Homology Medicines has been granted a patent for AAV compositions that can express a phenylalanine hydroxylase (PAH) polypeptide to restore gene function. The patent also covers methods of use and packaging systems for creating these compositions. GlobalData’s report on Homology Medicines gives a 360-degree view of the company including its patenting strategy. Buy the report here.
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 May 2024 was 5%. Grant share is based on the ratio of number of grants to total number of patents.
Packaging system for aav expressing pah gene
A recently granted patent (Publication Number: US11951183B2) discloses a packaging system designed for the preparation of a replication-defective Adeno-Associated Virus (AAV). The system comprises three nucleotide sequences: the first sequence encoding AAV Rep proteins, the second sequence encoding an AAV capsid protein, and the third sequence containing a transfer genome with specific elements including inverted terminal repeats, transcriptional regulatory elements, and a modified coding sequence. This innovative system aims to facilitate the production of replication-defective AAV for various applications.
Furthermore, the patent also describes a method for producing replication-defective AAV by introducing the packaging system into a cell to enable AAV production. The system can include multiple vectors, such as a first vector containing the first and second nucleotide sequences, a second vector containing the third nucleotide sequence, and potentially a third vector acting as a helper virus vector. The helper virus can be selected from various viruses like adenovirus or herpes simplex virus, each carrying specific genes to aid in the AAV production process. Overall, this patented packaging system offers a comprehensive approach to efficiently generate replication-defective AAV, showcasing advancements in gene therapy and viral vector technology.
To know more about GlobalData’s detailed insights on Homology Medicines, buy the report here.
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