Muscular dystrophies are a group of degenerative genetic conditions that gradually weaken the muscles and lead to progressive disability. Myotonic dystrophy (DM1) is the most common muscular dystrophy in adults and is a highly disabling disease with symptoms involving skeletal musculature, heart and nervous system.
ARTHEx Biotech S.L. is a University of Valencia' spin-off company focused on the development of a treatment against type 1 muscular dystrophy, which is based on microRNA inhibitors. The proof of concept of the efficacy of these molecules in animal models was generated in the translational genomics laboratory of the University of Valencia, directed by Dr. Ruben Artero.
The founders of this company are Dr. Beatriz Llamusí and Dr. Ruben Artero. The business model proposed to complete this project corresponds to the current open innovation model, in which the biotechnology company that emerges from a research center develops the products until clinical phase 2 (efficacy), and then it licenses them to pharmaceutical multinationals with the ability to complete the development, more advanced studies and marketing.
The information contained in this business data sheep has been provided by the company and the University of Valencia Science Park Foundation (FPCUV) is not responsible for its content.
The technology we are developing is based on a patent licensed to the University of Valencia (P201631216).
Benefits of Technology
The company will develop an innovative therapy technology based on antisense RNA. It is a way of modifying gene expression without altering the patient's genome, so all complications of this type of therapy are avoided.
"Our main competitive advantage over other companies dedicated to treatments against myotonic dystrophy is that we have discovered a new therapeutic target that was unknown until now," underlines Dr. Llamusí.
The technology will be used for the treatment of myotonic dystrophy type 1, but it could also be used in patients of type 2 and congenital dystrophy. Around the world, this therapy could benefit more than one million people.