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    Find Drugs for Genetic Disease in Phase II/ Phase III Clinical Development in FRANCE

    A collaboration with SCORR Marketing

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              Sanofi

              • Development Update

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              FRANCE France

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              ASCO 2020

              Participation Not Confirmed

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              Positive topline results demonstrated by olipudase alfa, first and only investigational therapy in late-stage development for acid sphingomy...

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              Lead Product(s): Olipudase alfa

              Therapeutic Area: Genetic Disease

              Highest Development Status: Phase II/ Phase III Product Type: Large molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable January 30, 2020

              Details:

              Olipudase alfa is the first and only investigational enzyme replacement therapy in late-stage development for the treatment of ASMD.

              Lysogene

              • Development Update

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              FRANCE France

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              ASCO 2020

              Participation Not Confirmed

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              Lysogene Receives FDA Fast Track Designation for LYS-SAF302 Gene Therapy in MPS IIIA

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              Lead Product(s): LYS-SAF302

              Therapeutic Area: Genetic Disease

              Highest Development Status: Phase II/ Phase III Product Type: Gene therapy

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable February 25, 2020

              Details:

              The AAVance Phase 2/3 clinical study is designed as an open-label, single-arm, multicenter study of LYS-SAF302 for the treatment of MPS IIIA.

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