JCR Pharmaceuticals has filed a patent for methods and compositions to treat a-L-Iduronidase deficiency in the central nervous system (CNS). The patent involves systemic administration of a bifunctional fusion antibody that combines an antibody to a human insulin receptor with a-L-Iduronidase. The therapeutic dose is determined based on the CNS uptake characteristics of the fusion antibody. GlobalData’s report on JCR Pharmaceuticals gives a 360-degree view of the company including its patenting strategy. Buy the report here.

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Data Insights JCR Pharmaceuticals Co Ltd - Company Profile

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According to GlobalData’s company profile on JCR Pharmaceuticals, Nanoparticle drug conjugates was a key innovation area identified from patents. JCR Pharmaceuticals's grant share as of September 2023 was 54%. Grant share is based on the ratio of number of grants to total number of patents.

Treatment for deficiency in a-l-iduronidase in the cns

Source: United States Patent and Trademark Office (USPTO). Credit: JCR Pharmaceuticals Co Ltd

A recently filed patent (Publication Number: US20230279156A1) describes a pharmaceutical composition that includes an alpha-L-iduronidase (IDUA) covalently linked to an antibody fragment. This composition is designed to bind to an endogenous receptor of the blood brain barrier (BBB) transport system. The IDUA in the composition retains at least 30% of its enzymatic activity compared to an unfused IDUA. The enzymatic activity is determined using a specific assay.

The patent claims also specify that the endogenous receptor of the BBB can be either an insulin receptor or a transferrin receptor. The antibody fragment in the composition is a Fab fragment, and its heavy chain and light chain have specific amino acid sequences. The IDUA is covalently linked to the carboxy terminus of the Fab fragment heavy chain, and a linker can be present between the two sequences.

The fusion antibody in the composition has an IDUA specific activity of at least about 200,000 units/mg. The pharmaceutical composition can be sterile filtered and may contain salts, such as mineral acid salts (hydrochlorides, hydrobromides, phosphates, and sulfates) or organic acid salts (acetates, propionates, malonates, and benzoates). Additionally, the composition may include polysorbate-80 in an amount between about 0.01% to about 1% and a carbohydrate additive, such as mannitol, sorbitol, or trehalose.

This patent application presents a novel pharmaceutical composition that combines an alpha-L-iduronidase (IDUA) with an antibody fragment to target the blood brain barrier (BBB) transport system. The composition retains a significant portion of IDUA's enzymatic activity and can bind to either insulin or transferrin receptors of the BBB. The specific amino acid sequences of the antibody fragment's heavy and light chains are provided, along with the option of including a linker between the IDUA and the Fab fragment. The fusion antibody in the composition has a high IDUA specific activity. The composition can be sterile filtered and may contain salts, polysorbate-80, and a carbohydrate additive. This patent application showcases a potential pharmaceutical solution for delivering IDUA to the brain, which could have implications for treating certain diseases or conditions related to IDUA deficiency.

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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

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