{"id":60433,"date":"2024-12-04T13:02:16","date_gmt":"2024-12-04T12:02:16","guid":{"rendered":"https:\/\/pharma-trend.com\/en\/arrakis-therapeutics-unveils-data-for-its-lead-rationally-designed-rna-targeted-small-molecule-rsm-drug-program-demonstrating-disease-modifying-binding-to-rna-to-treat-myotonic-dystrophy\/"},"modified":"2024-12-04T13:02:16","modified_gmt":"2024-12-04T12:02:16","slug":"arrakis-therapeutics-unveils-data-for-its-lead-rationally-designed-rna-targeted-small-molecule-rsm-drug-program-demonstrating-disease-modifying-binding-to-rna-to-treat-myotonic-dystrophy","status":"publish","type":"post","link":"https:\/\/pharma-trend.com\/en\/arrakis-therapeutics-unveils-data-for-its-lead-rationally-designed-rna-targeted-small-molecule-rsm-drug-program-demonstrating-disease-modifying-binding-to-rna-to-treat-myotonic-dystrophy\/","title":{"rendered":"Arrakis Therapeutics Unveils Data for its Lead, Rationally Designed, RNA-Targeted Small Molecule (rSM) Drug Program Demonstrating Disease-Modifying Binding to RNA to Treat Myotonic Dystrophy"},"content":{"rendered":"
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\nPreclinical data presented at Cell Symposia conference show a small molecule directed at a pathogenic RNA achieved reversal of myotonia in an animal model of myotonic dystrophy Type 1 (DM1)<\/p>\n

\nSupports additional product development with Company\u2019s multi-modality rSM drug platform, broadening the reach of genetic medicine and offering the potential to treat diseases at their root cause with oral medicines<\/p>\n

WALTHAM, Mass.–(BUSINESS WIRE)–Arrakis Therapeutics<\/a>, a biopharmaceutical company pioneering the discovery of a new class of small molecule medicines that directly target RNA, today announced the first presentation of data for its RNA-targeted small molecule (rSM) drug program for the treatment of myotonic dystrophy type 1 (DM1), a form of muscular dystrophy. These preclinical data demonstrate Arrakis\u2019s leading-edge capability to rationally design a disease-modifying RNA-targeted small molecule, using its pioneering and comprehensive drug discovery platform. The data are being presented today at the Cell Symposia conference Chemical Biology in Drugging the Undrugged, taking place in San Francisco, CA on December 2-4, 2024.<\/p>\n

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\nThe preclinical data from in vivo<\/i> animal studies demonstrated the reversal of myotonia with Arrakis rSMs binding the target pathogenic CUG repeat RNA element responsible for DM1. Arrakis molecules selectively bind the CUG repeat, disrupt the formation of nuclear aggregates, release sequestered splicing factors, specifically Muscleblind-Like Splicing Regulator 1 (MBNL1), and correct splicing defects responsible for myotonia. Arrakis\u2019s proprietary RNA\u2010specific chemical, biological, and structural methods and RNA-directed medicinal chemistry enabled structure-based small molecule drug design targeting the trinucleotide (CUG) repeat expansion in the mRNA of DMPK (myotonic dystrophy protein kinase) that drives DM1 pathology. The methodologies applied to the DM1 drug program are being used by Arrakis to advance its pipeline of proprietary and partnered rSM drug candidates for a range of other diseases.<\/p>\n

\n\u201cWhile there have been serendipitous discoveries of small molecule drugs that bind to RNA, we believe this is one of the clearest examples of a rationally designed small molecule that has been purpose-built to address an RNA disease target,\u201d said Michael Gilman, PhD, Chief Executive Officer of Arrakis Therapeutics. \u201cWith the many tools we\u2019ve built and lessons we\u2019ve learned along the way, we believe that our DM1 program is the first of many potential RNA-targeted small molecule drug candidates that will emerge from our platform in coming years.\u201d<\/p>\n

\nDr. Gilman added, \u201cFrom the beginning, we have believed in the power of targeting RNA and committed to deeply understand RNA sequence, structure and function. rSMs combine the specificity, validation, and impact of genetic medicines with the well understood benefits of small molecule drugs, including systemic biodistribution, oral delivery, and more streamlined and cost-efficient supply chains. With our platform now built and scaled, we look forward to advancing our pipeline and vastly expanding the reach of RNA-targeted medicines by deploying our industry\u2019s most established drug modality, oral small molecules.\u201d<\/p>\n

\nA presentation at Cell Symposia describes results from several preclinical studies. Highlights include:<\/p>\n