Beatriz Llamusí, MsC, PhD; Ruben Artero, MsC, PhD; Pedro Fernández, MsC, PhD; Sheila Soriano, MsC; Marc Carrscosa
Competition Sponsor: EIT Health of the European Union
ARTHEx is developing an advanced RNA therapy for myotonic dystrophy type 1 (DM1) based on a portfolio of patent-protected antimiRs. These antimiRs act as long-acting disease modifiers, targeting key pathways leading to disease through a clearly defined, first-in-class mechanism of action which is unique and novel.
These molecules have shown a promising long-term therapeutic effect after one single administration (proof of concept published in https://doi.org/10.1038/s41467-018-04892-4 and https://doi.org/10.1016/j.omtn.2020.07.021.
After the optimization and selection of the lead, our project entered into the IND-enabling toxicology studies in the last quarter of 2021.We plan to move forward into the clinical phase by the beginning of 2023.