Valencia, Spain, August 24, 2023 – ARTHEx Biotech S.L., a clinical-stage biotechnology company focused on developing innovativemedicines through the modulation of microRNAs, announced today a presentation at the RNA Leaders USA Congress taking place September 6-7, 2023, in Boston, MA.
Title: “Development of a Lipophilic-conjugated Antimir (ATX-01) to Treat Myotonic Dystrophy Type 1 (DM1)
Date: Wednesday, September 6, 2023
Time: 2:30 pm ET
Presenter: Frédéric Legros, Chairman and CEO Arthex Biotech
Location: Boston Hynes Convention Center
ATX-01 is an antimiR designed to target microRNA 23b (miR-23b), which is associated with regulating theexpression of MBNL protein involved in the pathogenesis of DM1. It has been demonstrated in human DM1 myoblast cell lines that ATX-01 has a unique, dual mechanism of action which targets toxic DMPK and MBNL proteins. Toxic DMPK and reduced levels of MBNL have been identified as the genetic cause of DM1. In December 2022, ARTHEx announced the achievement of key regulatory milestones for the ATX-01 development program, including receiving Orphan Drug Designation for ATX-01 in DM1 from both the US and European authorities.
ATX-01 was discovered through ARTHEx’ in-house discovery engine, which is designed to identify and optimizenovel microRNA modulators and ensure their preferential delivery to targeted tissues, for the treatment of diseases in which microRNAs are involved in the disease pathogenesis.
About ARTHEx Biotech
ARTHEx Biotech is a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of microRNAs. The Company’s lead investigational compound, ATX-01, is advancing into clinical development for the treatment of myotonic dystrophy type 1 (DM1), a rare progressive muscle wasting disease. ARTHEx is also advancing its in-house discovery engine to identify and develop microRNA modulators for other disorders with high unmet medical needs, including genetically-driven diseases like DM1. The Company headquarters is in Valencia, Spain.
For more information, please visit www.arthexbiotech.com and engage with us on LinkedIn.
Executive Chairman and CEO
Investor and Media Contact