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Morgan","pharmaFlowCategory":"D","amount":"$175.0 million","upfrontCash":"Undisclosed","newsHeadline":"Rocket Pharmaceuticals Announces Pricing of Public Offering of Common Stock","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Reaches FDA Alignment on Pivotal Phase 2 Trial Design for RP-A501 in Danon Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Announces FDA Acceptance of Biologics License Application with Priority Review for RP-L201 (marnetegragene autotemcel) for the Treatment of Severe Leukocyte Adhesion Deficiency-I (LAD-I)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Announces Update on FDA Review Timeline of KRESLADI\u2122 (marnetegragene autotemcel) for the Treatment of Severe Leukocyte Adhesion Deficiency-I (LAD-I)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Announces European Medicines Agency Acceptance of RP-L102 Marketing Authorization Application for the Treatment of Fanconi Anemia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"}]
Find Clinical Drug Pipeline Developments & Deals by Rocket Pharmaceuticals
RP-L102 is a lentiviral (LV) vector-based investigational gene therapy, which is being evaluated for the treatment of Fanconi Anemia, complementation group A.
Kresladi (marnetegragene autotemcel) is an investigational gene therapy that acts as a vector to deliver a functional copy of the ITGB2 gene. It is being developed for the treatment of severe leukocyte adhesion deficiency-I (LAD-I).
RP-L201 (marnetegragene autotemcel), a lentiviral vector (LV)-based investigational gene therapy, which is investigated for the treatment of Severe Leukocyte Adhesion Deficiency-I.
The proceeds will fund the development of company's pipeline of gene therapies for rare diseases, including advancement of RP-A501 in Phase 2 for Danon Disease. It consists of a recombinant AAV9 capsid containing a functional version of the human LAMP2B transgene.
RP-A501 is Rocket’s investigational gene therapy product for the treatment of Danon Disease and the first gene therapy for a cardiovascular condition to demonstrate safety and efficacy in clinical studies.
RP-A601 is an AAV.rh74-based gene therapy candidate for the treatment of arrhythmogenic cardiomyopathy due to plakophilin 2 pathogenic variants (PKP2-ACM).
RP-L301 is a gene therapy product containing autologous (patient-derived) hematopoietic stem cells (HSCs) that have been genetically modified with a lentiviral vector to contain a functional copy of the PKLR gene.
RP-A601 is an AAV.rh74-based gene therapy candidate for the treatment of arrhythmogenic cardiomyopathy due to plakophilin 2 pathogenic variants (PKP2-ACM).
RP-A501 (rAAV9 capsid) is a gene therapy product consisting of a recombinant adeno-associated serotype 9 capsid containing a functional version of the human LAMP2B transgene. RP-A501 has the potential to restore normal cardiac function in patients with Danon Disease.
RP-A501, the Company's investigational adeno-associated virus (AAV)-based gene therapy for Danon Disease. During the meeting, Rocket reviewed the positive Phase 1 dataset with the FDA and proposed a study design and endpoints of the investigational gene therapy.
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