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            Lead Product(s): rFVIIIFc-VWF-XTEN

            Therapeutic Area: Genetic Disease Product Name: BIVV001

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Swedish Orphan Biovitrum AB

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 10, 2020

            Details:

            BIVV001 is the first investigational factor VIII therapy independent of von Willebrand Factor and has the potential to transform replacement therapy for people with hemophilia A.

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            Lead Product(s): rFVIIIFc-VWF-XTEN,Recombinant Antihemophilic Factor

            Therapeutic Area: Genetic Disease Product Name: BIVV001

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 29, 2020

            Details:

            Presentations include findings on hemophilia and acquired thrombotic thrombocytopenic purpura portfolio therapies and pipeline candidates.

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            Lead Product(s): Givosiran

            Therapeutic Area: Genetic Disease Product Name: ALN-AT3SC

            Highest Development Status: Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 19, 2020

            Details:

            Long-term interim results from the Phase 2 open-label extension (OLE) study reinforce fitusiran’s potential to restore hemostatic balance and to lower annualized bleed rates (ABRs) over a period up to 57 months.

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            Lead Product(s): Avalglucosidase alfa

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 16, 2020

            Details:

            Avalglucosidase alfa showed a 2.4-point improvement in percent-predicted forced vital capacity, an important measure of respiratory function in Pompe disease, compared to alglucosidase alfa (standard of care).

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            Lead Product(s): Avalglucosidase alfa

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 08, 2020

            Details:

            Sanofi will host a virtual scientific session to present data from the Phase 3 COMET trial of investigational enzyme replacement therapy (ERT) avalglucosidase alfa in patients with late-onset Pompe disease (LOPD).

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            Lead Product(s): Avalglucosidase alfa

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 10, 2020

            Details:

            In patients with late-onset Pompe disease (LOPD), 5.5 years of safety and exploratory efficacy results showed avalglucosidase alfa was well-tolerated and stabilized pulmonary and motor function.

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            Lead Product(s): Palovarotene

            Therapeutic Area: Genetic Disease Product Name: IPNG 60120

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 10, 2020

            Details:

            The presentation will highlight data from Ipsen’s MOVE trial, the first and only multicenter Phase III study in fibrodysplasia ossificans progressiva.

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            Lead Product(s): Palovarotene

            Therapeutic Area: Genetic Disease Product Name: IPNG 60120

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 25, 2020

            Details:

            Post hoc analyses showed substantial reduction (62%) in mean annualized new heterotopic ossification volume in patients with FOP who were treated with oral investigational therapy palovarotene.

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            Lead Product(s): Lenadogene nolparvovec

            Therapeutic Area: Genetic Disease Product Name: Lumevoq

            Highest Development Status: Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Kreos Capital

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement July 09, 2020

            Details:

            Together with anticipated revenues from the Temporary Authorization for Use of LUMEVOQ® in France, The company expect to be financed until at least mid-2021.

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            Lead Product(s): Baclofen,Naltrexone Hydrochloride,Sorbitol API

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 10, 2020

            Details:

            PXT3003 is a novel drug candidate for the treatment of CMT1A and has been granted both Orphan Drug Designation and Fast Track Designation by the US Food and Drug Administration.

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