Arcturus Therapeutics. has been granted a patent for mRNA constructs encoding the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein. These constructs can be formulated in lipid formulations and administered via inhalation to potentially treat cystic fibrosis. GlobalData’s report on Arcturus 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 Arcturus Therapeutics, Nanoparticle drug conjugates was a key innovation area identified from patents. Arcturus Therapeutics's grant share as of July 2024 was 33%. Grant share is based on the ratio of number of grants to total number of patents.

Mrna constructs for treating cystic fibrosis via inhalation

Source: United States Patent and Trademark Office (USPTO). Credit: Arcturus Therapeutics Holdings Inc

The granted patent US12070509B2 outlines a series of claims related to messenger RNA (mRNA) encoding the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein. The primary claim specifies that the mRNA must contain an open reading frame (ORF) with a sequence identity of 100% to SEQ ID NO: 100, at least 94% to SEQ ID NO: 101, or at least 95% to any of SEQ ID NOs: 102-105. Additional claims detail the inclusion of specific untranslated regions (5' UTR and 3' UTR), a poly-adenosine tail, and a 5' cap, with options for various chemically-modified nucleotides. The patent also encompasses pharmaceutical compositions that combine the mRNA with specific lipids, particularly lipid nanoparticles designed for effective delivery.

Furthermore, the patent describes methods for treating diseases associated with reduced CFTR activity, such as Cystic Fibrosis, by administering the mRNA or the pharmaceutical compositions via nasal or inhalation routes. The claims specify effective dosing parameters and the composition of lipid nanoparticles, including the proportions of cationic lipids, helper lipids, cholesterol, and PEG-lipid conjugates. This patent represents a significant advancement in the development of mRNA-based therapies aimed at addressing CFTR-related disorders, providing a framework for both the composition of the mRNA and the delivery systems necessary for therapeutic efficacy.

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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