Arrowhead Pharmaceuticals. has filed a patent for delivery vehicles that efficiently target and deliver therapeutic payloads to skeletal muscle cells. The vehicles include targeting ligands and PK/PD modulators to facilitate delivery. The payloads, such as RNAi agents, can inhibit gene expression in skeletal muscle cells. The patent also covers pharmaceutical compositions and methods of use for treating diseases and disorders where targeting skeletal muscle cells is desired. GlobalData’s report on Arrowhead Pharmaceuticals 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 Arrowhead Pharmaceuticals, Targeted cancer therapy was a key innovation area identified from patents. Arrowhead Pharmaceuticals's grant share as of September 2023 was 39%. Grant share is based on the ratio of number of grants to total number of patents.

Delivery vehicle for targeted gene inhibition in skeletal muscle cells

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

A recently filed patent (Publication Number: US20230265429A1) describes a delivery vehicle designed to inhibit the expression of a gene in skeletal muscle cells. The delivery vehicle consists of several components, including an RNAi agent, a targeting ligand, and a PK/PD modulator.

The RNAi agent is composed of an antisense strand that is complementary to the mRNA sequence of the gene expressed in skeletal muscle cells, as well as a sense strand that is partially complementary to the antisense strand. The targeting ligand is a polypeptide that has an affinity for a receptor present on the surface of skeletal muscle cells. In particular, the targeting ligand may have an affinity for an integrin receptor, such as the avß6 integrin receptor. The PK/PD modulator, on the other hand, is a molecule that helps regulate the pharmacokinetics and pharmacodynamics of the delivery vehicle. It may contain polyethylene glycol (PEG) units, with at least one PEG unit or even up to ten PEG units.

The patent also describes various formulas for the targeting ligand and the PK/PD modulator, as well as the use of pharmaceutically acceptable salts, such as sodium salt. The delivery vehicle can be used to create compositions and pharmaceutical compositions, which may include a pharmaceutical excipient like water for injection or saline solution.

Furthermore, the patent outlines a method for making the delivery vehicle. This method involves synthesizing the sense and antisense strands of the RNAi agent, annealing them together, and then conjugating the targeting ligand and the PK/PD modulator to the sense or antisense strand.

Overall, this patent presents a novel delivery vehicle that can be used to inhibit the expression of specific genes in skeletal muscle cells. It offers potential applications in the treatment of diseases or disorders affecting skeletal muscle cells, such as muscular dystrophy. The patent provides detailed information on the composition, formulation, and manufacturing process of the delivery vehicle, making it a valuable resource for researchers and pharmaceutical companies working in this field.

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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