Company to present preclinical data for its lead, rationally designed, RNA-targeted small molecule drug program for treatment of myotonic dystrophy type 1 (DM1)
WALTHAM, Mass.--(BUSINESS WIRE)--Arrakis Therapeutics, a biopharmaceutical company pioneering the discovery of a new class of small molecule medicines that directly target RNA, today announced that the company will present preclinical data for its RNA-targeted small molecule (rSM) drug program for the treatment of myotonic dystrophy type 1 (DM1), a form of muscular dystrophy. The data will be presented in an oral session at the Muscular Dystrophy Association (MDA) 2025 Clinical and Scientific Conference taking place on March 16-19 in Dallas, Texas.
Details of the presentation are as follows:
- Presentation Title: Discovery of Small Molecules that Bind CUG Repeats, Displace Muscleblind Protein, and Improve Pathogenesis of Myotonic Dystrophy Type 1
- Session: Preclinical Studies in Dystrophies and Myopathies
- Date: Wednesday, March 19, 2025
- Time: 10:15 a.m. CDT
- Presenter: Domi Stickens, PhD, Vice President, Discovery and Translational Sciences
Arrakis’s DM1 drug program represents a rationally designed small molecule genetic medicine directed at a pathogenic RNA to achieve reversal of myotonia in an animal model of DM1. Utilizing the company’s multi-modality rSM drug platform, this approach broadens the potential of genetic medicines to treat diseases at their root cause with oral small molecule medicines that have benefits of systemic biodistribution, convenient oral delivery, cost-efficient manufacturing, and established supply chains.
About the rSM Program to Treat Myotonic Dystrophy Type 1 (DM1)
Myotonic dystrophy type 1 (DM1) is a form of muscular dystrophy and a genetic neuromuscular disease affecting at least 1 in 8,000 people worldwide or approximately 45,000 people in the United States. It is a multi-system disease, affecting the skeletal muscle, heart, diaphragm, central nervous system, and gastro-intestinal tract. DM1 is caused by a trinucleotide (CUG) repeat expansion of the RNA encoding myotonic dystrophy protein kinase (DMPK ), resulting in the formation of nuclear aggregates that bind and sequester splicing factors important for cellular function. Arrakis’s rSM selectively binds to CUG repeats, disrupting aggregate formation in cells, liberating splicing factors, and correcting splicing defects in genes that drive DM1 pathology, including myotonia.
About Arrakis Therapeutics
Arrakis Therapeutics is a biopharmaceutical company pioneering the discovery of a new class of medicines that directly target RNA. Arrakis is building a proprietary pipeline of RNA-targeted small molecule (rSM) medicines focused on genetically validated targets and numerous disease areas. The company brings together scientific leaders in RNA structure, chemistry and biology, along with a highly experienced management team and the backing of leading life sciences investors. The company is located in Waltham, Massachusetts. For more information, please visit www.arrakistx.com and engage with us on LinkedIn.
Contacts
Media Contact:
Kathryn Morris
The Yates Network LLC
914-204-6412
kathryn@theyatesnetwork.com
