The MyoArete pipeline consists of utrophin upregulation-based MyoAr products for treatment of Duchenne muscular dystrophy (DMD). These have been identified using new platform technologies developed at the founder's laboratory at the University of Pennsylvania, with patents pending, and are being exclusively licensed to MyoArete. Our utrophin upregulation-based treatments are predicted to increase utrophin expression and benefit all DMD patients, regardless of mutation status.
MyoAr Small Molecules are low molecular weight organic compounds with pharmacological properties suitable for drug development. The MyoAr Small Molecules alleviate mRNA repression and increase utrophin expression.
MyoAr SBOs are chemically synthesized, modified oligonucleotides designed to block miRNAs from binding and repressing the utrophin messenger RNA. The
MyoAr SBOs alleviate miRNA-mediated repression and increase utrophin expression.
MyoAr Gene Edit is our gene editing therapy that precisely and permanently removes repressor sites from the utrophin gene and increases utrophin expression.
Our lead program is MyoAr Small Molecules.