Rocket Pharmaceuticals. has filed a patent for a method to treat Danon disease using a recombinant adeno-associated virus (rAAV) virion containing a LAMP-2 protein. The method targets subjects with inactivating mutations in the LAMP-2 gene. GlobalData’s report on Rocket Pharmaceuticals gives a 360-degree view of the company including its patenting strategy. Buy the report here.
According to GlobalData’s company profile on Rocket Pharmaceuticals, NSAID cancer drugs was a key innovation area identified from patents. Rocket Pharmaceuticals's grant share as of January 2024 was 28%. Grant share is based on the ratio of number of grants to total number of patents.
Treatment method for danon disease using raav virion
A recently filed patent (Publication Number: US20240033325A1) outlines a method for treating Danon disease in individuals with an inactivating mutation in the LAMP-2 gene. The method involves administering a recombinant adeno-associated virus (rAAV) virion containing a polynucleotide sequence encoding a LAMP-2 protein, specifically LAMP-2B. The therapeutic amount of the rAAV is specified to be less than 2×1014 vector genomes per kilogram of body weight, with additional options for combining the treatment with medications like tacrolimus, rituximab, and eculizumab to enhance efficacy.
Furthermore, the patent details the potential outcomes of the treatment, including transduction of cardiomyocyte and skeletal muscle, correction of Danon disease-associated abnormalities, improvement in cardiovascular pathophysiology, and a low incidence of adverse events. The method also specifies the use of specific promoters and enhancer regions in the AAV expression cassette, as well as the use of an AAV9 capsid for delivery. The patent also includes provisions for unit doses, pharmaceutical compositions, and kits for treating Danon disease, providing a comprehensive approach to addressing this genetic disorder.
To know more about GlobalData’s detailed insights on Rocket Pharmaceuticals, buy the report here.
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