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            Lead Product(s): Voxelotor

            Therapeutic Area: Genetic Disease Product Name: Oxbryta

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 08, 2020

            Details:

            GBT intends to submit a New Drug Application for Oxbryta, which will include a new age-appropriate formulation, for the treatment of SCD in children ages 4 to 11 years. The NDA will include clinical data from the ongoing Phase 2a HOPE-KIDS 1 study.

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            Lead Product(s): Recombinant coagulation factor IX

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: MEDEXUS INC

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Acquisition May 22, 2020

            Details:

            The revenues from IXINITY® for March 2020 are in line with the Company’s expectations, including that the majority of the quarterly revenue from IXINITY® would be recognized in the final month of Q4 2020.

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            Lead Product(s): Lumacaftor,Ivacaftor

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: FSIO

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement April 21, 2020

            Details:

            Agreement enables the possibility of rapid patient access to future triple combination regimen (elexacaftor/tezacaftor/ivacaftor and ivacaftor) once approved in Switzerland .

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            Lead Product(s): Glutamine

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 16, 2020

            Details:

            Acceptance by Saudi Food & Drug Authority will expedite the review process for Emmaus Life Sciences's lead candidate Endari® for treatment of sickle cell disease.

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            Lead Product(s): Recombinant coagulation factor IX

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: MEDEXUS INC

            Deal Size: $30.0 million Upfront Cash: $30.0 million

            Deal Type: Acquisition February 28, 2020

            Details:

            Acquisition strengthens Medexus’ specialty product portfolio and represents a strategic fit with other products in the Medexus business development pipeline.