VALENCIA, Spain, Nov. 11, 2024 /PRNewswire/ — ARTHEx Biotech S.L., a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of gene expression, today announced an oral presentation at the upcoming TIDES Europe Annual Meeting, being held November 12-14, 2024 in Hamburg, Germany. 

“ARTHEx is excited to present our innovative science at TIDES, one of the premier oligonucleotide-focused meetings of the year.  We have made significant breakthroughs with our pipeline and platform, including creating a novel delivery method to deliver oligonucleotide therapies into the CNS through fatty-acid conjugation.  This discovery opens up a tremendous wealth of opportunity for ARTHEx and we look forward to applying our learnings and expanding our pipeline in neuromuscular and other neurological indications,” explained Dr. Beatriz Llamusí, ARTHEx Biotech Chief Scientific Officer and Co-Founder.

Details of the oral presentation are as follows:

Presenter: Beatriz Llamusí, PhD, ARTHEx Biotech Chief Scientific Officer and Co-Founder

Title: Fatty Acid Conjugation to Enable Oligonucleotide Delivery into the CNS

Session: Oligonucleotide Discovery, Preclinical and Clinical

Date/Time: Wednesday, November 13, 2024 – 15:00 – 15:30 CET

For more information about TIDES Europe, please visit the conference website here.

About ATX-01

ATX-01 is an oleic acid-conjugated antimiR oligonucleotide with preferential delivery to target tissues (muscle & brain) designed to inhibit microRNA 23b (miR-23b), which is a natural repressor of MBNL protein expression. In DM1 patients, loss of MBNL protein function caused by (1) reduced expression of MBNL protein due to miR-23b upregulation and (2) MBNL sequestration in toxic DMPK mRNA, lead to a spliceopathy, which is the cause of symptomatology in DM1 patients.

In human DM1 myoblast cell lines obtained from patients with a wide range of CTG repeat lengths, ATX-01 increased MBNL protein expression and significantly reduced toxic DMPK mRNA, correcting critical cellular defects such as spliceopathy. 

ATX-01 is currently being evaluated in the Phase I-IIa ArthemiR™ trial for the treatment of DM1. ATX-01 has received Orphan Drug Designation for ATX-01 in DM1 from the US FDA and European authorities, as well as and Rare Pediatric Disease (RPD) Designation from the FDA. 

About ARTHEx Biotech

ARTHEx Biotech is a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of gene expression.  The Company’s lead investigational compound, ATX-01, is being evaluated for the treatment of myotonic dystrophy type 1 (DM1), a rare neuromuscular disorder, in the Phase I-IIa ArthemiR™ trial. ARTHEx is also advancing its in-house discovery engine to identify and develop nucleic acid-based therapies for other disorders with high unmet medical needs, including genetically-driven diseases like DM1.  The Company headquarters are in Valencia, Spain.

For more information, please visit www.arthexbiotech.com and engage with us on LinkedIn.

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SOURCE ARTHEx Biotech

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