Valencia, Spain, May 9th, 2023 – ARTHEx Biotech S.L., a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of microRNAs, announced today a presentation at the TIDES USA Oligonucleotide & Peptide Therapeutics annual meeting being held May 7-10, 2023, in San Diego, CA.
Title: “Preclinical Development of a Lipophilic-conjugated Antimir to Treat Myotonic Dystrophy Type 1 (DM1)”
Date: Wednesday, May 10, 2023
Time: 8:30 am PT
Presenter: Dr. Beatriz Llamusi, Chief Scientific Officer & Co-Founder of ARTHEx
Location: Manchester Grand Hyatt, San Diego, CA
ATX-01 is an antimiR designed to target microRNA 23b (miR-23b), which is associated with regulating the expression 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 optimize novel 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.
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