Sanofi and next-gen RNA biotech miRecule announced this week a new partnership and licensing agreement for the development of an antibody-RNA conjugate (ARC) treatment for facioscapulohumeral muscular dystrophy (FSHD).
MiRecule is a relatively young biotech, founded in 2016 and headquartered in Gaithersburg, Maryland. As part of the new collaboration, the two companies will work together using miRecule’s proprietary DREAmiR platform which uses genomic analysis to develop targeted RNA therapies in combination with Sanofi’s proprietary muscle-targeted NANOBODY technology.
As a first endeavor, the companies announced that they will use miRecule’s DUX4 RNA therapy with Sanofi’s technology to join two molecules into an ARC to produce a “potential best-in-class” disease-modifying treatment for otherwise progressive and debilitating disease. Patients living with FSHD — roughly 870,000 worldwide — face lifelong muscle function deterioration, and currently, there is no treatment to slow down or cure it.
In exchange for exclusive worldwide license to intellectual property rights to the FSHD therapy developed, miRecule will receive an upfront payment and near-term milestone payments nearing $30 million combined. Future regulatory, development and commercial milestone payments could make it closer to $400 million.