virginia Commonwealth University
Dr. Johnson is an associate professor of Neurology and Human and Molecular Genetics and vice chair of research in Neurology at Virginia Commonwealth University with a focus in inherited neuromuscular disorders. He received his undergraduate degree in molecular and cellular biology and psychology at the University of Arizona. He then obtained his medical degree at the University of Arizona. He completed his neurology residency and combined fellowship in neuromuscular medicine and experimental therapeutics at the University of Rochester.
His laboratory is focused on identifying the pathogenesis of limb girdle muscular dystrophy, myotonic dystrophy, and facioscapulohumeral muscular dystrophy and identifying appropriate clinical endpoints for these conditions. Johnson conducts therapeutic trials in many other inherited nerve and muscle disorders.