VALENCIA, Spain, July 23, 2026
ARTHEx Biotech has announced the publication of groundbreaking preclinical data demonstrating that its investigational RNA therapeutic ATX-01 achieved central nervous system (CNS) activity and improved behavioral alterations in a preclinical model of Myotonic Dystrophy Type 1 (DM1). Published in Cell Reports Medicine, the study is the first to demonstrate a direct connection between molecular correction in the brain and functional behavioral improvement in a DM1 animal model. The findings strengthen the therapeutic potential of ATX-01, developed using the company’s proprietary BOOST-ON™ platform, and highlight its ability to cross the blood-brain barrier, address the underlying disease biology, and potentially treat both the muscular and neurological manifestations of this rare neuromuscular disorder. The results represent an important milestone in the development of disease-modifying RNA therapies for DM1, a debilitating genetic disease that currently has no approved cure or disease-modifying treatment.
ATX-01 Demonstrates Brain Delivery and Disease Modification
The newly published research provides compelling evidence that systemically delivered ATX-01 successfully reaches the brain following intravenous administration, overcoming one of the greatest challenges in treating Myotonic Dystrophy Type 1. Using ARTHEx’s BOOST-ON™ delivery platform, the investigational therapy crossed the blood-brain barrier, increased MBNL1 and MBNL2 protein expression, reduced toxic DMPK RNA transcripts, and partially corrected RNA splicing abnormalities across multiple brain regions. Importantly, researchers also observed normalization of behavioral alterations, including improvements in exploratory activity, while reporting no significant toxicity or neuroinflammation. These findings represent the first scientific evidence linking molecular correction within the brain to measurable functional improvements in a DM1 model, supporting the potential for ATX-01 to address both neurological and muscular disease manifestations through a single systemic treatment approach.
BOOST-ON™ Platform Expands RNA Therapeutic Potential
The study also highlights the capabilities of ARTHEx Biotech’s proprietary BOOST-ON™ platform, which combines selective oligonucleotide technology with optimized tissue delivery to target skeletal muscle, heart, and brain. Unlike many RNA therapeutics that face challenges reaching the central nervous system, BOOST-ON™ enabled efficient delivery of antimiR-23b to the brain, reinforcing its potential for multisystem diseases such as DM1. ATX-01 works by inhibiting microRNA-23b (miR-23b), restoring levels of Muscleblind-like (MBNL) proteins, which play a critical role in correcting the RNA mis-splicing responsible for the disease. Because DM1 affects multiple organ systems—including skeletal muscle, cardiac tissue, and the central nervous system—the ability to target these tissues simultaneously may provide a more comprehensive therapeutic strategy. The newly reported CNS findings build upon earlier preclinical studies that demonstrated improvements in skeletal muscle function, further validating the platform’s disease-modifying potential.
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Clinical Development Advances Toward Comprehensive DM1 Treatment
ARTHEx Biotech is currently advancing ATX-01 through the Phase I/IIa ArthemiR™ clinical study, evaluating the therapy in patients with Myotonic Dystrophy Type 1. Company leadership noted that while the initial clinical focus has been on improving systemic and muscular symptoms, the latest preclinical findings significantly expand the therapeutic outlook by demonstrating the potential to address central nervous system involvement, an area of major unmet medical need. The company continues to collaborate with regulatory authorities, clinical investigators, and patient advocacy organizations to accelerate clinical development. If future clinical studies confirm these promising preclinical results, ATX-01 could become one of the first RNA-based therapies capable of addressing both the neurological and muscular complications of DM1, offering a potentially transformative treatment option for patients affected by this progressive rare genetic disorder while reinforcing ARTHEx’s leadership in next-generation RNA therapeutics.
Source: ARTHEx Biotech press release
