BioMarin Pharmaceutical. has filed a patent for improved compositions and methods for efficient lysosomal targeting using the GILT technology. The patent claims a targeted therapeutic fusion protein that includes a lysosomal enzyme and an IGF-II mutein that is resistant to furin cleavage and binds to the human cation-independent mannose-6-phosphate receptor in a mannose-6-phosphate-independent manner. GlobalData’s report on BioMarin Pharmaceutical gives a 360-degree view of the company including its patenting strategy. Buy the report here.
Smarter leaders trust GlobalData
Discover B2B Marketing That Performs
Combine business intelligence and editorial excellence to reach engaged professionals across 36 leading media platforms.
According to GlobalData’s company profile on BioMarin Pharmaceutical, Human telomerase RT biomarker was a key innovation area identified from patents. BioMarin Pharmaceutical's grant share as of September 2023 was 40%. Grant share is based on the ratio of number of grants to total number of patents.
Targeted therapeutic fusion protein for lysosomal enzyme delivery
A recently filed patent (Publication Number: US20230241187A1) describes a targeted therapeutic fusion protein for the treatment of lysosomal storage diseases. The fusion protein consists of a lysosomal enzyme and an IGF-II mutein. The IGF-II mutein has an amino acid sequence that is at least 70% identical to mature human IGF-II and is resistant to furin cleavage. It also binds to the human cation-independent mannose-6-phosphate receptor in a mannose-6-phosphate-independent manner.
The patent claims also describe various mutations and substitutions within the IGF-II mutein that enhance its therapeutic properties. These mutations include amino acid substitutions, deletions, and insertions within specific regions of the amino acid sequence. The IGF-II mutein can also have diminished binding affinity for the insulin receptor compared to naturally-occurring human IGF-II.
The targeted therapeutic fusion protein can be used to treat various lysosomal storage diseases, including Pompe Disease, Fabry Disease, and Gaucher Disease. It can be administered to patients in need of treatment, and a pharmaceutical composition containing the fusion protein is suitable for this purpose.
Methods for producing the targeted therapeutic fusion protein are also described in the patent claims. This includes culturing mammalian cells carrying the nucleic acid encoding the fusion protein under specific conditions to allow for expression of the protein. Additionally, culturing furin-deficient cells carrying the nucleic acid encoding the fusion protein can also be used to produce the targeted therapeutic fusion protein.
Overall, this patent describes a targeted therapeutic fusion protein that shows promise for the treatment of lysosomal storage diseases. The fusion protein combines a lysosomal enzyme with an IGF-II mutein that has enhanced properties, including resistance to furin cleavage and specific binding to the mannose-6-phosphate receptor. The patent claims also cover methods for producing the fusion protein and its use in treating lysosomal storage diseases.
To know more about GlobalData’s detailed insights on BioMarin Pharmaceutical, buy the report here.
Data Insights
From
The gold standard of business intelligence.
Blending expert knowledge with cutting-edge technology, GlobalData’s unrivalled proprietary data will enable you to decode what’s happening in your market. You can make better informed decisions and gain a future-proof advantage over your competitors.
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
