Rocket Pharmaceuticals has developed a gene therapy using an adeno-associated virus vector for treating arrhythmogenic cardiomyopathy (ACM) caused by PKP2 gene mutations. The method involves administering the rAAV vector with specific genetic components through various routes. GlobalData’s report on Rocket Pharmaceuticals gives a 360-degree view of the company including its patenting strategy. Buy the report here.
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Gene therapy for arrhythmogenic cardiomyopathy using raav vector
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A recently granted patent (Publication Number: US11883506B2) discloses a method for treating arrhythmogenic cardiomyopathy (ACM) in individuals with a mutation in the Plakophilin-2 (PKP2) gene. The method involves administering a recombinant adeno-associated virus (rAAV) vector containing specific genetic components via various routes such as intravenous, intracoronary, intracardiac administration, or cardiac catheterization. The vector genome includes an expression cassette with a polynucleotide sequence encoding PKP2, a promoter sequence linked to the PKP2 sequence, and a polyA sequence. The rAAV vector utilized in the method is a recombinant AAVrh74 vector with a specific human cardiac troponin T (hTNNT2) promoter and PKP2 isoform A.
Furthermore, the patent claims specify additional details such as the percentage of sequence identity required for the capsid protein in the rAAV vector, the type of polyA sequence used, the dosage range for administering the rAAV vector, and the specific type of ACM targeted, which includes arrhythmogenic right ventricular cardiomyopathy (ARVC). The method allows for flexibility in administration methods, including injection or infusion, and provides precise dosing guidelines for optimal efficacy. This patent highlights a novel approach to treating ACM in individuals with a PKP2 gene mutation, offering potential advancements in the management of this genetic condition through gene therapy techniques involving rAAV vectors.
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GlobalData Patent Analytics tracks bibliographic data, legal events data, point in time patent ownerships, and backward and forward citations from global patenting offices. Textual analysis and official patent classifications are used to group patents into key thematic areas and link them to specific companies
