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            Lead Product(s): Crizanlizumab

            Therapeutic Area: Genetic Disease Product Name: Adakveo

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 24, 2020

            Details:

            CHMP opinion supported by data showing Adakveo significantly reduced the rate of vaso-occlusive crises, with patients on Adakveo spending fewer days in hospital.

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            Lead Product(s): Onasemnogene abeparvovec-xioi

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Approved Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 19, 2020

            Details:

            Zolgensma is a transformative and highly innovative one-time gene therapy for a devastating and progressive genetic disease and is consistently priced worldwide under a value-based framework.