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May 9-12, 2022Hynes Convention Center, Boston

Beatriz Llamusi, Ph.D.
Chief Executive Officer & Co Founder at Arthex Biotech


I have focused my scientific career on studying the molecular basis of pathogenesis and potential treatments by generating animal models of human diseases. As a postdoc my focus moved from neuro-regeneration in spinal cord, to rare diseases, specifically Myotonic dystrophy (DM), working under the supervision of Prof. Dr. Artero, in the laboratory of Translational Genomics (GT) affiliated to the University of Valencia (UV) and the INCLIVA health research institute. I have contributed 21 scientific articles in indexed journals, including original research and reviews on the state of the art in Myotonic Dystrophy. My main research interest has been to understand myotonic dystrophy neurodegeneration, heart dysfunction, and muscle atrophy, to investigate their molecular basis and to find drugs that suppress these phenotypes. As principal investigator, I have been awarded four projects. One founded by RTVE telemaratón allowed the development of an active compound against DM that we previously discovered. In the second investigation (European collaborative E-rare), I gathered a quite unique experience in the study of Drosophila heart, successfully describing cardiac dysfunction in DM type 1 and 2, and the effects of different chemical and genetic modifiers. In 2017, I was awarded a ‘Health’ research project, by the Institute of Research Carlos III as Co-IP (PI17/00352), focused on the genetic modulation of MBNL expression with microRNA inhibitors, as a new therapeutic approach to DM. For the development of these inhibitors, which we had previously protected by a patent, as a therapy against DM, I was also awarded a valorization grants from Caixaimpulse program and FIPSE. My motivation is the translation of my research to benefit society and, as such, I have discovered five anti-DM1 compounds or repurposing drugs, which are protected by patents and licensed to biotech companies. I participated in multiple published research collaborations with national or international clinical and research groups and also in collaborations with biotech companies leading to licensing of patents, confidential agreements, and contracts for the development of these inventions. Some collaborations have been reinforced by short staying periods in laboratories outside Spain over a total of 12 months. Reflecting my integration in the Genetics Department, I am interested in teaching innovation and I participate in different graduate and undergraduate teaching assignments. I have also supervised 7 Master theses, one successfully concluded PhD with three additional PhDs in progress. Currently, I founded a biotech company called ARTHEX Biotech, dedicated tothe development of an antimiR-based drug against Myotonic dystrophy disease. The company raised seed funds on december 2019 from Invivo Capital and is actually working on the preclinical development of these molecules, planning to enter FIH on 2022.

Agenda Sessions

  • ROOM 204AB: Development of An antimiR-based Therapy to Treat Myotonic Dystrophy Disease