Homology Medicines. has been granted a patent for adeno-associated virus (AAV) compositions designed to express a phenylalanine hydroxylase (PAH) polypeptide, aiming to restore PAH gene function. The patent includes specific AAV genome components and methods for their use and production. GlobalData’s report on Homology Medicines 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 Homology Medicines, Adeno-associated virus vectors was a key innovation area identified from patents. Homology Medicines's grant share as of July 2024 was 9%. Grant share is based on the ratio of number of grants to total number of patents.
Aav compositions for restoring phenylalanine hydroxylase gene function
The patent US12064486B2 describes a novel adeno-associated virus (AAV) genome designed for therapeutic applications. The genome includes a 5' inverted terminal repeat (ITR) from AAV2, a transcriptional regulatory element featuring a human hepatic control region and a human a1-antitrypsin promoter, and a silently altered phenylalanine hydroxylase (PAH) coding sequence that maintains at least 80% sequence identity to a specified reference sequence. Additionally, the genome is capped with a 3' ITR from AAV2. The claims further detail variations of the transcriptional regulatory element, including the potential incorporation of introns and specific nucleotide sequences that enhance regulatory functions.
Moreover, the patent outlines the construction of a replication-defective AAV that incorporates the described genome along with a capsid protein. The capsid protein can be derived from various segments of a specified amino acid sequence. The invention also encompasses pharmaceutical compositions that utilize this AAV, indicating its potential application in gene therapy or other medical treatments. The claims emphasize the importance of sequence identity and specific nucleotide arrangements, which are critical for the functionality and efficacy of the AAV in therapeutic contexts.
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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
