Amicus Therapeutics. has been granted a patent for methods to treat Fabry disease in patients with specific a-galactosidase A mutations. The treatment involves administering migalastat, specifically 150 mg of migalastat hydrochloride every other day, targeting mutations like G334E, N34D, and p.V254del. GlobalData’s report on Amicus 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 Amicus Therapeutics, Human telomerase RT biomarker was a key innovation area identified from patents. Amicus Therapeutics's grant share as of June 2024 was 41%. Grant share is based on the ratio of number of grants to total number of patents.
Treatment methods for fabry disease using migalastat
The granted patent US12042488B2 outlines claims related to a-galactosidase A proteins that incorporate specific mutations amenable to HEK (human embryonic kidney) cell expression. The claims detail various mutations, including G334E, N34D, p.V254del, and N215I, each of which is associated with the protein's binding to migalastat, a pharmacological chaperone. The patent emphasizes that these modified proteins exhibit increased stability compared to their naturally occurring counterparts with the same mutations, potentially enhancing their therapeutic efficacy.
In particular, claims 1 through 8 focus on the a-galactosidase A proteins with the aforementioned mutations and their stability when bound to migalastat. Claims 5 through 8 specifically highlight the increased stability of these proteins, reinforcing the significance of the mutations in improving the protein's characteristics. Additionally, claims 9 and 10 introduce the N215I mutation, further expanding the scope of the invention. Overall, the patent presents a targeted approach to modifying a-galactosidase A proteins to improve their stability and functionality in therapeutic applications, particularly in the context of lysosomal storage disorders.
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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
