Homology Medicines has patented a method for treating phenylketonuria (PKU) by administering a recombinant adeno-associated virus (rAAV) containing specific capsid proteins and a modified PAH coding sequence. The method aims to restore PAH gene function in subjects with PAH gene mutations. 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 April 2024 was 8%. Grant share is based on the ratio of number of grants to total number of patents.
Treatment of phenylketonuria using recombinant adeno-associated virus
A recently granted patent (Publication Number: US11952585B2) discloses a method for treating individuals with phenylketonuria (PKU) by administering a recombinant adeno-associated virus (rAAV) at specific doses. The rAAV used in the method contains an AAV capsid with specific amino acid sequences and a transfer genome with a modified PAH coding sequence. The method involves intravenously administering the rAAV to pediatric or adult subjects, either as a single dose or multiple doses, to address PKU effectively.
Furthermore, the patent details the use of a transcriptional regulatory element capable of mediating transcription in various cells, including those in the liver, kidney, brain, and other organs. The method also includes the administration of a first immunosuppressant, such as prednisolone, prior to the rAAV treatment, with a specific dosing regimen outlined in the claims. If certain liver transaminase levels are elevated during treatment, the dosing regimen may be adjusted accordingly to ensure the subject's safety and efficacy of the treatment. Overall, the patent provides a comprehensive approach to treating PKU using rAAV, highlighting specific genetic and dosing parameters for successful implementation in subjects with this condition.
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