Vertex Pharmaceuticals has been granted a patent for compounds useful in treating alpha-1 antitrypsin deficiency (AATD) as per formula (I). The patent covers tautomers, salts, deuterated derivatives, and solid forms of the compounds. GlobalData’s report on Vertex Pharmaceuticals gives a 360-degree view of the company including its patenting strategy. Buy the report here.

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Data Insights Vertex Pharmaceuticals Inc - Company Profile

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According to GlobalData’s company profile on Vertex Pharmaceuticals, Human telomerase RT biomarker was a key innovation area identified from patents. Vertex Pharmaceuticals's grant share as of January 2024 was 44%. Grant share is based on the ratio of number of grants to total number of patents.

Treatment of alpha-1 antitrypsin deficiency with compound of formula (i)

Source: United States Patent and Trademark Office (USPTO). Credit: Vertex Pharmaceuticals Inc

The granted patent (Publication Number: US11884672B2) discloses a compound of formula (I) and its various derivatives, including tautomers, salts, and deuterated derivatives. The compound is defined by specific structural variations denoted by R0 and R1, offering a range of possibilities for molecular modifications. Additionally, the patent covers specific compounds falling under Formula (I-A) to (I-H), each with distinct structural features denoted by Y17 to Y21, allowing for tailored chemical compositions.

Furthermore, the patent extends to the application of the compound in pharmaceutical compositions for treating alpha-1 antitrypsin deficiency. The method involves administering the compound, tautomer, salt, or deuterated derivative to patients with specific mutations in the alpha-1 antitrypsin gene, such as Z or SZ mutations. The disclosed method aims to address the deficiency associated with these mutations, particularly in patients homozygous for Z mutations, highlighting the potential therapeutic implications of the patented compound in precision medicine for genetic 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