ARTHEx

ARTHEx Biotech develops oligonucleotide therapies for neuromuscular diseases, with its lead investigational program being an anti-miR (anti-microRNA) therapeutic for Myotonic Dystrophy type 1 (DM1). The company's lead candidate works by inhibiting miRNAs that repress MBNL1/2 proteins, compensating for the loss of MBNL function that is the root cause of myotonic dystrophy. This first-in-class mechanism of action targets the root cause of the disease by addressing both toxic DMPK RNA and insufficient MBNL, rather than treating symptoms. The therapeutic oligonucleotide is conjugated to a fatty acid, enabling improved delivery to muscle tissue and allowing for very low active doses, which differentiates ARTHEx's approach from conventional oligonucleotide therapies that struggle with muscle tissue delivery. The company focuses on investigating anti-miRs to treat diseases with unmet medical needs in the neuromuscular and genetic disease space. Myotonic dystrophy is the most common form of adult-onset muscular dystrophy, and ARTHex's approach represents a potential disease-modifying therapy rather than symptomatic treatment. The company conducts clinical trials and maintains a focus on RNA medicine and gene therapy approaches for muscle disorders. Founded in 2018 and headquartered in Valencia, Spain, ARTHEx Biotech has raised approximately $93.6 million in a Series B round backed by the European Investment Bank, EIC Fund, Columbus Venture Partners, Invivo Ventures, Invivo Partners, AdBio Partners, Sound Bioventures, Hadean Ventures, Advent France Biotechnology, Bpifrance, CDTI, EIT Health, and Europa.

Orthopedics > Drugs > Muscle Disorders > Myotonic Dystrophy,

Genomics > Applied Genomics > Clinical Genomics > Therapeutics