ARTHEx Biotech to Present Preclinical Data on ATX-01 for Myotonic Dystrophy Type 1 (DM1) at RNA Leaders USA Congress
29 Aug 2023
Orphan DrugClinical Study
VALENCIA, Spain, Aug. 29, 2023 /PRNewswire/ -- 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 RNA Leaders USA Congress taking place September 6-7, 2023, in Boston, MA.
Presentation Details:
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
About ATX-01
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.
Company Contact
Frédéric Legros
Executive Chairman and CEO
[email protected]
+33679495790
Investor and Media Contact
Amy Conrad
Juniper Point
[email protected]
+1 858-366-3243
SOURCE ARTHEx Biotech
For more details,please visit the original website
The content of the article does not represent any opinions of Synapse and its affiliated companies. If there is any copyright infringement or error, please contact us, and we will deal with it within 24 hours.
Organizations
Indications
Drugs
Hot reports
Get started for free today!
Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.
Start your data trial now!
Synapse data is also accessible to external entities via APIs or data packages. Leverages most recent intelligence information, enabling fullest potential.