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[{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Chugai\u2019s Huntington\\'s disease drug, RG6042 gets Japanese orphan drug designation","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Chugai Pharmaceutical"},{"orgOrder":0,"company":"Nippon Shinyaku","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"EC Grants Orphan Drug Designation to Nippon Shinyaku's viltolarsen (NS-065\/NCNP-01)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Nippon Shinyaku"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Takeda Data at ISTH 2021 Highlight the Benefits of Prophylaxis for Patients with Rare Bleeding Disorders","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Takeda Pharmaceutical"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Takeda to Commercialize Next-Generation Hunter Syndrome Therapy Through Collaboration with JCR Pharmaceuticals","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"September 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"JCR Pharmaceuticals"},{"orgOrder":0,"company":"Nippon Shinyaku","sponsor":"Capricor Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Nippon Shinyaku and Capricor Therapeutics enter into an Exclusive Partnership for Commercialization and Distribution of CAP-1002 for the Treatment of Duchenne Muscular Dystrophy in US","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Nippon Shinyaku"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"JCR Announces First Patient Dosed in Phase 3 Global Clinical Trial of JR-141 for Treatment of MPS II (Hunter Syndrome)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"February 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"JCR Pharmaceuticals"},{"orgOrder":0,"company":"Kubota Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Kubota Vision Announced End of Phase 3 Clinical Trial of Emixustat in Patients with Stargardt Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Kubota Pharmaceutical"},{"orgOrder":0,"company":"Kubota Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Kubota Vision Announces Positive Post Hoc Analysis from Phase 3 Clinical Trial of Emixustat in Patients with Stargardt Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Kubota Pharmaceutical"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"U.S. Food and Drug Administration Accepts Takeda\u2019s Supplemental Biologics License Application for Use of TAKHZYRO (lanadelumab-flyo) to Prevent Hereditary Angioedema (HAE) Attacks in Children 2 Years of Age and Older","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Takeda Pharmaceutical"}]

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            TAKHZYRO® (lanadelumab-flyo) Injection is a prescription medicine used to prevent attacks of hereditary angioedema (HAE) in people 12 years of age and older.

            Lead Product(s): Lanadelumab

            Therapeutic Area: Genetic Disease Product Name: Takhzyro

            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 October 05, 2022

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            Emixustat modulates visual cycle by inhibiting a critical enzyme of this pathway, retinal pigment epithelium protein 65, in analysis of Phase 3 trial it is determined that emixustat treatment resulted in a 40.8% reduction in lesion progression compared to placebo at month 24.

            Lead Product(s): Emixustat Hydrochloride

            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 October 03, 2022

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            Emixustat modulates the visual cycle by inhibiting a critical enzyme of this pathway, retinal pigment epithelium protein 65 (RPE65). The visual cycle is the process by which vitamin A is recycled in the eye; vitamin A is crucial to the visual process.

            Lead Product(s): Emixustat Hydrochloride

            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 23, 2022

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            JR-141 is the first-ever approved enzyme replacement therapy (ERT) in Japan that penetrates the BBB via intravenous administration for the treatment of MPS II. JR-141 is expected to treat not only the systemic symptoms but also the neuronopathic manifestations of MPS II.

            Lead Product(s): Pabinafusp Alfa

            Therapeutic Area: Genetic Disease Product Name: JR-141

            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 15, 2022

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            CAP-1002 is comprised of human allogeneic cardiosphere-derived cells. Exosomes (extracellular vesicles) secreted by CAP-1002 are thought to be the mechanism of action of CAP-1002 and have been shown to reduce oxidative stress, inflammation, fibrosis.

            Lead Product(s): Allogeneic Cardiosphere-derived Cell Therapy

            Therapeutic Area: Genetic Disease Product Name: CAP-1002

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

            Partner/Sponsor/Collaborator: Capricor Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership January 25, 2022

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            Under the terms of the collaboration and license agreement, Takeda will exclusively commercialize JR-141, a recombinant fusion protein of an antibody against the human transferrin receptor and iduronate-2-sulfatase in the U.S. upon completion of the Phase 3 program.

            Lead Product(s): Pabinafusp Alfa

            Therapeutic Area: Genetic Disease Product Name: JR-141

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Takeda Pharmaceutical

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration September 30, 2021

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            In Europe, VEYVONDI is indicated in adults (age 18 and older) with von Willebrand disease (VWD), when desmopressin (DDAVP) treatment alone is ineffective or not indicated for the treatment of hemorrhage and treatment/prevention of surgical bleeding.

            Lead Product(s): Recombinant von Willebrand Factor

            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 July 20, 2021

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            The NDA for viltolarsen is also being reviewed under an accelerated approval pathway in the U.S. The clinical study in the US is being conducted by NS Pharma, Inc.

            Lead Product(s): Viltolarsen

            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

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            The company is running a clinical study in cooperation with Roche to deliver RG6042 to patients with Huntington's disease, as the first disease-modifying drug.

            Lead Product(s): Tominersen

            Therapeutic Area: Genetic Disease Product Name: RG6042

            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 18, 2020

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