Prevail Therapeutics has been granted a patent for compositions and methods to treat diseases related to lysosomal function, like Parkinson’s and Gaucher disease. The patent involves administering expression constructs encoding specific proteins to subjects in need, offering potential therapeutic benefits. GlobalData’s report on Prevail Therapeutics gives a 360-degree view of the company including its patenting strategy. Buy the report here.

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According to GlobalData’s company profile on Prevail Therapeutics, was a key innovation area identified from patents.

Gene therapy for gaucher disease using raav with gcase protein

Source: United States Patent and Trademark Office (USPTO). Credit: Prevail Therapeutics Inc

A recently granted patent (Publication Number: US11903985B2) discloses a method for treating Type 2 and Type 3 Gaucher disease by administering a recombinant adeno-associated virus (rAAV) containing a specific nucleic acid sequence encoding a glucocerebrosidase (Gcase) protein. The rAAV is administered at a specified dose range directly into the brain. Additionally, the patent covers methods for treating Type 1 Gaucher disease and Parkinson's disease with GBA1 mutations using similar rAAV vectors, with specific administration routes and dosages outlined in the claims.

The patent details the use of rAAV vectors with specific genetic sequences and capsid proteins for targeted treatment of Gaucher disease and Parkinson's disease with GBA1 mutations. The administration methods include suboccipital injections into the cisterna magna and intravenous delivery, with precise dosages specified for optimal therapeutic effects. Furthermore, the patent includes formulations for the rAAV vectors to ensure stability and efficacy during administration. Overall, the patent provides a comprehensive guide for utilizing rAAV technology in the treatment of these genetic disorders, offering potential advancements in therapeutic interventions for patients with Type 1 and Type 2 Gaucher disease, as well as Parkinson's disease with GBA1 mutations.

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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