Voyager Therapeutics has been granted a patent for small interfering RNA (siRNA) molecules targeting the HTT gene to treat Huntington’s Disease. The patent also covers adeno-associated viral (AAV) vectors encoding the siRNA molecules. Claim 1 specifies a specific nucleotide sequence for modulatory polynucleotide. GlobalData’s report on Voyager 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 Voyager Therapeutics, Adeno-associated virus vectors was a key innovation area identified from patents. Voyager Therapeutics's grant share as of April 2024 was 15%. Grant share is based on the ratio of number of grants to total number of patents.

A recently granted patent (Publication Number: US11951121B2) discloses a modulatory polynucleotide with a specific nucleotide sequence (SEQ ID NO: 349) aimed at inhibiting the expression of the huntingtin (HTT) gene, mRNA, and/or protein in cells. The patent further describes an adeno-associated viral (AAV) vector genome containing the nucleotide sequence, along with additional elements such as promoters, enhancers, and introns. The invention also covers recombinant AAV viral particles, vectors, isolated cells, and pharmaceutical compositions containing the modulatory polynucleotide for potential therapeutic applications.

Moreover, the patent details methods for inhibiting HTT gene expression in cells, treating Huntington's Disease (HD) in subjects, and administering the AAV vector genome via various routes such as intravenous or intracisternal administration. The treatment methods encompass different stages and forms of HD, including juvenile, early stage, late stage, fully penetrant, incomplete penetrance, and asymptomatic HD. Additionally, the patent suggests combining the AAV vector genome with other therapeutic agents like neuroprotective agents, dopamine-depleting agents, benzodiazepines, and mood stabilizers for enhanced treatment outcomes. The patent also outlines a method for producing the recombinant AAV particle by providing a cell containing the AAV vector genome and a nucleic acid encoding a capsid protein, followed by purification of the viral particle from the cell.

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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

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