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Rocket Pharmaceuticals Receives Orphan Medicinal Product Designation from the European Commission for RP-A601 for PKP2 Arrhythmogenic Cardiomyopathy

Rocket Pharmaceuticals, Inc. , a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the European Commission (EC), based on a positive. ....

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Agios Pharmaceuticals to Present Clinical and Translational Data in Rare Blood Disorders at European Hematology Association 2024 Hybrid Congress

Agios Pharmaceuticals to Present Clinical and Translational Data in Rare Blood Disorders at European Hematology Association 2024 Hybrid Congress
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Rocket Pharmaceuticals Presents Positive Data from LV Hematology Portfolio at the 27th Annual Meeting of the American Society of Gene and Cell Therapy

Rocket Pharmaceuticals Presents Positive Data from LV Hematology Portfolio at the 27th Annual Meeting of the American Society of Gene and Cell Therapy
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Rocket Pharmaceuticals Presents Positive Data from LV Hematology Portfolio at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)

Rocket Pharmaceuticals Presents Positive Data from LV Hematology Portfolio at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)
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Rocket Pharmaceuticals Reports First Quarter 2024 Financial Results and Highlights Recent Progress

Advanced RP-L102 for Fanconi Anemia towards regulatory reviews; EMA accepted MAA for review and BLA submission anticipated in the first half of 2024

Continued preparations to launch LV portfolio. ....

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