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[{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"uniQure Announces Positive CHMP Opinion for Etranacogene Dezaparvovec \u2013 Gene Therapy for Adults with Hemophilia B","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"Approved","date":"December 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Uniqure"},{"orgOrder":0,"company":"Uniqure","sponsor":"HealthCare Royalty","pharmaFlowCategory":"D","amount":"$400.0 million","upfrontCash":"$400.0 million","newsHeadline":"uniQure Announces Sale of Royalty Interest in HEMGENIX for Up To $400 Million","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"Approved","date":"May 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Uniqure"},{"orgOrder":0,"company":"Uniqure","sponsor":"CSL Behring","pharmaFlowCategory":"D","amount":"$2,050.0 million","upfrontCash":"$450.0 million","newsHeadline":"uniQure Announces Achievement of $100 Million Milestone Related to Hemophilia B Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"Approved","date":"June 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Uniqure"},{"orgOrder":0,"company":"Pharming","sponsor":"NewBridge Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Pharming Signs Agreement with NewBridge for the Commercialization of RUCONEST","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"NETHERLANDS","productType":"Large molecule","productStatus":"Approved","date":"July 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Pharming"}]

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            Details:

            The licensing agreement provided CSL Behring with exclusive global rights to Hemgenix (etranacogene dezaparvovec), uniQure’s first approved gene therapy for hemophilia B in the United States, EU and European Economic Area, and the UK, for patients with hemophilia B.

            Lead Product(s): Etranacogene Dezaparvovec-drlb

            Therapeutic Area: Genetic Disease Product Name: Hemgenix

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

            Partner/Sponsor/Collaborator: CSL Behring

            Deal Size: $2,050.0 million Upfront Cash: $450.0 million

            Deal Type: Licensing Agreement June 20, 2023

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            The divestment aims to sell a portion of the royalty rights due to uniQure from CSL Behring from the net sales of Hemgenix (etranacogene dezaparvovec-drbl), the first and only FDA-approved gene therapy for hemophilia B, to HealthCare Royalty and Sagard Healthcare.

            Lead Product(s): Etranacogene Dezaparvovec-drbl

            Therapeutic Area: Genetic Disease Product Name: Hemgenix

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

            Partner/Sponsor/Collaborator: HealthCare Royalty

            Deal Size: $400.0 million Upfront Cash: $400.0 million

            Deal Type: Divestment May 15, 2023

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            Details:

            Hemgenix (Etranacogene dezaparvovec), is a one-time gene therapy for the treatment of appropriate adults with hemophilia B that reduces the rate of annual bleeds after a single infusion.

            Lead Product(s): Etranacogene Dezaparvovec-drlb

            Therapeutic Area: Genetic Disease Product Name: Hemgenix

            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 December 16, 2022

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            Under the terms of the agreement, NewBridge will work closely with Pharming to provide access for RUCONEST® for the treatment of acute hereditary angioedema (“HAE”) in MENA.

            Lead Product(s): Conestat Alfa

            Therapeutic Area: Genetic Disease Product Name: Ruconest

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: NewBridge Pharmaceuticals

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement July 20, 2021

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