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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.

Rocket pharmaceuticals incCommittee for orphan medicinal productsEuropean medicines agencyProduct serviceRocket pharmaceuticals receives orphan medicinal product designationEuropean unionEuropean commissionOrphan medicinal productsFast trackOrphan drugArrhythmogenic cardiomyopathyRocket pharmaceuticalsFanconi anemiaLeukocyte adhesion deficiency iPyruvate kinase deficiencyDanon disease

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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United kingdomUnited statesRio de janeiroEstado do rioFuad el rassiLaron browneBiree andemariamAlit taherDan budwickKevinhm kuoChris taylorDepartment of hematologyEuropean hematology associationHarvard medical schoolDivision of hematologyBlood disorder center

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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United statesState of californiaInvestigacion biomedicaFundacion jimenezInvestigaciones energeticasKevin giordanoMedioambientalesy tecnologicasJonathan schwartzUniversity college londonEuropean medicines agencyRocket pharmaceuticals incAmerican society of geneDrug administrationCalifornia institute for regenerative medicine grant numberRocket pharmaceuticalsAnnual meeting

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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United statesState of californiaEnfermedades rarasInvestigaciones energJonathan schwartzMedioambientalesy tecnolRocket pharmaceuticals incDrug administrationUniversity college londonRocket pharmaceuticalsCalifornia institute for regenerative medicine grant numberEuropean medicines agencyMeeting of the american society geneAnnual meetingAmerican societyCell therapy

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.

United statesStanford universityLos angelesAgnieszka czechowiczDonaldb kohnGaurav shahCompany cranburyStanford university school of medicineAmerican society of geneDrug administrationRocket pharmaceuticalsDepartment of pediatricsRocket pharmaceuticals incTreatment centerAlexion pharmaceuticalsDivision of hematology oncology