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MiRecule Inc.
miRecule is a precision RNA therapeutics company developing first-in-class antibody-oligonucleotide conjugates (AOCs) to treat rare neuromuscular and oncology indications. Our lead programs focus on delivering disease-modifying RNA therapeutics to specific tissues using a proprietary antibody-targeting platform.
We are currently advancing:
- A first-in-class treatment for centronuclear myopathy (CNM) with strong preclinical efficacy and clear clinical endpoints, and no available treatments for the disease or therapeutics in clinical development
- A partnered FSHD program with Sanofi, benefiting from platform synergies,
- Additional discovery-stage programs in rare neuromuscular diseases, and one in oncology.
miRecule’s platform enables systemic delivery of RNA therapies with tissue- and cell-type specificity, solving a major challenge in the field. Our mission is to rapidly bring transformative treatments to patients with high unmet medical needs, with a focus on rare and ultra-rare diseases.
Address
GaithersburgMD
United States
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