Voyager Therapeutics has filed a patent for compositions and methods involving adeno-associated virus (AAV) particles. The patent aims to develop therapies for the prevention and treatment of diseases. 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 September 2023 was 13%. Grant share is based on the ratio of number of grants to total number of patents.

Therapeutic use of adeno-associated virus particles for disease prevention/treatment

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

A recently filed patent (Publication Number: US20230304032A1) describes a method for treating muscular dystrophy using an engineered adeno-associated virus (AAV) particle. The method involves administering a therapeutically effective amount of the AAV particle to a subject. The engineered AAV capsid in the particle has an amino acid sequence that is at least 95% identical to a specific sequence (SEQ ID NO: 1), and the viral genome encodes an RNAi molecule that targets a muscle mRNA.

The patent claims specify various aspects of the method. Claim 78 states that the engineered AAV capsid should have an amino acid sequence that is at least 99% identical to SEQ ID NO: 1. Claim 79 further specifies that the engineered AAV capsid can be encoded by the nucleic acid sequence of SEQ ID NO: 4 or have the amino acid sequence of SEQ ID NO: 3 or SEQ ID NO: 2. Additionally, claim 83 mentions that the engineered AAV capsid can be encoded by the nucleic acid sequence of SEQ ID NO: 1809 or have the amino acid sequence of SEQ ID NO: 1.

The RNAi molecule used in the method can be an siRNA, a dsRNA, or an miRNA, as stated in claim 85. However, claim 86 specifies that the RNAi molecule is an siRNA. The AAV particle is capable of transducing muscle cells, heart cells, brain cells, or a combination thereof, as mentioned in claim 87. Claim 88 further specifies that the AAV particle primarily transduces muscle cells. These muscle cells can be skeletal muscle cells or cardiomyocytes, as stated in claim 89.

The method can be administered to the subject through intramuscular delivery or intravenous delivery, as mentioned in claim 90. Another aspect of the patent involves a method for decreasing the expression of a gene associated with muscular dystrophy in a cell. This method is similar to the previous method but focuses on targeting the mRNA of the gene of interest, as stated in claim 91.

Overall, the patent describes a method for treating muscular dystrophy by administering an engineered AAV particle with a specific capsid and viral genome. The method aims to decrease the expression of genes associated with muscular dystrophy in muscle cells through the use of RNAi molecules. The patent claims provide further details and variations of the method, including specific amino acid sequences, delivery methods, and cell types targeted by the AAV particle.

To know more about GlobalData’s detailed insights on Voyager Therapeutics, buy the report here.

This content was updated on 25 January 2024

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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