Partnership combines miRecule’s unique RNA therapeutic discovery and conjugation platforms with Sanofi’s NANOBODY® technology to create a groundbreaking treatment for FSHD. Under the partnership, the anti-DUX4 RNA therapy of miRecule will be merged with the muscle-targeted NANOBODY technology of Sanofi to fuse the two molecules into an ARC using the NAVIgGator conjugation and formulation chemistry of the former.
The anti-DUX4 RNA therapy was discovered using the DREAmiR platform of miRecule. The potential resulting best-in-class therapy may become a disease-modifying treatment that selectively targets and suppresses the underlying cause of FSHD in muscle tissue. This groundbreaking approach would enable FSHD patients to resume their normal course of aging free from the debilitating effects of this progressive disease.
miRecule started its program in 2019 with strong support from the patient and academic community.
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