miRecule and Sanofi partner to develop therapy for muscular dystrophy

miRecule and Sanofi they have introduced strategic partnership and exclusive license agreement to develop and commercialize an antibody-RNA conjugate (ARC) for the treatment of facioscapulohumeral muscular dystrophy (FSHD).

Under the partnership, miRecule’s anti-DUX4 RNA therapy will be combined with Sanofi’s muscle-targeting NANOBODY technology to fuse the two molecules into an ARC using NAVIgGator conjugation and the former’s formulation chemistry.

Anti-DUX4 RNA therapy was discovered using miRecule’s DREAmiR platform.

The resulting therapy may become a disease-modifying treatment that selectively targets and reduces the underlying cause of FSHD in muscle tissue.

This innovative strategy would potentially allow FSHD patients to restart their normal aging process without the debilitating effects of the progressive disease.

Under the deal, Sanofi will receive an exclusive global license of intellectual property rights for the FSHD therapy from miRecule.

The companies will work together on research activities until the lead candidate is selected.

Subsequently, Sanofi will be fully responsible for investigational new drug (IND) application studies, as well as subsequent development and marketing work worldwide.

Sanofi will make over $30 million in upfront payments as well as short-term principal payments to miRecule.

In addition, miRecule is eligible to receive further development, regulatory and commercial milestone payments of approximately $400 million to advance the FSHD drug candidate.

Sanofi will exclusively develop and commercialize the drug candidate.

miRecule is also eligible to receive differential royalty payments on worldwide net sales of the approved product developed under the partnership.

Sanofi’s Global Head of Rare and Neurological Research, Pablo Sardi, said: “We look forward to working with miRecule to bring together our two breakthrough technologies that synergize into a best-in-class therapy designed to suppress the underlying cause of FSHD.

“We hope this will allow patients to live a life free from the debilitating symptoms of the disorder.”

In September of this year, the company and Scribe Therapeutics entered into a strategic partnership to accelerate the development of CRISPR-based cell therapies for cancer.

Cell and gene therapy coverage for pharmaceutical technologies is supported by Cytiva.

Editorial content is independently created and follows highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.

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