-Study highlights dual targeting action, demonstrating significant therapeutic potential for treating myotonic dystrophy type 1 (DM1) across a variety of disease severities –
Valencia, Spain, October 15th, 2024 – ARTHEx Biotech S.L., a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of gene expression, today announced a publication in Science Advances showcasing the dual mechanism of two anti-miRs offering a promising therapeutic avenue in myotonic dystrophy type 1 (DM1) by addressing the reduced free Muscle-Blind Like Splicing regulator (MBNL) proteins due to sequestrationin DMPK mRNA foci and due to reduced production .
“This published research demonstrates the promising potential of RNA therapeutics to address the underlying disease-driving mechanisms of DM1,” said Beatriz Llamusi, PhD, Chief Scientific Officer and Co-Founder of ARTHEx Biotech. “We are pleased to have our science recognized by a high-quality journal like Science Advances and we look forward to realizing the potential impact of our approach on DM1 scientific research.”
This study described in the publication evaluated the potential therapeutic efficacy of two AntimiRs in primary myoblasts derived from patients with DM1. DM1, caused by unstable CTG repeat expansions in the DMPK gene, leads to muscle defects due to depletion of the crucial splicing regulator protein MBNL1. AntimiRs specifically targeting miR-23b and miR-218, which are natural repressors of MBNL1, demonstrated significant reductions in toxic DMPK mRNA, restoring normal MBNL1 levels and correcting critical cellular defects such as myoblast fusion, myotube formation and reversing splicing defects. Notably, the AntimiR-23b treatment reduced DMPK transcripts and reversed 68% of dysregulated genes, and did so across various forms of DM1, independent of the CTG repeat size, suggesting broad potential. These findings underscore the dual mechanism of AntimiR-23b, offering a promising therapeutic avenue by addressing MBNL1 depletion due to both DMPK accumulation and sequestration, as well as reduced production.
The published manuscript, titled “AntimiR treatment corrects myotonic dystrophy primary cell defects across several CTG repeat expansions with a dual mechanism of action“, is available online.
About Myotonic Dystrophy Type1 (DM1)
Myotonic dystrophy type 1 (DM1) is a highly disabling disease affecting more than one million people worldwide. The condition affects muscles and other tissues (causing respiratory problems, fatigue, hypersomnia, cardiac abnormalities, severe gastrointestinal complications, and cognitive and behavioral impairment). Most commonly, it manifests during adulthood (classic DM1), although DM1 can develop at birth in a congenital form, or during childhood. Although signs and symptoms vary among affected individuals, sadly, with progression of the disease, DM1 patients experience a reduction in the ability to perform activities of daily living. Moreover, patients have a significantly shortened lifespan and there is currently no approved treatment to slow the progression of the disease.
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 gene expression modulators 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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