ARTHEx biotech drug delivery Platform: Enhancing antimiR delivery to treat unmet diseases

The drug delivery Platform by ARTHEx biotech allows fast and flexible development of short-length investigational oligonucleotides to treat unmet diseases by inhibiting microRNAs in the affected tissues.

ARTHEx Biotech is a global leader in the development of investigational anti-microRNA (antimiR) oligonucleotide drugs for “undruggable” and difficult to treat diseases such as Myotonic Dystrophy Type 1 (DM1).

DM1 is a highly disabling disease affecting more than one million people worldwide. The condition affects muscles and other body parts and most commonly first manifests during adolescence. Sadly, affected individuals have a significantly shortened lifespan and there is no current cure available for DM1.

ARTHEx biotech’s research has revealed novel therapeutic targets to treat DM1. Our most advanced drug, ATX-01, is a first-in-class disease-modifying therapy that addresses the cause of DM1 with a patented unique mechanism of action, which targets a micro-RNA that is key to the disease pathogenesis. ATX-01 has the potential to treat DM1 in a specific, effective and safe way, and this is supported by promising effects in pre-clinical studies of ATX-01 in two mouse models of the disease.

“Our mission is to develop treatments that allow modification of gene  expression by controlling miRNA levels. We are working to develop treatments for unmet medical needs including myotonic dystrophy, cancer cachexia and osteoarthritis,” said Dr Beatriz Llamusí, ARTHEx biotech CEO and Founder.

ARTHEx biotech launches a platform that will allow it to advance its pipeline of drugs by providing tissue-enriched delivery of antimiRs.

The ARTHEx biotech proprietary platform will open up new treatment paths for people with difficult to treat diseases.

“Our innovative platform will potentially not only bring patients with DM1 a much-needed therapy but will lead to further development of life-changing drugs in other conditions. Improving tissue selectivity of our drugs, we are bringing the solution to the place where it is need ” said Dr Llamusi, who presented the platform in May at the TIDES conference in Boston, the industry's largest and most renowned event highlighting cutting edge research in protein and oligonucleotide therapeutics.

The platform improves drug delivery by linking an antisense oligonucleotide to a fatty acid. The oligonucleotide, an antimiR, inhibits a specific miRNA involved in the pathogenesis of a disease, while the fatty acid is selected to deliver the antimiR to the most affected tissues in the disease, enhancing therapeutic efficacy and safety.

ATX-01, developed to treat Myotonic Dystrophy, is the first clinical drug candidate to come from the platform. A second drug is in development for cancer cachexia, and ARTHEx expects more to follow. ARTHEx biotech is currently seeking to raise their Series A to obtain proof of concept of their therapeutic approach in humans with their first indication, and to deepen and enhance the overall capability and capacity of the platform.

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