USPTO Patent Grant US12582723B2 for FKRP Protein Polynucleotides
Summary
The USPTO has granted patent US12582723B2 to Genethon for synthetic polynucleotides encoding a human FKRP protein. These polynucleotides are designed to avoid frameshift mutations and are useful for treating pathologies linked to FKRP deficiency or defects in alpha-dystroglycan glycosylation, such as LGMD2I.
What changed
The United States Patent and Trademark Office (USPTO) has issued patent US12582723B2, titled 'Polynucleotides encoding a human FKRP protein,' to Genethon. The patent covers synthetic polynucleotides that encode a human fukutin-related protein (FKRP) and include specific mutations to prevent supplementary transcripts from frameshift start codons. These polynucleotides are intended for therapeutic use, particularly in treating conditions arising from FKRP deficiency or defects in alpha-dystroglycan glycosylation, such as Limb-Girdle Muscular Dystrophy type 2I (LGMD2I).
This patent grant signifies a new intellectual property right for Genethon concerning specific genetic therapies. While this is a patent grant and not a regulatory rule imposing direct compliance obligations on other entities, it may impact research and development activities in the biotechnology and pharmaceutical sectors. Companies operating in this space, particularly those developing therapies for muscular dystrophies or related glycosylation disorders, should be aware of this patent's claims to ensure they do not infringe upon the granted intellectual property rights. No immediate compliance actions are required for entities not directly involved with the patented technology, but legal counsel should be consulted for any potential overlap with ongoing or planned research and development.
Source document (simplified)
Polynucleotides encoding a human FKRP protein
Grant US12582723B2 Kind: B2 Mar 24, 2026
Assignee
GENETHON
Inventors
Isabelle Richard, Evelyne Gicquel-Zouida, William Lostal
Abstract
The present invention concerns synthetic polynucleotides encoding a human fukutin-related protein (FKRP) wherein the synthetic polynucleotides contain at least a mutation avoiding supplementary transcript(s) generated from frameshift start codon(s). The synthetic polynucleotides are useful, especially for treating a pathology linked to a FKRP deficiency or induced by a defect in α-dystroglycan (α-DG) glycosylation, such as LGMD2I.
CPC Classifications
C12N 15/102 C12N 2800/22 C12N 2840/105 A61K 47/549 A61K 48/0091 A61K 48/005 A61K 31/7088
Filing Date
2023-03-02
Application No.
18177446
Claims
9
Named provisions
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