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Find Approved Drugs for Genetic Disease in Clinical Development

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            Lead Product(s): Ivacaftor

            Therapeutic Area: Genetic Disease Product Name: Kalydeco

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator:

            Deal Size: Not Available Upfront Cash: Not Available

            Deal Type: Not Available November 05, 2020

            Details:

            KALYDECO® (ivacaftor) will be now available to additional eligible patients in Germany and will be available shortly in countries that have entered into innovative long-term reimbursement agreements with Vertex, including the UK, Denmark and the Republic of Ireland.

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            Lead Product(s): Ivacaftor

            Therapeutic Area: Genetic Disease Product Name: Kalydeco

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Royalty Pharma

            Deal Size: $650.0 million Upfront Cash: $575.0 million

            Deal Type: Acquisition November 02, 2020

            Details:

            Funding will support the CF Foundation’s work to fund research and drug development and advance high-quality, specialized CF care. KALYDECO® (ivacaftor) as First and Only CFTR Modulator, indicate to treat Eligible Infants With CF as Early as Four Months of Age.

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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 October 30, 2020

            Details:

            Data shows that nine out of ten people living with sickle cell disease experience one or more VOCs in a year, with a third of those crises leading to hospitalization, underscoring the significant unmet need among a vulnerable group of patients.

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            Lead Product(s): Onasemnogene Abeparvovec,NAV AAV9 vector

            Therapeutic Area: Genetic Disease Product Name: Zolgensma

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

            Partner/Sponsor/Collaborator: Novartis Pharmaceuticals Corporation

            Deal Size: $80.0 million Upfront Cash: Undisclosed

            Deal Type: Agreement October 27, 2020

            Details:

            REGENXBIO will receive a milestone payment of $80.0 million from Novartis AG based upon the achievement of $1.0 billion in cumulative net sales of Zolgensma. REGENXBIO expects to recognize this revenue in the third quarter of 2020.

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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 October 12, 2020

            Details:

            Discovered and developed by GBT, Oxbryta is the first and only therapy that directly inhibits hemoglobin polymerization, the root cause of the sickling and destruction of red blood cells in sickle cell disease (SCD).

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            Lead Product(s): Alpha1-Proteinase Inhibitor (Human)

            Therapeutic Area: Genetic Disease Product Name: Glassia

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Takeda Pharmaceutical

            Deal Size: $25.0 million Upfront Cash: Undisclosed

            Deal Type: Agreement October 07, 2020

            Details:

            Kamada Expects to Receive $25 Million in Revenues from Sales of GLASSIA® to Takeda in 2021 which is Takeda’s Minimum Commitment for 2021 Pursuant to the Existing Supply Agreement.

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            Lead Product(s): Burosumab

            Therapeutic Area: Genetic Disease Product Name: Crysvita

            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 October 05, 2020

            Details:

            CRYSVITA, created by Kyowa Kirin, is a recombinant fully human monoclonal IgG1 antibody against the FGF23. More people in Europe are now eligible for treatment with CRYSVITA, the only therapy that targets the underlying pathophysiology of XLH.

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            Lead Product(s): Onasemnogene Abeparvovec

            Therapeutic Area: Genetic Disease Product Name: Zolgensma

            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 October 01, 2020

            Details:

            New interim Phase 3 STR1VE-EU data presented at WMS support the robust clinical evidence that have demonstrated a consistent, transformative benefit across Zolgensma clinical trials for the treatment of patients with SMA.

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            Lead Product(s): Porcine recombinant Factor VIII

            Therapeutic Area: Genetic Disease Product Name: Obizur

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: HLS Therapeutics

            Deal Size: $49.3 million Upfront Cash: $30.8 million

            Deal Type: Acquisition September 30, 2020

            Details:

            HLS has acquired certain entities that hold the rights to a diversified portfolio of royalty interests on global sales of four different products, this includes Takeda Pharmaceutical's Obizur, a porcine recombinant Factor VIII for acquired hemophilia A.

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            Lead Product(s): Human C1 Esterase Inhibitor

            Therapeutic Area: Genetic Disease Product Name: Haegarda

            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 September 28, 2020

            Details:

            The latest FDA approval was based on results from two CSL Behring-sponsored COMPACT trials. In the COMPACT pivotal study, HAEGARDA, at the FDA approved dose of 60 IU/kg, reduced the number of HAE attacks by a median of 95% relative to placebo.

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