Sanofi In-Licenses Preclinical Genetic Disease Candidate From An Unknown Biotech

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  • French pharma major Sanofi SA SNY entered into a collaboration and licensing agreement with little-known U.S. biotech miRecule, an innovator of next-generation RNA therapeutics.
  • Under the terms of the deal, Sanofi will develop and commercialize a best-in-class antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD). 
  • Sanofi is getting exclusive worldwide rights for the program, known as MC-DX4, while miRecule gets upfront and near-term milestones of about $30 million.
  • Additional biobucks could bring the deal's total value to nearly $400 million.
  • FSHD is the second most common type of muscular dystrophy – affecting more than one million individuals worldwide. 
  • Patients with causative genetic mutations experience lifelong deterioration of muscle function and progressive disability. 
  • Related: Amid RNA Setbacks, Start-Up Bets On Sanofi's siRNA Platform.
  • The collaboration will combine miRecule's anti-DUX4 RNA therapy (discovered through its proprietary DREAmiR platform) with Sanofi's proprietary muscle-targeted NANOBODY technology to join the two molecules into an ARC utilizing miRecule's NAVIgGator conjugation and formulation chemistry. 
  • The collaboration marks the first licensing transaction leveraging miRecule's proprietary DREAmiR discovery platform bolstering commitment to further expansion of the company's RNA therapeutic pipeline.
  • Price Action: SNY shares are up 1.76% at $39.88 on the last check Tuesday.
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